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Ratko TA, Belinson SE, Brown HM, et al. Hematopoietic Stem-Cell Transplantation in the Pediatric Population [Internet]. Rockville (MD): Agency for Healthcare Research and Quality (US); 2012 Feb. (Comparative Effectiveness Reviews, No. 48.)

  • This publication is provided for historical reference only and the information may be out of date.

This publication is provided for historical reference only and the information may be out of date.

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Hematopoietic Stem-Cell Transplantation in the Pediatric Population [Internet].

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References

1.
Devetten M, Armitage JO. Hematopoietic cell transplantation: progress and obstacles. Ann Oncol. 2007 Sep;18(9):1450–6. [PubMed: 17355954]
2.
Shimoni A, Nagler A. Non-myeloablative stem-cell transplantation in the treatment of malignant and non-malignant disorders. Isr Med Assoc J. 2002 Apr;4(4):272–9. [PubMed: 12001702]
3.
Urbano-Ispizua A, Schmitz N, de Witte T, et al. Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders: definitions and current practice in Europe. Bone Marrow Transplant. 2002 Apr;29(8):639–46. [PubMed: 12180107]
4.
Pelus LM. Peripheral blood stem cell mobilization: new regimens, new cells, where do we stand. Curr Opin Hematol. 2008 Jul;15(4):285–92. [PMC free article: PMC2806229] [PubMed: 18536564]
5.
Barrett AJ, Savani BN. Stem cell transplantation with reduced-intensity conditioning regimens: a review of ten years experience with new transplant concepts and new therapeutic agents. Leukemia. 2006 Oct;20(10):1661–72. [PubMed: 16871277]
6.
Sandmaier BM, Mackinnon S, Childs RW. Reduced intensity conditioning for allogeneic hematopoietic cell transplantation: current perspectives. Biol Blood Marrow Transplant. 2007 Jan;13(1 Suppl 1):87–97. [PMC free article: PMC1829153] [PubMed: 17222778]
7.
Barfield RC, Kasow KA, Hale GA. Advances in pediatric hematopoietic stem cell transplantation. Cancer Biol Ther. 2008 Oct;7(10):1533–9. [PubMed: 18927494]
8.
Eiser C. Practitioner review: long-term consequences of childhood cancer. J Child Psychol Psychiatry. 1998 Jul;39(5):621–33. [PubMed: 9690926]
9.
Locatelli F, Giorgiani G, Di-Cesare-Merlone A, et al. The changing role of stem cell transplantation in childhood. Bone Marrow Transplant. 2008 Jun;41 Suppl 2:S3–7. [PubMed: 18545240]
10.
Reulen RC, Winter DL, Frobisher C, et al. Long-term cause-specific mortality among survivors of childhood cancer. JAMA. 2010 Jul 14;304(2):172–9. [PubMed: 20628130]
11.
Liberati A, Altman DG, Tetzlaff J, et al. The PRISMA statement for reporting systematic reviews and meta-analyses of studies that evaluate health care interventions: explanation and elaboration. Ann Intern Med. 2009 Aug 18;151(4):W65–94. [PubMed: 19622512]
12.
Instutute of Medicine Committee on Accelerating Rare Diseases Research and Orphan Product Development: Board on Health Sciences Policy. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: National Academies Press; 2011. [cited 2011 January]. http://books​.nap.edu/openbook​.php?record_id=12953. [PubMed: 21796826]
13.
Owens DK, Lohr KN, Atkins D, et al. AHRQ series paper 5: grading the strength of a body of evidence when comparing medical interventions--agency for healthcare research and quality and the effective health-care program. J Clin Epidemiol. 2010 May;63(5):513–23. [PubMed: 19595577]
14.
Guyatt GH, Oxman AD, Vist GE, et al. GRADE: an emerging consensus on rating quality of evidence and strength of recommendations. BMJ. 2008 Apr 26;336(7650):924–6. [PMC free article: PMC2335261] [PubMed: 18436948]
15.
National Cancer Institute Physician Data Query. Childhood acute lymphoblastic leukemia treatment. 2010. [updated 06/29/2010]; www​.cancer.gov/cancertopics​/pdq/treatment​/childALL/healthprofessional/allpages.
16.
Redaelli A, Laskin BL, Stephens JM, et al. A systematic literature review of the clinical and epidemiological burden of acute lymphoblastic leukaemia (ALL). Eur J Cancer Care (Engl). 2005 Mar;14(1):53–62. [PubMed: 15698386]
17.
Gaynon PS. Childhood acute lymphoblastic leukaemia and relapse. Br J Haematol. 2005 Dec;131(5):579–87. [PubMed: 16351633]
18.
Jeha S, Pui CH. Risk-adapted treatment of pediatric acute lymphoblastic leukemia. Hematol Oncol Clin North Am. 2009 Oct;23(5):973–90. v. [PubMed: 19825448]
19.
Krance R. Transplantation for children with acute lymphoblastic leukemia. Bone Marrow Transplant. 2008 Aug;42 Suppl 1:S25–S7. [PubMed: 18724293]
20.
Pui CH, Evans WE. Treatment of acute lymphoblastic leukemia. N Engl J Med. 2006 Jan 12;354(2):166–78. [PubMed: 16407512]
21.
Pulsipher MA, Bader P, Klingebiel T, et al. Allogeneic transplantation for pediatric acute lymphoblastic leukemia: the emerging role of peritransplantation minimal residual disease/chimerism monitoring and novel chemotherapeutic, molecular, and immune approaches aimed at preventing relapse. Biol Blood Marrow Transplant. 2009 Jan;15(1 Suppl):62–71. [PubMed: 19147081]
22.
Schrauder A, von Stackelberg A, Schrappe M, et al. Allogeneic hematopoietic SCT in children with ALL: current concepts of ongoing prospective SCT trials. Bone Marrow Transplant. 2008 Jun;41 Suppl 2:S71–4. [PubMed: 18545248]
23.
Stanulla M, Schrappe M. Treatment of childhood acute lymphoblastic leukemia. Semin Hematol. 2009 Jan;46(1):52–63. [PubMed: 19100368]
24.
Pui CH, Campana D, Evans WE. Childhood acute lymphoblastic leukaemia--current status and future perspectives. Lancet Oncol. 2001 Oct;2(10):597–607. [PubMed: 11902549]
25.
Pieters R, Schrappe M, De Lorenzo P, et al. A treatment protocol for infants younger than 1 year with acute lymphoblastic leukaemia (Interfant-99): an observational study and a multicentre randomised trial. Lancet. 2007 Jul 21;370(9583):240–50. [PubMed: 17658395]
26.
Hahn T, Wall D, Camitta B, et al. The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of acute lymphoblastic leukemia in children: an evidence-based review. Biol Blood Marrow Transplant. 2005 Nov;11(11):823–61. [PubMed: 16275588]
27.
Eapen M, Raetz E, Zhang MJ, et al. Outcomes after HLA-matched sibling transplantation or chemotherapy in children with B-precursor acute lymphoblastic leukemia in a second remission: a collaborative study of the Children's Oncology Group and the Center for International Blood and Marrow Transplant Research. Blood. 2006 Jun 15;107(12):4961–7. [PMC free article: PMC1895819] [PubMed: 16493003]
28.
Baker KS, Armenian S, Bhatia S. Long-term consequences of hematopoietic stem cell transplantation: current state of the science. Biol Blood Marrow Transplant. 2010 Jan;16(1 Suppl):S90–6. [PMC free article: PMC2832710] [PubMed: 19782145]
29.
National Cancer Institute Physician Data Query. Late effects of treatment of childhood cancer. 2010. [updated 08/06/2010; cited 2010 October]; www​.cancer.gov/cancertopics​/pdq/treatment​/lateeffects/HealthProfessional.
30.
Shenoy S, Smith FO. Hematopoietic stem cell transplantation for childhood malignancies of myeloid origin. Bone Marrow Transplant. 2008 Jan;41(2):141–8. [PubMed: 18176616]
31.
Landier W, Bhatia S, Eshelman DA, et al. Development of risk-based guidelines for pediatric cancer survivors: the Children's Oncology Group Long-Term Follow-Up Guidelines from the Children's Oncology Group Late Effects Committee and Nursing Discipline. J Clin Oncol. 2004 Dec 15;22(24):4979–90. [PubMed: 15576413]
32.
Pasquini MC, Wang Z. Current use and outcome of hematopoietic stem cell transplantation: CIBMTR Summary Slides, 2010. Center for International Blood & Marrow Transplant and Research (CIBMTR); 2010.
33.
Miano M, Cancedda R, Hartmann O, et al. Survey on haematopoietic stem cell transplantation for children in Europe. Bone Marrow Transplant. 2005 Mar;35 Suppl 1:S3–8. [PubMed: 15812526]
34.
Mehta PA, Davies SM. Allogeneic transplantation for childhood ALL. Bone Marrow Transplant. 2008 Jan;41(2):133–9. [PubMed: 17994118]
35.
Bleakley M, Lau L, Shaw PJ, et al. Bone marrow transplantation for paediatric AML in first remission: a systematic review and meta-analysis. Bone Marrow Transplant. 2002 May;29(10):843–52. [PubMed: 12058234]
36.
Alonzo TA, Wells RJ, Woods WG, et al. Postremission therapy for children with acute myeloid leukemia: the children's cancer group experience in the transplant era. Leukemia. 2005 Jun;19(6):965–70. [PubMed: 15830007]
37.
Woods WG. Curing childhood acute myeloid leukemia (AML) at the half-way point: promises to keep and miles to go before we sleep. Pediatr Blood Cancer. 2006 May 1;46(5):565–9. [PubMed: 16261562]
38.
Bierings M, Nachman JB, Zwaan CM. Stem cell transplantation in pediatric leukemia and myelodysplasia: state of the art and current challenges. Curr Stem Cell Res Ther. 2007 Jan;2(1):53–63. [PubMed: 18240454]
39.
Amadori S, Testi AM, Arico M, et al. Prospective comparative study of bone marrow transplantation and postremission chemotherapy for childhood acute myelogenous leukemia. The Associazione Italiana Ematologia ed Oncologia Pediatrica Cooperative Group. J Clin Oncol. 1993 Jun;11(6):1046–54. [PubMed: 8501490]
40.
Michel G, Leverger G, Leblanc T, et al. Allogeneic bone marrow transplantation vs aggressive post-remission chemotherapy for children with acute myeloid leukemia in first complete remission. A prospective study from the French Society of Pediatric Hematology and Immunology (SHIP). Bone Marrow Transplant. 1996 Feb;17(2):191–6. [PubMed: 8640165]
41.
Shaw PJ, Bergin ME, Burgess MA, et al. Childhood acute myeloid leukemia: outcome in a single center using chemotherapy and consolidation with busulfan/cyclophosphamide for bone marrow transplantation. J Clin Oncol. 1994 Oct;12(10):2138–45. [PubMed: 7931485]
42.
Stevens RF, Hann IM, Wheatley K, et al. Marked improvements in outcome with chemotherapy alone in paediatric acute myeloid leukemia: results of the United Kingdom Medical Research Council's 10th AML trial. MRC Childhood Leukaemia Working Party. Br J Haematol. 1998 Apr;101(1):130–40. [PubMed: 9576193]
43.
Wells RJ, Woods WG, Buckley JD, et al. Treatment of newly diagnosed children and adolescents with acute myeloid leukemia: a Childrens Cancer Group study. J Clin Oncol. 1994 Nov;12(11):2367–77. [PubMed: 7964952]
44.
Woods WG, Kobrinsky N, Buckley JD, et al. Timed-sequential induction therapy improves postremission outcome in acute myeloid leukemia: a report from the Children's Cancer Group. Blood. 1996 Jun 15;87(12):4979–89. [PubMed: 8652810]
45.
Ravindranath Y, Yeager AM, Chang MN, et al. Autologous bone marrow transplantation versus intensive consolidation chemotherapy for acute myeloid leukemia in childhood. Pediatric Oncology Group. N Engl J Med. 1996 May 30;334(22):1428–34. [PubMed: 8618581]
46.
Lange BJ, Dinndorf P, Smith FO, et al. Pilot study of idarubicin-based intensive-timing induction therapy for children with previously untreated acute myeloid leukemia: Children's Cancer Group Study 2941. J Clin Oncol. 2004 Jan 1;22(1):150–6. [PubMed: 14701777]
47.
Smith FO, Alonzo TA, Gerbing RB, et al. Long-term results of children with acute myeloid leukemia: a report of three consecutive Phase III trials by the Children's Cancer Group: CCG 251, CCG 213 and CCG 2891. Leukemia. 2005 Dec;19(12):2054–62. [PubMed: 16136168]
48.
Woods WG, Kobrinsky N, Buckley J, et al. Intensively timed induction therapy followed by autologous or allogeneic bone marrow transplantation for children with acute myeloid leukemia or myelodysplastic syndrome: a Childrens Cancer Group pilot study. J Clin Oncol. 1993 Aug;11(8):1448–57. [PubMed: 8336184]
49.
Arico M, Valsecchi MG, Camitta B, et al. Outcome of treatment in children with Philadelphia chromosome-positive acute lymphoblastic leukemia. N Engl J Med. 2000 Apr 6;342(14):998–1006. [PubMed: 10749961]
50.
Jacobsohn DA, Hewlett B, Morgan E, et al. Favorable outcome for infant acute lymphoblastic leukemia after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2005 Dec;11(12):999–1005. [PubMed: 16338622]
51.
Kosaka Y, Koh K, Kinukawa N, et al. Infant acute lymphoblastic leukemia with MLL gene rearrangements: outcome following intensive chemotherapy and hematopoietic stem cell transplantation. Blood. 2004 Dec 1;104(12):3527–34. [PubMed: 15297313]
52.
Sanders JE, Im HJ, Hoffmeister PA, et al. Allogeneic hematopoietic cell transplantation for infants with acute lymphoblastic leukemia. Blood. 2005 May 1;105(9):3749–56. [PMC free article: PMC1895011] [PubMed: 15637143]
53.
Hilden JM, Dinndorf PA, Meerbaum SO, et al. Analysis of prognostic factors of acute lymphoblastic leukemia in infants: report on CCG 1953 from the Children's Oncology Group. Blood. 2006 Jul 15;108(2):441–51. [PMC free article: PMC1895499] [PubMed: 16556894]
54.
Ribera JM, Ortega JJ, Oriol A, et al. Comparison of intensive chemotherapy, allogeneic, or autologous stem-cell transplantation as postremission treatment for children with very high risk acute lymphoblastic leukemia: PETHEMA ALL-93 Trial. J Clin Oncol. 2007 Jan 1;25(1):16–24. [PubMed: 17194902]
55.
Satwani P, Sather H, Ozkaynak F, et al. Allogeneic bone marrow transplantation in first remission for children with ultra-high-risk features of acute lymphoblastic leukemia: A children's oncology group study report. Biol Blood Marrow Transplant. 2007 Feb;13(2):218–27. [PMC free article: PMC2731715] [PubMed: 17241927]
56.
Schrauder A, Reiter A, Gadner H, et al. Superiority of allogeneic hematopoietic stem-cell transplantation compared with chemotherapy alone in high-risk childhood T-cell acute lymphoblastic leukemia: results from ALL-BFM 90 and 95. J Clin Oncol. 2006 Dec 20;24(36):5742–9. [PubMed: 17179108]
57.
Boulad F, Steinherz P, Reyes B, et al. Allogeneic bone marrow transplantation versus chemotherapy for the treatment of childhood acute lymphoblastic leukemia in second remission: a single-institution study. J Clin Oncol. 1999 Jan;17(1):197–207. [PubMed: 10458234]
58.
Eapen M, Zhang MJ, Devidas M, et al. Outcomes after HLA-matched sibling transplantation or chemotherapy in children with acute lymphoblastic leukemia in a second remission after an isolated central nervous system relapse: a collaborative study of the Children's Oncology Group and the Center for International Blood and Marrow Transplant Research. Leukemia. 2008 Feb;22(2):281–6. [PubMed: 18033318]
59.
Einsiedel HG, von Stackelberg A, Hartmann R, et al. Long-term outcome in children with relapsed ALL by risk-stratified salvage therapy: results of trial acute lymphoblastic leukemia-relapse study of the Berlin-Frankfurt-Munster Group 87. J Clin Oncol. 2005 Nov 1;23(31):7942–50. [PubMed: 16258094]
60.
Gaynon PS, Harris RE, Altman AJ, et al. Bone marrow transplantation versus prolonged intensive chemotherapy for children with acute lymphoblastic leukemia and an initial bone marrow relapse within 12 months of the completion of primary therapy: Children's Oncology Group study CCG-1941. J Clin Oncol. 2006 Jul 1;24(19):3150–6. [PubMed: 16717292]
61.
Cwynarski K, Roberts IA, Iacobelli S, et al. Stem cell transplantation for chronic myeloid leukemia in children. Blood. 2003 Aug 15;102(4):1224–31. [PubMed: 12714525]
62.
Woods WG, Barnard DR, Alonzo TA, et al. Prospective study of 90 children requiring treatment for juvenile myelomonocytic leukemia or myelodysplastic syndrome: a report from the Children's Cancer Group. J Clin Oncol. 2002 Jan 15;20(2):434–40. [PubMed: 11786571]
63.
Locatelli F, Nollke P, Zecca M, et al. Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial. Blood. 2005 Jan 1;105(1):410–9. [PubMed: 15353481]
64.
National Cancer Institute Physician Data Query. Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment. 2010. [updated 08/09/2010]; www​.cancer.gov/cancertopics​/pdq/treatment​/childAML/healthprofessional/allpages.
65.
Arceci RJ, Aplenc R. Acute myelogenous leukemia in children. In: Greer JP, Foerster J, Rodgers GM, editors. Wintrobe's Clinical Hematology. Philadelphia: Lippincott Williams & Wilkins; 2009. pp. 1918–37.
66.
Meshinchi S, Arceci RJ. Prognostic factors and risk-based therapy in pediatric acute myeloid leukemia. Oncologist. 2007 Mar;12(3):341–55. [PubMed: 17405900]
67.
Kaspers GJ, Zwaan CM. Pediatric acute myeloid leukemia: towards high-quality cure of all patients. Haematologica. 2007 Nov;92(11):1519–32. [PubMed: 18024401]
68.
Oliansky DM, Rizzo JD, Aplan PD, et al. The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of acute myeloid leukemia in children: an evidence-based review. Biol Blood Marrow Transplant. 2007 Jan;13(1):1–25. [PubMed: 17222748]
69.
Klingebiel T, Reinhardt D, Bader P. Place of HSCT in treatment of childhood AML. Bone Marrow Transplant. 2008 Oct;42 Suppl 2:S7–9. [PubMed: 18978749]
70.
Kolb HJ, Schattenberg A, Goldman JM, et al. Graft-versus-leukemia effect of donor lymphocyte transfusions in marrow grafted patients. Blood. 1995 Sep 1;86(5):2041–50. [PubMed: 7655033]
71.
O'Dwyer ME, Mauro MJ, Kurilik G, et al. The impact of clonal evolution on response to imatinib mesylate (STI571) in accelerated phase CML. Blood. 2002 Sep 1;100(5):1628–33. [PubMed: 12176881]
72.
Millot F, Esperou H, Bordigoni P, et al. Allogeneic bone marrow transplantation for chronic myeloid leukemia in childhood: a report from the Societe Francaise de Greffe de Moelle et de Therapie Cellulaire (SFGM-TC). Bone Marrow Transplant. 2003 Nov;32(10):993–9. [PubMed: 14595387]
73.
Weisdorf DJ, Anasetti C, Antin JH, et al. Allogeneic bone marrow transplantation for chronic myelogenous leukemia: comparative analysis of unrelated versus matched sibling donor transplantation. Blood. 2002 Mar 15;99(6):1971–7. [PubMed: 11877268]
74.
Fernandez HF, Kharfan-Dabaja MA. Tyrosine kinase inhibitors and allogeneic hematopoietic cell transplantation for chronic myeloid leukemia: targeting both therapeutic modalities. Cancer Control. 2009 Apr;16(2):153–7. [PubMed: 19337201]
75.
Suttorp M. Innovative approaches of targeted therapy for CML of childhood in combination with paediatric haematopoietic SCT. Bone Marrow Transplant. 2008 Oct;42 Suppl 2:S40–6. [PubMed: 18978743]
76.
Hasle H, Kerndrup G, Jacobsen BB. Childhood myelodysplastic syndrome in Denmark: incidence and predisposing conditions. Leukemia. 1995 Sep;9(9):1569–72. [PubMed: 7658725]
77.
Aul C, Gattermann N, Schneider W. Age-related incidence and other epidemiological aspects of myelodysplastic syndromes. Br J Haematol. 1992 Oct;82(2):358–67. [PubMed: 1419819]
78.
Arico M, Biondi A, Pui CH. Juvenile myelomonocytic leukemia. Blood. 1997 Jul 15;90(2):479–88. [PubMed: 9226148]
79.
Smith FO, King R, Nelson G, et al. Unrelated donor bone marrow transplantation for children with juvenile myelomonocytic leukaemia. Br J Haematol. 2002 Mar;116(3):716–24. [PubMed: 11849238]
80.
Bradley MB, Cairo MS. Stem cell transplantation for pediatric lymphoma: past, present and future. Bone Marrow Transplant. 2008 Jan;41(2):149–58. [PubMed: 18084337]
81.
National Cancer Institute Physician Data Query. Childhood Hodgkin lymphoma treatment. 2010. [updated 08/14/2009; cited 2010 October]; www​.cancer.gov/cancertopics​/pdq/treatment​/childhodgkins/healthprofessional.
82.
Baker KS, Gordon BG, Gross TG, et al. Autologous hematopoietic stem-cell transplantation for relapsed or refractory Hodgkin's disease in children and adolescents. J Clin Oncol. 1999 Mar;17(3):825–31. [PubMed: 10071273]
83.
Lieskovsky YE, Donaldson SS, Torres MA, et al. High-dose therapy and autologous hematopoietic stem-cell transplantation for recurrent or refractory pediatric Hodgkin's disease: results and prognostic indices. J Clin Oncol. 2004 Nov 15;22(22):4532–40. [PubMed: 15542804]
84.
Stoneham S, Ashley S, Pinkerton CR, et al. Outcome after autologous hemopoietic stem cell transplantation in relapsed or refractory childhood Hodgkin disease. J Pediatr Hematol Oncol. 2004 Nov;26(11):740–5. [PubMed: 15543009]
85.
Verdeguer A, Pardo N, Madero L, et al. Autologous stem cell transplantation for advanced Hodgkin's disease in children. Spanish group for BMT in children (GETMON), Spain. Bone Marrow Transplant. 2000 Jan;25(1):31–4. [PubMed: 10654011]
86.
Williams CD, Goldstone AH, Pearce R, et al. Autologous bone marrow transplantation for pediatric Hodgkin's disease: a case-matched comparison with adult patients by the European Bone Marrow Transplant Group Lymphoma Registry. J Clin Oncol. 1993 Nov;11(11):2243–9. [PubMed: 8229140]
87.
National Comprehensive Cancer Network. Hodgkin Lymphoma. 2010. [cited 2010 October]; V.1.2010: www​.nccn.org/professionals​/physician_gls/PDF/hodgkins.pdf.
88.
Linch DC, Winfield D, Goldstone AH, et al. Dose intensification with autologous bone-marrow transplantation in relapsed and resistant Hodgkin's disease: results of a BNLI randomised trial. Lancet. 1993 Apr 24;341(8852):1051–4. [PubMed: 8096958]
89.
Schmitz N, Pfistner B, Sextro M, et al. Aggressive conventional chemotherapy compared with high-dose chemotherapy with autologous haemopoietic stem-cell transplantation for relapsed chemosensitive Hodgkin's disease: a randomised trial. Lancet. 2002 Jun 15;359(9323):2065–71. [PubMed: 12086759]
90.
National Cancer Institute Physician Data Query. Childhood Non-Hodgkin Lymphoma Treatment. 2010. [updated 08/14/2009; cited 2010 October]; www​.cancer.gov/cancertopics​/pdq/treatment​/child-non-hodgkins​/healthprofessional/allpages.
91.
Gross TG, Termuhlen AM. Pediatric non-Hodgkin's lymphoma. Curr Oncol Rep. 2007 Nov;9(6):459–65. [PubMed: 17991353]
92.
Levine JE, Harris RE, Loberiza FR Jr, et al. A comparison of allogeneic and autologous bone marrow transplantation for lymphoblastic lymphoma. Blood. 2003 Apr 1;101(7):2476–82. [PubMed: 12456505]
93.
Bureo E, Ortega JJ, Munoz A, et al. Bone marrow transplantation in 46 pediatric patients with non-Hodgkin's lymphoma. Spanish Working Party for Bone Marrow Transplantation in Children. Bone Marrow Transplant. 1995 Mar;15(3):353–9. [PubMed: 7599558]
94.
Kobrinsky NL, Sposto R, Shah NR, et al. Outcomes of treatment of children and adolescents with recurrent non-Hodgkin's lymphoma and Hodgkin's disease with dexamethasone, etoposide, cisplatin, cytarabine, and l-asparaginase, maintenance chemotherapy, and transplantation: Children's Cancer Group Study CCG-5912. J Clin Oncol. 2001 May 1;19(9):2390–6. [PubMed: 11331317]
95.
Loiseau HA, Hartmann O, Valteau D, et al. High-dose chemotherapy containing busulfan followed by bone marrow transplantation in 24 children with refractory or relapsed non-Hodgkin's lymphoma. Bone Marrow Transplant. 1991 Dec;8(6):465–72. [PubMed: 1790426]
96.
Philip T, Hartmann O, Biron P, et al. High-dose therapy and autologous bone marrow transplantation in partial remission after first-line induction therapy for diffuse non-Hodgkin's lymphoma. J Clin Oncol. 1988 Jul;6(7):1118–24. [PubMed: 3292712]
97.
Woessmann W, Peters C, Lenhard M, et al. Allogeneic haematopoietic stem cell transplantation in relapsed or refractory anaplastic large cell lymphoma of children and adolescents--a Berlin-Frankfurt-Munster group report. Br J Haematol. 2006 Apr;133(2):176–82. [PubMed: 16611309]
98.
Won SC, Han JW, Kwon SY, et al. Autologous peripheral blood stem cell transplantation in children with non-Hodgkin's lymphoma: A report from the Korean society of pediatric hematology-oncology. Ann Hematol. 2006 Nov;85(11):787–94. [PubMed: 16932891]
99.
Fanin R, Ruiz de Elvira MC, Sperotto A, et al. Autologous stem cell transplantation for T and null cell CD30-positive anaplastic large cell lymphoma: analysis of 64 adult and paediatric cases reported to the European Group for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant. 1999 Mar;23(5):437–42. [PubMed: 10100556]
100.
Ladenstein R, Pearce R, Hartmann O, et al. High-dose chemotherapy with autologous bone marrow rescue in children with poor-risk Burkitt's lymphoma: a report from the European Lymphoma Bone Marrow Transplantation Registry. Blood. 1997 Oct 15;90(8):2921–30. [PubMed: 9376572]
101.
Hahn T, Wolff SN, Czuczman M, et al. The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of diffuse large cell B-cell non-Hodgkin's lymphoma: an evidence-based review. Biol Blood Marrow Transplant. 2001;7(6):308–31. [PubMed: 11464975]
102.
National Comprehensive Cancer Network. Non-Hodgkin's Lymphoma. 2010. [cited 2010 October]; V.1.2010:[www​.nccn.org/professionals​/physician_gls/PDF/nhl.pdf.
103.
Park JR, Eggert A, Caron H. Neuroblastoma: biology, prognosis, and treatment. Pediatr Clin North Am. 2008 Feb;55(1):97–120. x. [PubMed: 18242317]
104.
National Cancer Institute Physician Data Query. Neuroblastoma Treatment. 2010. [updated 02/11/2010; cited 2010 May]; www​.cancer.gov/cancertopics​/pdq/treatment​/neuroblastoma/healthprofessional​/allpages.
105.
Berthold F, Boos J, Burdach S, et al. Myeloablative megatherapy with autologous stem-cell rescue versus oral maintenance chemotherapy as consolidation treatment in patients with high-risk neuroblastoma: a randomised controlled trial. Lancet Oncol. 2005 Sep;6(9):649–58. [PubMed: 16129365]
106.
Fish JD, Grupp SA. Stem cell transplantation for neuroblastoma. Bone Marrow Transplant. 2008 Jan;41(2):159–65. [PMC free article: PMC2892221] [PubMed: 18037943]
107.
Matthay KK, Villablanca JG, Seeger RC, et al. Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. Children's Cancer Group. N Engl J Med. 1999 Oct 14;341(16):1165–73. [PubMed: 10519894]
108.
Pritchard J, Cotterill SJ, Germond SM, et al. High dose melphalan in the treatment of advanced neuroblastoma: results of a randomised trial (ENSG-1) by the European Neuroblastoma Study Group. Pediatr Blood Cancer. 2005 Apr;44(4):348–57. [PubMed: 15546135]
109.
Laverdiere C, Liu Q, Yasui Y, et al. Long-term outcomes in survivors of neuroblastoma: a report from the Childhood Cancer Survivor Study. J Natl Cancer Inst. 2009 Aug 19;101(16):1131–40. [PMC free article: PMC2728747] [PubMed: 19648511]
110.
Yalcin B, Kremer LC, Caron HN, et al. High-dose chemotherapy and autologous haematopoietic stem cell rescue for children with high-risk neuroblastoma. Cochrane Database Syst Rev. 2010;5:CD006301. [PubMed: 20464740]
111.
Matthay KK, Reynolds CP, Seeger RC, et al. Long-term results for children with high-risk neuroblastoma treated on a randomized trial of myeloablative therapy followed by 13-cis-retinoic acid: a children's oncology group study. J Clin Oncol. 2009 Mar 1;27(7):1007–13. [PMC free article: PMC2738615] [PubMed: 19171716]
112.
Garaventa A, Parodi S, De Bernardi B, et al. Outcome of children with neuroblastoma after progression or relapse. A retrospective study of the Italian neuroblastoma registry. Eur J Cancer. 2009 Nov;45(16):2835–42. [PubMed: 19616426]
113.
Ladenstein R, Potschger U, Hartman O, et al. 28 years of high-dose therapy and SCT for neuroblastoma in Europe: lessons from more than 4000 procedures. Bone Marrow Transplant. 2008 Jun;41 Suppl 2:S118–27. [PubMed: 18545256]
114.
De Giorgi U, Rosti G, Slavin S, et al. Salvage high-dose chemotherapy for children with extragonadal germ-cell tumours. Br J Cancer. 2005 Aug 22;93(4):412–7. [PMC free article: PMC2361583] [PubMed: 16106248]
115.
National Cancer Institute Physician Data Query. Childhood Extracranial Germ Cell Tumors Treatment. 2010. [updated 04/29/2010]; www​.cancer.gov/cancertopics​/pdq/treatment​/extracranial-germ-cell​/healthprofessional/allpages.
116.
Bernstein L, Smith MA, Liu L, et al. Germ Cell, Trophoblastic and Other Gonadal Neoplasms. In: Ries LAG, Smith MA, Gurney JG, Linet M, Tamra T, Young JL, et al., editors. Cancer Incidence and Survival among Children and Adolescents: United States SEER Program 1975-1995. NIH Pub. No. 99-4649 ed. Bethesda, MD: National Cancer Institute, SEER Program; 1999.
117.
National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology. Ovarian Cancer. 2010. [updated V.2.2010]; www​.nccn.org/professionals​/physician_gls/PDF/ovarian.pdf. [PubMed: 20202462]
118.
National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology. Testicular Cancer. 2010. [updated V.2.2010]; www​.nccn.org/professionals​/physician_gls/PDF/testicular.pdf. [PubMed: 19555582]
119.
Lazarus HM, Stiff PJ, Carreras J, et al. Utility of single versus tandem autotransplants for advanced testes/germ cell cancer: a center for international blood and marrow transplant research (CIBMTR) analysis. Biol Blood Marrow Transplant. 2007 Jul;13(7):778–89. [PubMed: 17580256]
120.
Agarwal R, Dvorak CC, Stockerl-Goldstein KE, et al. High-dose chemotherapy followed by stem cell rescue for high-risk germ cell tumors: the Stanford experience. Bone Marrow Transplant. 2009 Apr;43(7):547–52. [PubMed: 18997833]
121.
National Cancer Institute Clinical Trials (PDQ). Busulfan, Melphalan, Topotecan Hydrochloride, and a Stem Cell Transplant in Treating Patients With Newly Diagnosed or Relapsed Solid Tumor. 2010. [cited 2010 May]; Clinical trial protocol]. www​.cancer.gov/search​/ViewClinicalTrials​.aspx?cdrid=589296&version​=HealthProfessional&protocolsearchid​=7788376.
122.
National Cancer Institute Physician Data Query. Childhood Central Nervous System Embryonal Tumors Treatment. 2010. [updated 05/20/2010; cited 2010 June]; www​.cancer.gov/cancertopics​/pdq/treatment​/childCNSembryonal/healthprofessional​/allpages.
123.
MacDonald TJ. Aggressive infantile embryonal tumors. J Child Neurol. 2008 Oct;23(10):1195–204. [PMC free article: PMC3674573] [PubMed: 18952586]
124.
Mueller S, Chang S. Pediatric brain tumors: current treatment strategies and future therapeutic approaches. Neurotherapeutics. 2009 Jul;6(3):570–86. [PMC free article: PMC5084192] [PubMed: 19560746]
125.
Lafay-Cousin L, Strother D. Current treatment approaches for infants with malignant central nervous system tumors. Oncologist. 2009 Apr;14(4):433–44. [PubMed: 19342475]
126.
National Cancer Institute Physician Data Query. Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment. 2010. www​.cancer.gov/cancertopics​/pdq/treatment​/child-CNS-ATRT/healthprofessional​/allpages.
127.
Butturini AM, Jacob M, Aguajo J, et al. High-dose chemotherapy and autologous hematopoietic progenitor cell rescue in children with recurrent medulloblastoma and supratentorial primitive neuroectodermal tumors: the impact of prior radiotherapy on outcome. Cancer. 2009 Jul 1;115(13):2956–63. [PubMed: 19402050]
128.
Cheuk DK, Lee TL, Chiang AK, et al. Autologous hematopoietic stem cell transplantation for high-risk brain tumors in children. J Neurooncol. 2008 Feb;86(3):337–47. [PMC free article: PMC7100104] [PubMed: 17906911]
129.
Grodman H, Wolfe L, Kretschmar C. Outcome of patients with recurrent medulloblastoma or central nervous system germinoma treated with low dose continuous intravenous etoposide along with dose-intensive chemotherapy followed by autologous hematopoietic stem cell rescue. Pediatr Blood Cancer. 2009 Jul;53(1):33–6. [PubMed: 19326417]
130.
Kadota RP, Mahoney DH, Doyle J, et al. Dose intensive melphalan and cyclophosphamide with autologous hematopoietic stem cells for recurrent medulloblastoma or germinoma. Pediatr Blood Cancer. 2008 Nov;51(5):675–8. [PMC free article: PMC2900925] [PubMed: 18623206]
131.
Ridola V, Grill J, Doz F, et al. High-dose chemotherapy with autologous stem cell rescue followed by posterior fossa irradiation for local medulloblastoma recurrence or progression after conventional chemotherapy. Cancer. 2007 Jul 1;110(1):156–63. [PubMed: 17541945]
132.
Shih CS, Hale GA, Gronewold L, et al. High-dose chemotherapy with autologous stem cell rescue for children with recurrent malignant brain tumors. Cancer. 2008 Mar 15;112(6):1345–53. [PubMed: 18224664]
133.
Sung KW, Yoo KH, Cho EJ, et al. High-dose chemotherapy and autologous stem cell rescue in children with newly diagnosed high-risk or relapsed medulloblastoma or supratentorial primitive neuroectodermal tumor. Pediatr Blood Cancer. 2007 Apr;48(4):408–15. [PubMed: 17066462]
134.
Smiers FJ, Krishnamurti L, Lucarelli G. Hematopoietic stem cell transplantation for hemoglobinopathies: current practice and emerging trends. Pediatr Clin North Am. 2010 Feb;57(1):181–205. [PubMed: 20307718]
135.
Modell B, Khan M, Darlison M, et al. Improved survival of thalassaemia major in the UK and relation to T2* cardiovascular magnetic resonance. J Cardiovasc Magn Reson. 2008;10:42. [PMC free article: PMC2563008] [PubMed: 18817553]
136.
Platt OS, Brambilla DJ, Rosse WF, et al. Mortality in sickle cell disease. Life expectancy and risk factors for early death. N Engl J Med. 1994 Jun 9;330(23):1639–44. [PubMed: 7993409]
137.
Nietert PJ, Abboud MR, Silverstein MD, et al. Bone marrow transplantation versus periodic prophylactic blood transfusion in sickle cell patients at high risk of ischemic stroke: a decision analysis. Blood. 2000 May 15;95(10):3057–64. [PubMed: 10807769]
138.
Walters MC, Storb R, Patience M, et al. Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Multicenter investigation of bone marrow transplantation for sickle cell disease. Blood. 2000 Mar 15;95(6):1918–24. [PubMed: 10706855]
139.
Bhatia M, Walters MC. Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future. Bone Marrow Transplant. 2008 Jan;41(2):109–17. [PubMed: 18059330]
140.
Inati A. Recent advances in improving the management of sickle cell disease. Blood Rev. 2009 Dec;23 Suppl 1:S9–13. [PubMed: 20116638]
141.
Strouse JJ, Lanzkron S, Beach MC, et al. Hydroxyurea for sickle cell disease: a systematic review for efficacy and toxicity in children. Pediatrics. 2008 Dec;122(6):1332–42. [PubMed: 19047254]
142.
Walters MC, Patience M, Leisenring W, et al. Bone marrow transplantation for sickle cell disease. N Engl J Med. 1996 Aug 8;335(6):369–76. [PubMed: 8663884]
143.
Brichard B, Vermylen C, Ninane J, et al. Persistence of fetal hemoglobin production after successful transplantation of cord blood stem cells in a patient with sickle cell anemia. J Pediatr. 1996 Feb;128(2):241–3. [PubMed: 8636820]
144.
Locatelli F, Rocha V, Reed W, et al. Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood. 2003 Mar 15;101(6):2137–43. [PubMed: 12424197]
145.
Centers for Disease Control and Prevention. RuSH Questions and Answers. 2010. [updated September 30, 2010]; www​.cdc.gov/ncbddd/sicklecell/RuSH_FAQs​.html.
146.
Jadad AR, Moore RA, Carroll D, et al. Assessing the quality of reports of randomized clinical trials: is blinding necessary? Control Clin Trials. 1996 Feb;17(1):1–12. [PubMed: 8721797]
147.
Vermylen C, Cornu G, Ferster A, et al. Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant. 1998 Jul;22(1):1–6. [PubMed: 9678788]
148.
Bernaudin F, Socie G, Kuentz M, et al. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood. 2007 Oct 1;110(7):2749–56. [PubMed: 17606762]
149.
Lucarelli G, Andreani M, Angelucci E. The cure of thalassemia by bone marrow transplantation. Blood Rev. 2002 Jun;16(2):81–5. [PubMed: 12127951]
150.
La Nasa G, Argiolu F, Giardini C, et al. Unrelated bone marrow transplantation for beta-thalassemia patients: The experience of the Italian Bone Marrow Transplant Group. Ann N Y Acad Sci. 2005;1054:186–95. [PubMed: 16339665]
151.
Hongeng S, Pakakasama S, Chuansumrit A, et al. Outcomes of transplantation with related- and unrelated-donor stem cells in children with severe thalassemia. Biol Blood Marrow Transplant. 2006 Jun;12(6):683–7. [PubMed: 16737942]
152.
Gaziev D, Galimberti M, Lucarelli G, et al. Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA-nonidentical sibling or parent transplants. Bone Marrow Transplant. 2000 Apr;25(8):815–21. [PMC free article: PMC7102364] [PubMed: 10808201]
153.
Li CK, Chik KW, Wong GW, et al. Growth and endocrine function following bone marrow transplantation for thalassemia major. Pediatr Hematol Oncol. 2004 Jul-Aug;21(5):411–9. [PubMed: 15205084]
154.
Ferster A, Vermylen C, Cornu G, et al. Hydroxyurea for treatment of severe sickle cell anemia: a pediatric clinical trial. Blood. 1996 Sep 15;88(6):1960–4. [PubMed: 8822914]
155.
Olivieri NF, Vichinsky EP. Hydroxyurea in children with sickle cell disease: impact on splenic function and compliance with therapy. J Pediatr Hematol Oncol. 1998 Jan-Feb;20(1):26–31. [PubMed: 9482409]
156.
Santos A, Pinheiro V, Anjos C, et al. Scintigraphic follow-up of the effects of therapy with hydroxyurea on splenic function in patients with sickle cell disease. Eur J Nucl Med Mol Imaging. 2002 Apr;29(4):536–41. [PubMed: 11914893]
157.
Svarch E, Machin S, Nieves RM, et al. Hydroxyurea treatment in children with sickle cell anemia in Central America and the Caribbean countries. Pediatr Blood Cancer. 2006 Jul;47(1):111–2. [PubMed: 16550531]
158.
Hankins JS, Ware RE, Rogers ZR, et al. Long-term hydroxyurea therapy for infants with sickle cell anemia: the HUSOFT extension study. Blood. 2005 Oct 1;106(7):2269–75. [PMC free article: PMC1895275] [PubMed: 16172253]
159.
Adams RJ, McKie VC, Hsu L, et al. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography. N Engl J Med. 1998 Jul 2;339(1):5–11. [PubMed: 9647873]
160.
Borgna-Pignatti C, Galanello R. Thalassemias and related disorders: quantitative disorders of hemoglobin synthesis. In: Wintrobe MM, Greer JP, editors. Wintrobe's clinical hematology. 12th ed. Philadelphia: Wolters Kluwer Health/Lippincott Williams & Wilkins; 2009. pp. 1083–131.
161.
Weatherall DJ. The thalassemias. In: Stamatoyannapoulos G, Niehuis AW, Majerus PW, Varmus H, editors. The Molecular Basis of Blood Disease. 2nd ed. Philadelphia: WB Saunders Company; 1994. p. 157.
162.
Borgna-Pignatti C, Cappellini MD, De Stefano P, et al. Survival and complications in thalassemia. Ann N Y Acad Sci. 2005;1054:40–7. [PubMed: 16339650]
163.
Borgna-Pignatti C, Cappellini MD, De Stefano P, et al. Cardiac morbidity and mortality in deferoxamine- or deferiprone-treated patients with thalassemia major. Blood. 2006 May 1;107(9):3733–7. [PubMed: 16373663]
164.
Ehlers KH, Giardina PJ, Lesser ML, et al. Prolonged survival in patients with beta-thalassemia major treated with deferoxamine. J Pediatr. 1991 Apr;118(4 Pt 1):540–5. [PubMed: 2007928]
165.
Yavarian M, Karimi M, Bakker E, et al. Response to hydroxyurea treatment in Iranian transfusion-dependent beta-thalassemia patients. Haematologica. 2004 Oct;89(10):1172–8. [PubMed: 15477200]
166.
Delea TE, Edelsberg J, Sofrygin O, et al. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007 Oct;47(10):1919–29. [PubMed: 17880620]
167.
Modell B, Khan M, Darlison M. Survival in beta-thalassaemia major in the UK: data from the UK Thalassaemia Register. Lancet. 2000 Jun 10;355(9220):2051–2. [PubMed: 10885361]
168.
Myers KC, Davies SM. Hematopoietic stem cell transplantation for bone marrow failure syndromes in children. Biol Blood Marrow Transplant. 2009 Mar;15(3):279–92. [PubMed: 19203719]
169.
Alter BP, Greene MH, Velazquez I, et al. Cancer in Fanconi anemia. Blood. 2003 Mar 1;101(5):2072. [PubMed: 12584146]
170.
Gluckman E, Wagner JE. HSCT for hereditary bone marrow failure syndromes, Chapter 42. In: Apperley J, Carreras E, Gluckman E, Gratwohl A, Masszi T, editors. The 2008 revised edition of the EBMT-ESH Handbook on Haemopoietic Stem Cell Transplantation. 2008.
171.
Dokal I, Vulliamy T. Inherited aplastic anaemias/bone marrow failure syndromes. Blood Rev. 2008 May;22(3):141–53. [PubMed: 18164793]
172.
Bagby GC, Lipton JM, Sloand EM, et al. Marrow failure. Hematology Am Soc Hematol Educ Program. 2004:318–36. [PubMed: 15561690]
173.
Ferry C, Ouachee M, Leblanc T, et al. Hematopoietic stem cell transplantation in severe congenital neutropenia: experience of the French SCN register. Bone Marrow Transplant. 2005 Jan;35(1):45–50. [PubMed: 15489867]
174.
Dufour C, Svahn J. Fanconi anaemia: new strategies. Bone Marrow Transplant. 2008 Jun;41 Suppl 2:S90–5. [PubMed: 18545254]
175.
Burroughs L, Woolfrey A, Shimamura A. Shwachman-Diamond syndrome: a review of the clinical presentation, molecular pathogenesis, diagnosis, and treatment. Hematol Oncol Clin North Am. 2009 Apr;23(2):233–48. [PMC free article: PMC2754297] [PubMed: 19327581]
176.
de la Fuente J, Dokal I. Dyskeratosis congenita: advances in the understanding of the telomerase defect and the role of stem cell transplantation. Pediatr Transplant. 2007 Sep;11(6):584–94. [PubMed: 17663679]
177.
MacMillan ML, Davies SM, Wagner JE, et al. Engraftment of unrelated donor stem cells in children with familial amegakaryocytic thrombocytopenia. Bone Marrow Transplant. 1998 Apr;21(7):735–7. [PubMed: 9578317]
178.
Kudo K, Kato K, Matsuyama T, et al. Successful engraftment of unrelated donor stem cells in two children with congenital amegakaryocytic thrombocytopenia. J Pediatr Hematol Oncol. 2002 Jan;24(1):79–80. [PubMed: 11902750]
179.
Steele M, Hitzler J, Doyle JJ, et al. Reduced intensity hematopoietic stem-cell transplantation across human leukocyte antigen barriers in a patient with congenital amegakaryocytic thrombocytopenia and monosomy 7. Pediatr Blood Cancer. 2005 Aug;45(2):212–6. [PubMed: 15782403]
180.
Al-Ahmari A, Ayas M, Al-Jefri A, et al. Allogeneic stem cell transplantation for patients with congenital amegakaryocytic thrombocytopenia (CAT). Bone Marrow Transplant. 2004 Apr;33(8):829–31. [PubMed: 14968137]
181.
Yesilipek, Hazar V, Kupesiz A, et al. Peripheral stem cell transplantation in a child with amegakaryocytic thrombocytopenia. Bone Marrow Transplant. 2000 Sep;26(5):571–2. [PubMed: 11019849]
182.
Lackner A, Basu O, Bierings M, et al. Haematopoietic stem cell transplantation for amegakaryocytic thrombocytopenia. Br J Haematol. 2000 Jun;109(4):773–5. [PubMed: 10929028]
183.
Lipton JM, Ellis SR. Diamond-Blackfan anemia: diagnosis, treatment, and molecular pathogenesis. Hematol Oncol Clin North Am. 2009 Apr;23(2):261–82. [PMC free article: PMC2886591] [PubMed: 19327583]
184.
Elhasid R, Rowe JM. Hematopoetic stem cell transplantation in neutrophil disorders: severe congenital neutropenia, leukocyte adhesion deficiency and chronic granulomatous disease. Clin Rev Allergy Immunol. 2010 Feb;38(1):61–7. [PubMed: 19452286]
185.
Pongtanakul B, Das PK, Charpentier K, et al. Outcome of children with aplastic anemia treated with immunosuppressive therapy. Pediatr Blood Cancer. 2008 Jan;50(1):52–7. [PubMed: 17941069]
186.
Scheinberg P, Wu CO, Nunez O, et al. Long-term outcome of pediatric patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine. J Pediatr. 2008 Dec;153(6):814–9. [PMC free article: PMC3971527] [PubMed: 18672253]
187.
Kojima S, Ohara A, Tsuchida M, et al. Risk factors for evolution of acquired aplastic anemia into myelodysplastic syndrome and acute myeloid leukemia after immunosuppressive therapy in children. Blood. 2002 Aug 1;100(3):786–90. [PubMed: 12130487]
188.
Frickhofen N, Heimpel H, Kaltwasser JP, et al. Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood. 2003 Feb 15;101(4):1236–42. [PubMed: 12393680]
189.
Kosaka Y, Yagasaki H, Sano K, et al. Prospective multicenter trial comparing repeated immunosuppressive therapy with stem-cell transplantation from an alternative donor as second-line treatment for children with severe and very severe aplastic anemia. Blood. 2008 Feb 1;111(3):1054–9. [PubMed: 17989314]
190.
Locasciulli A, Oneto R, Bacigalupo A, et al. Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT). Haematologica. 2007 Jan;92(1):11–8. [PubMed: 17229630]
191.
Bacigalupo A, Brand R, Oneto R, et al. Treatment of acquired severe aplastic anemia: bone marrow transplantation compared with immunosuppressive therapy--The European Group for Blood and Marrow Transplantation experience. Semin Hematol. 2000 Jan;37(1):69–80. [PubMed: 10676912]
192.
Locatelli F, Bruno B, Zecca M, et al. Cyclosporin A and short-term methotrexate versus cyclosporin A as graft versus host disease prophylaxis in patients with severe aplastic anemia given allogeneic bone marrow transplantation from an HLA-identical sibling: results of a GITMO/EBMT randomized trial. Blood. 2000 Sep 1;96(5):1690–7. [PubMed: 10961865]
193.
Farzin A, Davies SM, Smith FO, et al. Matched sibling donor haematopoietic stem cell transplantation in Fanconi anaemia: an update of the Cincinnati Children's experience. Br J Haematol. 2007 Feb;136(4):633–40. [PubMed: 17367413]
194.
Schrezenmeier H, Passweg JR, Marsh JC, et al. Worse outcome and more chronic GVHD with peripheral blood progenitor cells than bone marrow in HLA-matched sibling donor transplants for young patients with severe acquired aplastic anemia. Blood. 2007 Aug 15;110(4):1397–400. [PMC free article: PMC1939910] [PubMed: 17475907]
195.
Deeg HJ, Socie G, Schoch G, et al. Malignancies after marrow transplantation for aplastic anemia and fanconi anemia: a joint Seattle and Paris analysis of results in 700 patients. Blood. 1996 Jan 1;87(1):386–92. [PubMed: 8547667]
196.
Deeg HJ, Leisenring W, Storb R, et al. Long-term outcome after marrow transplantation for severe aplastic anemia. Blood. 1998 May 15;91(10):3637–45. [PubMed: 9572999]
197.
Bacigalupo A, Locatelli F, Lanino E, et al. Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party. Bone Marrow Transplant. 2005 Dec;36(11):947–50. [PubMed: 16205733]
198.
Deeg HJ, O'Donnell M, Tolar J, et al. Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy. Blood. 2006 Sep 1;108(5):1485–91. [PMC free article: PMC1895515] [PubMed: 16684959]
199.
Viollier R, Socie G, Tichelli A, et al. Recent improvement in outcome of unrelated donor transplantation for aplastic anemia. Bone Marrow Transplant. 2008 Jan;41(1):45–50. [PubMed: 17982502]
200.
Guardiola P, Pasquini R, Dokal I, et al. Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation. Blood. 2000 Jan 15;95(2):422–9. [PubMed: 10627445]
201.
de Medeiros CR, Bitencourt MA, Zanis-Neto J, et al. Allogeneic hematopoietic stem cell transplantation from an alternative stem cell source in Fanconi anemia patients: analysis of 47 patients from a single institution. Braz J Med Biol Res. 2006 Oct;39(10):1297–304. [PubMed: 17053839]
202.
Zanis-Neto J, Flowers ME, Medeiros CR, et al. Low-dose cyclophosphamide conditioning for haematopoietic cell transplantation from HLA-matched related donors in patients with Fanconi anaemia. Br J Haematol. 2005 Jul;130(1):99–106. [PubMed: 15982351]
203.
Wagner JE, Eapen M, MacMillan ML, et al. Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. Blood. 2007 Mar 1;109(5):2256–62. [PMC free article: PMC1801062] [PubMed: 17038525]
204.
Locatelli F, Zecca M, Pession A, et al. The outcome of children with Fanconi anemia given hematopoietic stem cell transplantation and the influence of fludarabine in the conditioning regimen: a report from the Italian pediatric group. Haematologica. 2007 Oct;92(10):1381–8. [PubMed: 18024375]
205.
Yabe H, Inoue H, Matsumoto M, et al. Allogeneic haematopoietic cell transplantation from alternative donors with a conditioning regimen of low-dose irradiation, fludarabine and cyclophosphamide in Fanconi anaemia. Br J Haematol. 2006 Jul;134(2):208–12. [PubMed: 16846479]
206.
Chaudhury S, Auerbach AD, Kernan NA, et al. Fludarabine-based cytoreductive regimen and T-cell-depleted grafts from alternative donors for the treatment of high-risk patients with Fanconi anaemia. Br J Haematol. 2008 Mar;140(6):644–55. [PubMed: 18302713]
207.
Gluckman E, Rocha V, Ionescu I, et al. Results of unrelated cord blood transplant in fanconi anemia patients: risk factor analysis for engraftment and survival. Biol Blood Marrow Transplant. 2007 Sep;13(9):1073–82. [PubMed: 17697970]
208.
Vibhakar R, Radhi M, Rumelhart S, et al. Successful unrelated umbilical cord blood transplantation in children with Shwachman-Diamond syndrome. Bone Marrow Transplant. 2005 Nov;36(10):855–61. [PubMed: 16113664]
209.
Donadieu J, Michel G, Merlin E, et al. Hematopoietic stem cell transplantation for Shwachman-Diamond syndrome: experience of the French neutropenia registry. Bone Marrow Transplant. 2005 Nov;36(9):787–92. [PMC free article: PMC7091863] [PubMed: 16151425]
210.
Bhatla D, Davies SM, Shenoy S, et al. Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman-Diamond syndrome. Bone Marrow Transplant. 2008 Aug;42(3):159–65. [PubMed: 18500373]
211.
Cesaro S, Oneto R, Messina C, et al. Haematopoietic stem cell transplantation for Shwachman-Diamond disease: a study from the European Group for blood and marrow transplantation. Br J Haematol. 2005 Oct;131(2):231–6. [PubMed: 16197455]
212.
Beran M, Spitzer G, Verma DS. Testosterone and synthetic and androgens improve the in vitro survival of human marrow progenitor cells in serum-free suspension cultures. J Lab Clin Med. 1982 Feb;99(2):247–53. [PubMed: 7061919]
213.
Ayas M, Al-Jefri A, Al-Mahr M, et al. Stem cell transplantation for patients with Fanconi anemia with low-dose cyclophosphamide and antithymocyte globulins without the use of radiation therapy. Bone Marrow Transplant. 2005 Mar;35(5):463–6. [PubMed: 15654354]
214.
Dror Y, Freedman MH, Leaker M, et al. Low-intensity hematopoietic stem-cell transplantation across human leucocyte antigen barriers in dyskeratosis congenita. Bone Marrow Transplant. 2003 May;31(10):847–50. [PubMed: 12748659]
215.
Brazzola P, Duval M, Fournet JC, et al. Fatal diffuse capillaritis after hematopoietic stem-cell transplantation for dyskeratosis congenita despite low-intensity conditioning regimen. Bone Marrow Transplant. 2005 Dec;36(12):1103–5. author reply 5. [PubMed: 16205731]
216.
Gungor T, Corbacioglu S, Storb R, et al. Nonmyeloablative allogeneic hematopoietic stem cell transplantation for treatment of Dyskeratosis congenita. Bone Marrow Transplant. 2003 Mar;31(5):407–10. [PubMed: 12634734]
217.
Cossu F, Vulliamy TJ, Marrone A, et al. A novel DKC1 mutation, severe combined immunodeficiency (T+B-NK- SCID) and bone marrow transplantation in an infant with Hoyeraal-Hreidarsson syndrome. Br J Haematol. 2002 Dec;119(3):765–8. [PubMed: 12437656]
218.
Nobili B, Rossi G, De Stefano P, et al. Successful umbilical cord blood transplantation in a child with dyskeratosis congenita after a fludarabine-based reduced-intensity conditioning regimen. Br J Haematol. 2002 Nov;119(2):573–4. [PubMed: 12406104]
219.
Vlachos A, Ball S, Dahl N, et al. Diagnosing and treating Diamond Blackfan anaemia: results of an international clinical consensus conference. Br J Haematol. 2008 Sep;142(6):859–76. [PMC free article: PMC2654478] [PubMed: 18671700]
220.
Lipton JM, Atsidaftos E, Zyskind I, et al. Improving clinical care and elucidating the pathophysiology of Diamond Blackfan anemia: an update from the Diamond Blackfan Anemia Registry. Pediatr Blood Cancer. 2006 May 1;46(5):558–64. [PubMed: 16317735]
221.
Roy V, Perez WS, Eapen M, et al. Bone marrow transplantation for diamond-blackfan anemia. Biol Blood Marrow Transplant. 2005 Aug;11(8):600–8. [PubMed: 16041310]
222.
Zeidler C, Boxer L, Dale DC, et al. Management of Kostmann syndrome in the G-CSF era. Br J Haematol. 2000 Jun;109(3):490–5. [PubMed: 10886193]
223.
Rosenberg PS, Alter BP, Bolyard AA, et al. The incidence of leukemia and mortality from sepsis in patients with severe congenital neutropenia receiving long-term G-CSF therapy. Blood. 2006 Jun 15;107(12):4628–35. [PMC free article: PMC1895804] [PubMed: 16497969]
224.
Yakisan E, Schirg E, Zeidler C, et al. High incidence of significant bone loss in patients with severe congenital neutropenia (Kostmann's syndrome). J Pediatr. 1997 Oct;131(4):592–7. [PubMed: 9386665]
225.
Zeidler C, Welte K, Barak Y, et al. Stem cell transplantation in patients with severe congenital neutropenia without evidence of leukemic transformation. Blood. 2000 Feb 15;95(4):1195–8. [PubMed: 10666190]
226.
Fanconi G. Familiare infantile perniziosaartige Anämie (perniziöses Blutbild und Konstitution). Z Kinderheilkunde. 1927;117:257–80.
227.
Fanconi G. Familial constitutional panmyelocytopathy, Fanconi's anemia (F.A.). I. Clinical aspects. Semin Hematol. 1967 Jul;4(3):233–40. [PubMed: 6074578]
228.
Rogatko A, Auerbach AD. Segregation analysis with uncertain ascertainment: application to Fanconi anemia. Am J Hum Genet. 1988 Jun;42(6):889–97. [PMC free article: PMC1715212] [PubMed: 3369448]
229.
Schroeder TM, Tilgen D, Kruger J, et al. Formal genetics of Fanconi's anemia. Hum Genet. 1976 Jun 29;32(3):257–88. [PubMed: 939547]
230.
Kutler DI, Singh B, Satagopan J, et al. A 20-year perspective on the International Fanconi Anemia Registry (IFAR). Blood. 2003 Feb 15;101(4):1249–56. [PubMed: 12393516]
231.
Fanconi Anemia: Guidelines for diagnosis and management. Eugene, OR: Fanconi Anemia Research Fund, Inc.; 2008. [cited 2010 November]. www​.fanconi.org/images​/uploads/other/Guidelines​_for_Diagnosis_and_Management.pdf.
232.
Boocock GR, Morrison JA, Popovic M, et al. Mutations in SBDS are associated with Shwachman-Diamond syndrome. Nat Genet. 2003 Jan;33(1):97–101. [PubMed: 12496757]
233.
Alter BP, Giri N, Savage SA, et al. Malignancies and survival patterns in the National Cancer Institute inherited bone marrow failure syndromes cohort study. Br J Haematol. 2010 Jul;150(2):179–88. [PMC free article: PMC3125983] [PubMed: 20507306]
234.
Ginzberg H, Shin J, Ellis L, et al. Shwachman syndrome: phenotypic manifestations of sibling sets and isolated cases in a large patient cohort are similar. J Pediatr. 1999 Jul;135(1):81–8. [PubMed: 10393609]
235.
Mack DR, Forstner GG, Wilschanski M, et al. Shwachman syndrome: exocrine pancreatic dysfunction and variable phenotypic expression. Gastroenterology. 1996 Dec;111(6):1593–602. [PubMed: 8942739]
236.
Smith OP, Hann IM, Chessells JM, et al. Haematological abnormalities in Shwachman-Diamond syndrome. Br J Haematol. 1996 Aug;94(2):279–84. [PubMed: 8759887]
237.
Mitchell JR, Wood E, Collins K. A telomerase component is defective in the human disease dyskeratosis congenita. Nature. 1999 Dec 2;402(6761):551–5. [PubMed: 10591218]
238.
Vulliamy T, Dokal I. Dyskeratosis congenita. Semin Hematol. 2006 Jul;43(3):157–66. [PubMed: 16822458]
239.
Drachtman RA, Alter BP. Dyskeratosis congenita. Dermatol Clin. 1995 Jan;13(1):33–9. [PubMed: 7712648]
240.
Alter BP, Giri N, Savage SA, et al. Cancer in dyskeratosis congenita. Blood. 2009 Jun 25;113(26):6549–57. [PMC free article: PMC2710915] [PubMed: 19282459]
241.
Dokal I. Fanconi's anaemia and related bone marrow failure syndromes. Br Med Bull. 2006:77–78. 37–53. [PubMed: 16968690]
242.
Walne AJ, Dokal I. Advances in the understanding of dyskeratosis congenita. Br J Haematol. 2009 Apr;145(2):164–72. [PMC free article: PMC2882229] [PubMed: 19208095]
243.
Savage SA, Alter BP. Dyskeratosis congenita. Hematol Oncol Clin North Am. 2009 Apr;23(2):215–31. [PMC free article: PMC2702847] [PubMed: 19327580]
244.
Ballmaier M, Germeshausen M, Schulze H, et al. c-mpl mutations are the cause of congenital amegakaryocytic thrombocytopenia. Blood. 2001 Jan 1;97(1):139–46. [PubMed: 11133753]
245.
Ballmaier M, Germeshausen M. Advances in the understanding of congenital amegakaryocytic thrombocytopenia. Br J Haematol. 2009 Jun;146(1):3–16. [PubMed: 19388932]
246.
King S, Germeshausen M, Strauss G, et al. Congenital amegakaryocytic thrombocytopenia: a retrospective clinical analysis of 20 patients. Br J Haematol. 2005 Dec;131(5):636–44. [PubMed: 16351641]
247.
Kostman R. Infantile genetic agranulocytosis. A review with presentation of ten new cases. Acta Paediatr Scand. 1975 Mar;64(2):362–8. [PubMed: 1130195]
248.
Welte K, Zeidler C, Dale DC. Severe congenital neutropenia. Semin Hematol. 2006 Jul;43(3):189–95. [PubMed: 16822461]
249.
Dong F, Brynes RK, Tidow N, et al. Mutations in the gene for the granulocyte colony-stimulating-factor receptor in patients with acute myeloid leukemia preceded by severe congenital neutropenia. N Engl J Med. 1995 Aug 24;333(8):487–93. [PubMed: 7542747]
250.
Gennery AR, Cant AJ. Advances in hematopoietic stem cell transplantation for primary immunodeficiency. Immunol Allergy Clin North Am. 2008 May;28(2):439–56. x–xi. [PubMed: 18424341]
251.
Filipovich A. Hematopoietic cell transplantation for correction of primary immunodeficiencies. Bone Marrow Transplant. 2008 Aug;42 Suppl 1:S49–S52. [PubMed: 18724301]
252.
Orange JS, Hossny EM, Weiler CR, et al. Use of intravenous immunoglobulin in human disease: a review of evidence by members of the Primary Immunodeficiency Committee of the American Academy of Allergy, Asthma and Immunology. J Allergy Clin Immunol. 2006 Apr;117(4 Suppl):S525–53. [PubMed: 16580469]
253.
Antoine C, Muller S, Cant A, et al. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet. 2003 Feb 15;361(9357):553–60. [PubMed: 12598139]
254.
Filipovich AH, Stone JV, Tomany SC, et al. Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program. Blood. 2001 Mar 15;97(6):1598–603. [PubMed: 11238097]
255.
Grunebaum E, Mazzolari E, Porta F, et al. Bone marrow transplantation for severe combined immune deficiency. JAMA. 2006 Feb 1;295(5):508–18. [PubMed: 16449616]
256.
Buckley RH, Schiff SE, Schiff RI, et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med. 1999 Feb 18;340(7):508–16. [PubMed: 10021471]
257.
Porta F, Forino C, De Martiis D, et al. Stem cell transplantation for primary immunodeficiencies. Bone Marrow Transplant. 2008 Jun;41 Suppl 2:S83–6. [PubMed: 18545252]
258.
Bhattacharya A, Slatter MA, Chapman CE, et al. Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency. Bone Marrow Transplant. 2005 Aug;36(4):295–9. [PubMed: 15968287]
259.
Myers LA, Patel DD, Puck JM, et al. Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood. 2002 Feb 1;99(3):872–8. [PubMed: 11806989]
260.
Kohn DB. Update on gene therapy for immunodeficiencies. Clin Immunol. 2010 May;135(2):247–54. [PMC free article: PMC2856741] [PubMed: 20071242]
261.
McGovern MM, Desnick RJ. Lipidoses (Chapter 86.4). In: Kliegman R, Nelson WE, editors. Nelson textbook of pediatrics. 18th ed. Philadelphia: Saunders; 2007. p. lii. 3147 p.
262.
National Institute of Neurological Disorders and Stroke. Mucolipidoses Fact Sheet. Bethesda, MD: Office of Communications and Public Liaison; 2007. (Publication No. 03-5115)
263.
Spranger J. Metabolic diseases (Chapter 88). In: Kliegman R, Nelson WE, editors. Nelson Textbook of Pediatrics. 18th ed. Philadelphia: Saunders; 2007. p. lii. 3147 p.
264.
Wraith JE. Mucopolysaccharidoses and oligosaccharidoses (Chapter 39). In: Fernandes J, editor. Inborn metabolic diseases : diagnosis and treatment. 4th, rev. ed. Heidelberg: Springer; 2006. p. xxii. 561 p.
265.
Brady RO, Schiffmann R. Enzyme-replacement therapy for metabolic storage disorders. Lancet Neurol. 2004 Dec;3(12):752–6. [PubMed: 15556808]
266.
Wraith JE. The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. Expert Opin Pharmacother. 2005 Mar;6(3):489–506. [PubMed: 15794739]
267.
Tolar J, Orchard PJ. alpha-L-iduronidase therapy for mucopolysaccharidosis type I. Biologics. 2008 Dec;2(4):743–51. [PMC free article: PMC2727896] [PubMed: 19707455]
268.
Prasad VK, Kurtzberg J. Transplant outcomes in mucopolysaccharidoses. Semin Hematol. 2010 Jan;47(1):59–69. [PubMed: 20109613]
269.
Boelens JJ. Trends in haematopoietic cell transplantation for inborn errors of metabolism. J Inherit Metab Dis. 2006 Apr-Jun;29(2-3):413–20. [PubMed: 16763911]
270.
Peters C. Metabolic diseases (Chapter 15). In: Mehta P, editor. Pediatric stem cell transplantation. Boston: Jones and Bartlett; 2004. p. xxiii. 486 p.
271.
Aldenhoven M, Boelens JJ, de Koning TJ. The clinical outcome of Hurler syndrome after stem cell transplantation. Biol Blood Marrow Transplant. 2008 May;14(5):485–98. [PubMed: 18410891]
272.
Harmatz P, Giugliani R, Schwartz IV, et al. Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Mol Genet Metab. 2008 Aug;94(4):469–75. [PubMed: 18502162]
273.
Kakkis ED, Muenzer J, Tiller GE, et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med. 2001 Jan 18;344(3):182–8. [PubMed: 11172140]
274.
Wraith JE, Clarke LA, Beck M, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr. 2004 May;144(5):581–8. [PubMed: 15126990]
275.
Sifuentes M, Doroshow R, Hoft R, et al. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. Mol Genet Metab. 2007 Feb;90(2):171–80. [PubMed: 17011223]
276.
Boelens JJ, Wynn RF, O'Meara A, et al. Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant. 2007 Aug;40(3):225–33. [PMC free article: PMC7094454] [PubMed: 17529997]
277.
Krivit W, Lockman LA, Watkins PA, et al. The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome. J Inherit Metab Dis. 1995;18(4):398–412. [PubMed: 7494399]
278.
Field RE, Buchanan JA, Copplemans MG, et al. Bone-marrow transplantation in Hurler's syndrome. Effect on skeletal development. J Bone Joint Surg Br. 1994 Nov;76(6):975–81. [PubMed: 7983131]
279.
Weisstein JS, Delgado E, Steinbach LS, et al. Musculoskeletal manifestations of Hurler syndrome: long-term follow-up after bone marrow transplantation. J Pediatr Orthop. 2004 Jan-Feb;24(1):97–101. [PubMed: 14676543]
280.
Souillet G, Guffon N, Maire I, et al. Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources. Bone Marrow Transplant. 2003 Jun;31(12):1105–17. [PubMed: 12796790]
281.
Braunlin EA, Stauffer NR, Peters CH, et al. Usefulness of bone marrow transplantation in the Hurler syndrome. Am J Cardiol. 2003 Oct 1;92(7):882–6. [PubMed: 14516901]
282.
Peters C, Shapiro EG, Anderson J, et al. Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood. 1998 Apr 1;91(7):2601–8. [PubMed: 9516162]
283.
Harmatz P, Giugliani R, Schwartz I, et al. Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr. 2006 Apr;148(4):533–9. [PubMed: 16647419]
284.
Scarpa M, Barone R, Fiumara A, et al. Mucopolysaccharidosis VI: the Italian experience. Eur J Pediatr. 2009 Oct;168(10):1203–6. [PubMed: 19130082]
285.
Krivit W, Pierpont ME, Ayaz K, et al. Bone-marrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). Biochemical and clinical status 24 months after transplantation. N Engl J Med. 1984 Dec 20;311(25):1606–11. [PubMed: 6150438]
286.
Herskhovitz E, Young E, Rainer J, et al. Bone marrow transplantation for Maroteaux-Lamy syndrome (MPS VI): long-term follow-up. J Inherit Metab Dis. 1999 Feb;22(1):50–62. [PubMed: 10070618]
287.
Yamada Y, Kato K, Sukegawa K, et al. Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation. Bone Marrow Transplant. 1998 Mar;21(6):629–34. [PubMed: 9543069]
288.
Vellodi A, Young EP, Cooper A, et al. Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres. Arch Dis Child. 1997 Feb;76(2):92–9. [PMC free article: PMC1717089] [PubMed: 9068295]
289.
Giugliani R, Harmatz P, Wraith JE. Management guidelines for mucopolysaccharidosis VI. Pediatrics. 2007 Aug;120(2):405–18. [PubMed: 17671068]
290.
Muenzer J, Wraith JE, Clarke LA. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics. 2009 Jan;123(1):19–29. [PubMed: 19117856]
291.
Peters C, Steward CG. Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant. 2003 Feb;31(4):229–39. [PubMed: 12621457]
292.
Johnston MV. Sphingolipidoses (Chapter 599.1). In: Kliegman R, Nelson WE, editors. Nelson textbook of pediatrics. 18th ed. Philadelphia: Saunders; 2007. p. lii. 3147 p.
293.
Chen M, Wang J. Gaucher disease: review of the literature. Arch Pathol Lab Med. 2008 May;132(5):851–3. [PubMed: 18466035]
294.
Beutler E, Grabowski GA. Gaucher disease. In: Scriver CR, editor. The metabolic and molecular bases of inherited disease. 8th ed. New York: McGraw-Hill; 2001. pp. 3635–68.
295.
Biegstraaten M, van Schaik IN, Aerts JM, et al. ‘Non-neuronopathic’ Gaucher disease reconsidered. Prevalence of neurological manifestations in a Dutch cohort of type I Gaucher disease patients and a systematic review of the literature. J Inherit Metab Dis. 2008 Jun;31(3):337–49. [PubMed: 18404411]
296.
Jmoudiak M, Futerman AH. Gaucher disease: pathological mechanisms and modern management. Br J Haematol. 2005 Apr;129(2):178–88. [PubMed: 15813845]
297.
Charrow J, Andersson HC, Kaplan P, et al. Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations. J Pediatr. 2004 Jan;144(1):112–20. [PubMed: 14722528]
298.
Pastores GM, Weinreb NJ, Aerts H, et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol. 2004 Oct;41(4 Suppl 5):4–14. [PubMed: 15468045]
299.
Weinreb NJ, Charrow J, Andersson HC, et al. Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry. Am J Med. 2002 Aug 1;113(2):112–9. [PubMed: 12133749]
300.
Starzyk K, Richards S, Yee J, et al. The long-term international safety experience of imiglucerase therapy for Gaucher disease. Mol Genet Metab. 2007 Feb;90(2):157–63. [PubMed: 17079176]
301.
Andersson HC, Charrow J, Kaplan P, et al. Individualization of long-term enzyme replacement therapy for Gaucher disease. Genet Med. 2005 Feb;7(2):105–10. [PubMed: 15714077]
302.
Chan KW, Wong LT, Applegarth D, et al. Bone marrow transplantation in Gaucher's disease: effect of mixed chimeric state. Bone Marrow Transplant. 1994 Aug;14(2):327–30. [PubMed: 7994251]
303.
Ringden O, Groth CG, Erikson A, et al. Ten years' experience of bone marrow transplantation for Gaucher disease. Transplantation. 1995 Mar 27;59(6):864–70. [PubMed: 7701581]
304.
Yen CC, Chiou TJ, Lin CY, et al. Allogeneic bone marrow transplantation for Gaucher disease--a case report. Zhonghua Yi Xue Za Zhi (Taipei). 1997 Jun;59(6):372–6. [PubMed: 9294918]
305.
Hobbs JR, Jones KH, Shaw PJ, et al. Beneficial effect of pre-transplant splenectomy on displacement bone marrow transplantation for Gaucher's syndrome. Lancet. 1987 May 16;1(8542):1111–5. [PubMed: 2883444]
306.
Schuchman EH. The pathogenesis and treatment of acid sphingomyelinase-deficient Niemann-Pick disease. J Inherit Metab Dis. 2007 Oct;30(5):654–63. [PubMed: 17632693]
307.
Pastores GM. Krabbe disease: an overview. Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S75–81. [PubMed: 20040316]
308.
Biffi A, Lucchini G, Rovelli A, et al. Metachromatic leukodystrophy: an overview of current and prospective treatments. Bone Marrow Transplant. 2008 Oct;42 Suppl 2:S2–6. [PubMed: 18978739]
309.
Vellodi A, Hobbs JR, O'Donnell NM, et al. Treatment of Niemann-Pick disease type B by allogeneic bone marrow transplantation. Br Med J (Clin Res Ed). 1987 Nov 28;295(6610):1375–6. [PMC free article: PMC1248536] [PubMed: 3121020]
310.
Shah AJ, Kapoor N, Crooks GM, et al. Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B. Pediatrics. 2005 Oct;116(4):1022–5. [PubMed: 16199719]
311.
Schneiderman J, Thormann K, Charrow J, et al. Correction of enzyme levels with allogeneic hematopoeitic progenitor cell transplantation in Niemann-Pick type B. Pediatr Blood Cancer. 2007 Dec;49(7):987–9. [PubMed: 17635007]
312.
Victor S, Coulter JB, Besley GT, et al. Niemann-Pick disease: sixteen-year follow-up of allogeneic bone marrow transplantation in a type B variant. J Inherit Metab Dis. 2003;26(8):775–85. [PubMed: 14739682]
313.
Krivit W, Shapiro EG, Peters C, et al. Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med. 1998 Apr 16;338(16):1119–26. [PubMed: 9545360]
314.
Escolar ML, Poe MD, Provenzale JM, et al. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med. 2005 May 19;352(20):2069–81. [PubMed: 15901860]
315.
Kurtzberg J, Richards K, Wenger D, et al. Correction of Krabbe disease with neonatal hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2002;8:97–8. [abstract]
316.
Krivit W, Shapiro E, Kennedy W, et al. Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation. N Engl J Med. 1990 Jan 4;322(1):28–32. [PubMed: 1967188]
317.
Malm G, Ringden O, Winiarski J, et al. Clinical outcome in four children with metachromatic leukodystrophy treated by bone marrow transplantation. Bone Marrow Transplant. 1996 Jun;17(6):1003–8. [PubMed: 8807106]
318.
Pierson TM, Bonnemann CG, Finkel RS, et al. Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy. Ann Neurol. 2008 Nov;64(5):583–7. [PMC free article: PMC2605197] [PubMed: 19067349]
319.
Stillman AE, Krivit W, Shapiro E, et al. Serial MR after bone marrow transplantation in two patients with metachromatic leukodystrophy. AJNR Am J Neuroradiol. 1994 Nov;15(10):1929–32. [PMC free article: PMC8334268] [PubMed: 7863944]
320.
Gorg M, Wilck W, Granitzny B, et al. Stabilization of juvenile metachromatic leukodystrophy after bone marrow transplantation: a 13-year follow-up. J Child Neurol. 2007 Sep;22(9):1139–42. [PubMed: 17890417]
321.
Wasserstein MP, Desnick RJ, Schuchman EH, et al. The natural history of type B Niemann-Pick disease: results from a 10-year longitudinal study. Pediatrics. 2004 Dec;114(6):e672–7. [PubMed: 15545621]
322.
Wenger DA, Suzuki K, Suzuki Y, et al. Galactosylceramide lipidosis: globoid cell leukodystrophy (Krabbe disease). In: Scriver CR, Sly WS, Childs B, Beaudet AL, editors. The metabolic and molecular bases of inherited disease. 8th ed. New York: McGraw-Hill; 2001. pp. 3669–94.
323.
Thomas GH. Disorders of glycoprotein degradation: alpha-mannosidosis, beta-mannosidosis, fucosidosis, and sialidosis. In: Scriver CR, Sly WS, Childs B, Beaudet AL, editors. The metabolic and molecular bases of inherited disease. 8th ed. New York: McGraw-Hill; 2001. pp. 3507–33.
324.
Willems PJ, Gatti R, Darby JK, et al. Fucosidosis revisited: a review of 77 patients. Am J Med Genet. 1991 Jan;38(1):111–31. [PubMed: 2012122]
325.
Heese BA. Current strategies in the management of lysosomal storage diseases. Semin Pediatr Neurol. 2008 Sep;15(3):119–26. [PubMed: 18708002]
326.
Miano M, Lanino E, Gatti R, et al. Four year follow-up of a case of fucosidosis treated with unrelated donor bone marrow transplantation. Bone Marrow Transplant. 2001 Apr;27(7):747–51. [PubMed: 11360116]
327.
Vellodi A, Cragg H, Winchester B, et al. Allogeneic bone marrow transplantation for fucosidosis. Bone Marrow Transplant. 1995 Jan;15(1):153–8. [PubMed: 7742750]
328.
Wall DA, Grange DK, Goulding P, et al. Bone marrow transplantation for the treatment of alpha-mannosidosis. J Pediatr. 1998 Aug;133(2):282–5. [PubMed: 9709723]
329.
Grewal SS, Shapiro EG, Krivit W, et al. Effective treatment of alpha-mannosidosis by allogeneic hematopoietic stem cell transplantation. J Pediatr. 2004 May;144(5):569–73. [PubMed: 15126988]
330.
Broomfield AA, Chakrapani A, Wraith JE. The effects of early and late bone marrow transplantation in siblings with alpha-mannosidosis. Is early haematopoietic cell transplantation the preferred treatment option? J Inherit Metab Dis. 2010 Feb 18 [PubMed: 20165920]
331.
Gotoda Y, Wakamatsu N, Kawai H, et al. Missense and nonsense mutations in the lysosomal alpha-mannosidase gene (MANB) in severe and mild forms of alpha-mannosidosis. Am J Hum Genet. 1998 Oct;63(4):1015–24. [PMC free article: PMC1377481] [PubMed: 9758606]
332.
Schutgens RB, Heymans HS, Wanders RJ, et al. Peroxisomal disorders: a newly recognised group of genetic diseases. Eur J Pediatr. 1986 Feb;144(5):430–40. [PubMed: 3514227]
333.
van Geel BM, Assies J, Wanders RJ, et al. X linked adrenoleukodystrophy: clinical presentation, diagnosis, and therapy. J Neurol Neurosurg Psychiatry. 1997 Jul;63(1):4–14. [PMC free article: PMC2169642] [PubMed: 9221959]
334.
Moser HW, Naidu S, Kumar AJ, et al. The adrenoleukodystrophies. Crit Rev Neurobiol. 1987;3(1):29–88. [PubMed: 3552451]
335.
Moser HW. Adrenoleukodystrophy. Curr Opin Neurol. 1995 Jun;8(3):221–6. [PubMed: 7551122]
336.
Moser HW. Komrower Lecture. Adrenoleukodystrophy: natural history, treatment and outcome. J Inherit Metab Dis. 1995;18(4):435–47. [PubMed: 7494402]
337.
Krivit W, Peters C, Shapiro EG. Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and Sly syndromes, and Gaucher disease type III. Curr Opin Neurol. 1999 Apr;12(2):167–76. [PubMed: 10226749]
338.
Ohi T, Takechi S, Itokazu N, et al. Two novel mutations in the adrenoleukodystrophy gene in two unrelated Japanese families and the long-term effect of bone marrow transplantation. J Neurol Sci. 2000 Aug 15;177(2):131–8. [PubMed: 10980309]
339.
Peters C, Charnas LR, Tan Y, et al. Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999. Blood. 2004 Aug 1;104(3):881–8. [PubMed: 15073029]
340.
Aubourg P, Blanche S, Jambaque I, et al. Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation. N Engl J Med. 1990 Jun 28;322(26):1860–6. [PubMed: 2348839]
341.
Shapiro E, Krivit W, Lockman L, et al. Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy. Lancet. 2000 Aug 26;356(9231):713–8. [PubMed: 11085690]
342.
Loes DJ, Stillman AE, Hite S, et al. Childhood cerebral form of adrenoleukodystrophy: short-term effect of bone marrow transplantation on brain MR observations. AJNR Am J Neuroradiol. 1994 Oct;15(9):1767–71. [PMC free article: PMC8333734] [PubMed: 7847226]
343.
Tolar J, Teitelbaum SL, Orchard PJ. Osteopetrosis. N Engl J Med. 2004 Dec 30;351(27):2839–49. [PubMed: 15625335]
344.
Stark Z, Savarirayan R. Osteopetrosis. Orphanet J Rare Dis. 2009;4:5. [PMC free article: PMC2654865] [PubMed: 19232111]
345.
Wilson CJ, Vellodi A. Autosomal recessive osteopetrosis: diagnosis, management, and outcome. Arch Dis Child. 2000 Nov;83(5):449–52. [PMC free article: PMC1718540] [PubMed: 11040159]
346.
Askmyr MK, Fasth A, Richter J. Towards a better understanding and new therapeutics of osteopetrosis. Br J Haematol. 2008 Mar;140(6):597–609. [PubMed: 18241253]
347.
Or R, Aker M, Shapira MY, et al. Allogeneic stem cell transplantation for the treatment of diseases associated with a deficiency in bone marrow products. Springer Semin Immunopathol. 2004 Nov;26(1-2):133–42. [PubMed: 15549305]
348.
Steward CG. Hematopoietic stem cell transplantation for osteopetrosis. Pediatr Clin North Am. 2010 Feb;57(1):171–80. [PubMed: 20307717]
349.
Driessen GJ, Gerritsen EJ, Fischer A, et al. Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report. Bone Marrow Transplant. 2003 Oct;32(7):657–63. [PubMed: 13130312]
350.
Eapen M, Davies SM, Ramsay NK, et al. Hematopoietic stem cell transplantation for infantile osteopetrosis. Bone Marrow Transplant. 1998 Nov;22(10):941–6. [PubMed: 9849690]
351.
Gerritsen EJ, Vossen JM, Fasth A, et al. Bone marrow transplantation for autosomal recessive osteopetrosis. A report from the Working Party on Inborn Errors of the European Bone Marrow Transplantation Group. J Pediatr. 1994 Dec;125(6 Pt 1):896–902. [PubMed: 7996361]
352.
National Cancer Institute Physician Data Query. Ewing Sarcoma Family of Tumors Treatment. 2010. [updated 07/02/2010]; www​.cancer.gov/cancertopics​/pdq/treatment​/ewings/healthprofessional/allpages.
353.
Burdach S, van Kaick B, Laws HJ, et al. Allogeneic and autologous stem-cell transplantation in advanced Ewing tumors. An update after long-term follow-up from two centers of the European Intergroup study EICESS. Stem-Cell Transplant Programs at Dusseldorf University Medical Center, Germany and St. Anna Kinderspital, Vienna, Austria. Ann Oncol. 2000 Nov;11(11):1451–62. [PubMed: 11142486]
354.
Burdach S, Meyer-Bahlburg A, Laws HJ, et al. High-dose therapy for patients with primary multifocal and early relapsed Ewing's tumors: results of two consecutive regimens assessing the role of total-body irradiation. J Clin Oncol. 2003 Aug 15;21(16):3072–8. [PubMed: 12915596]
355.
Burke MJ, Walterhouse DO, Jacobsohn DA, et al. Tandem high-dose chemotherapy with autologous peripheral hematopoietic progenitor cell rescue as consolidation therapy for patients with high-risk Ewing family tumors. Pediatr Blood Cancer. 2007 Aug;49(2):196–8. [PubMed: 17417796]
356.
Drabko K, Zawitkowska-Klaczynska J, Wojcik B, et al. Megachemotherapy followed by autologous stem cell transplantation in children with Ewing's sarcoma. Pediatr Transplant. 2005 Oct;9(5):618–21. [PubMed: 16176419]
357.
Hara J, Osugi Y, Ohta H, et al. Double-conditioning regimens consisting of thiotepa, melphalan and busulfan with stem cell rescue for the treatment of pediatric solid tumors. Bone Marrow Transplant. 1998 Jul;22(1):7–12. [PubMed: 9678789]
358.
Harimaya K, Oda Y, Matsuda S, et al. Primitive neuroectodermal tumor and extraskeletal Ewing sarcoma arising primarily around the spinal column: report of four cases and a review of the literature. Spine (Phila Pa 1976). 2003 Oct 1;28(19):E408–12. [PubMed: 14520055]
359.
Hawkins D, Barnett T, Bensinger W, et al. Busulfan, melphalan, and thiotepa with or without total marrow irradiation with hematopoietic stem cell rescue for poor-risk Ewing-Sarcoma-Family tumors. Med Pediatr Oncol. 2000 May;34(5):328–37. [PubMed: 10797354]
360.
Kasper B, Ho AD, Egerer G. Dose-intensive chemotherapy with stem cell support as a treatment strategy for bone and soft-tissue sarcomas. Curr Stem Cell Res Ther. 2006 Jan;1(1):29–35. [PubMed: 18220851]
361.
Kushner BH, Meyers PA. How effective is dose-intensive/myeloablative therapy against Ewing's sarcoma/primitive neuroectodermal tumor metastatic to bone or bone marrow? The Memorial Sloan-Kettering experience and a literature review. J Clin Oncol. 2001 Feb 1;19(3):870–80. [PubMed: 11157041]
362.
Laws HJ, van Kaick B, Pape H, et al. Relapse after high-dose therapy in relapsed Ewing's tumor patients: effects of maintenance chemotherapy in two selected patients? Onkologie. 2003 Dec;26(6):573–7. [PubMed: 14709933]
363.
Lucidarme N, Valteau-Couanet D, Oberlin O, et al. Phase II study of high-dose thiotepa and hematopoietic stem cell transplantation in children with solid tumors. Bone Marrow Transplant. 1998 Sep;22(6):535–40. [PubMed: 9758339]
364.
Meyers PA, Krailo MD, Ladanyi M, et al. High-dose melphalan, etoposide, total-body irradiation, and autologous stem-cell reconstitution as consolidation therapy for high-risk Ewing's sarcoma does not improve prognosis. J Clin Oncol. 2001 Jun 1;19(11):2812–20. [PubMed: 11387352]
365.
Navid F, Santana VM, Billups CA, et al. Concomitant administration of vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide for high-risk sarcomas: the St. Jude Children's Research Hospital experience. Cancer. 2006 Apr 15;106(8):1846–56. [PubMed: 16541446]
366.
Oberlin O, Rey A, Lyden E, et al. Prognostic factors in metastatic rhabdomyosarcomas: results of a pooled analysis from United States and European cooperative groups. J Clin Oncol. 2008 May 10;26(14):2384–9. [PMC free article: PMC4558625] [PubMed: 18467730]
367.
Ozkaynak MF, Sandoval C, Levendoglu-Tugal O, et al. A pilot trial of tandem autologous peripheral blood progenitor cell transplantation following high-dose thiotepa and carboplatin in children with poor-risk central nervous system tumors. Pediatr Hematol Oncol. 2004 Oct-Nov;21(7):635–45. [PubMed: 15626020]
368.
Pession A, Prete A, Locatelli F, et al. Phase I study of high-dose thiotepa with busulfan, etoposide, and autologous stem cell support in children with disseminated solid tumors. Med Pediatr Oncol. 1999 Nov;33(5):450–4. [PubMed: 10531568]
369.
Prete A, Rosito P, Alvisi P, et al. G-CSF-primed peripheral blood progenitor cells (PBPC) support in high-risk Ewing sarcoma of childhood. Bone Marrow Transplant. 1998 Dec;22 Suppl 5:S21–3. [PubMed: 9989884]
370.
Tanaka K, Matsunobu T, Sakamoto A, et al. High-dose chemotherapy and autologous peripheral blood stem-cell transfusion after conventional chemotherapy for patients with high-risk Ewing's tumors. J Orthop Sci. 2002;7(4):477–82. [PubMed: 12181663]
371.
Yaniv I, Cohen IJ, Stein J, et al. Tumor cells are present in stem cell harvests of Ewings sarcoma patients and their persistence following transplantation is associated with relapse. Pediatr Blood Cancer. 2004 May;42(5):404–9. [PubMed: 15049010]
372.
Diaz MA, Lassaletta A, Perez A, et al. High-dose busulfan and melphalan as conditioning regimen for autologous peripheral blood progenitor cell transplantation in high-risk ewing sarcoma patients: a long-term follow-up single-center study. Pediatric hematology and oncology. 2010 May;27(4):272–82. [PubMed: 20426518]
373.
Kwon SY, Won SC, Han JW, et al. Feasibility of sequential high-dose chemotherapy in advanced pediatric solid tumors. Pediatric hematology and oncology. 2010 Feb;27(1):1–12. [PubMed: 20121550]
374.
Ilari I, De Ioris MA, Milano GM, et al. Toxicity of high-dose chemotherapy with etoposide, thiotepa and CY in treating poor-prognosis Ewing's sarcoma family tumors: the experience of the Bambino Gesu Children's Hospital. Bone Marrow Transplant. 2010 Aug;45(8):1274–80. [PubMed: 20098456]
375.
Ladenstein R, Potschger U, Le Deley MC, et al. Primary disseminated multifocal Ewing sarcoma: results of the Euro-EWING 99 trial. Journal of clinical oncology : official journal of the American Society of Clinical Oncology. 2010 Jul 10;28(20):3284–91. [PubMed: 20547982]
376.
Burdach S, Thiel U, Schoniger M, et al. Total body MRI-governed involved compartment irradiation combined with high-dose chemotherapy and stem cell rescue improves long-term survival in Ewing tumor patients with multiple primary bone metastases. Bone Marrow Transplant. 2010 Mar;45(3):483–9. [PubMed: 19684633]
377.
Costa LJ, Rodriguez V, Porrata LF, et al. Autologous HSC transplant in t-MDS/AML using cells harvested prior to the development of the secondary malignancy. 2008 [PubMed: 18622421]
378.
Fazekas T, Wiesbauer P, Kronberger M, et al. Nodular pulmonary lesions in children after autologous stem cell transplantation: a source of misinterpretation. Br J Haematol. 2008 Feb;140(4):429–32. [PubMed: 18162118]
379.
Kogawa M, Asazuma T, Iso K, et al. Primary cervical spinal epidural Extra-osseous Ewing's sarcoma. Acta Neurochir (Wien). 2004 Sep;146(9):1051–3. discussion 3. [PubMed: 15340820]
380.
Koscielniak E, Gross-Wieltsch U, Treuner J, et al. Graft-versus-Ewing sarcoma effect and long-term remission induced by haploidentical stem-cell transplantation in a patient with relapse of metastatic disease. 2005 [PubMed: 15625381]
381.
Lucas KG, Schwartz C, Kaplan J. Allogeneic stem cell transplantation in a patient with relapsed Ewing sarcoma. Pediatr Blood Cancer. 2008 Jul;51(1):142–4. [PubMed: 18266223]
382.
Numata A, Shimoda K, Gondo H, et al. Therapy-related chronic myelogenous leukaemia following autologous stem cell transplantation for Ewing's sarcoma. Br J Haematol. 2002 Jun;117(3):613–6. [PubMed: 12028029]
383.
Bhatia S, Krailo MD, Chen Z, et al. Therapy-related myelodysplasia and acute myeloid leukemia after Ewing sarcoma and primitive neuroectodermal tumor of bone: A report from the Children's Oncology Group. Blood. 2007 Jan 1;109(1):46–51. [PMC free article: PMC1785079] [PubMed: 16985182]
384.
Kushner BH, Meyers PA, Gerald WL, et al. Very-high-dose short-term chemotherapy for poor-risk peripheral primitive neuroectodermal tumors, including Ewing's sarcoma, in children and young adults. J Clin Oncol. 1995 Nov;13(11):2796–804. [PubMed: 7595741]
385.
Milano GM, Cozza R, Ilari I, et al. High histologic and overall response to dose intensification of ifosfamide, carboplatin, and etoposide with cyclophosphamide, doxorubicin, and vincristine in patients with high-risk Ewing sarcoma family tumors: the Bambino Gesu Children's Hospital experience. Cancer. 2006 Apr 15;106(8):1838–45. [PubMed: 16532434]
386.
Sari N, Togral G, Cetindag MF, et al. Treatment results of the Ewing sarcoma of bone and prognostic factors. Pediatr Blood Cancer. 2010 Jan;54(1):19–24. [PubMed: 19760772]
387.
Van Winkle P, Angiolillo A, Krailo M, et al. Ifosfamide, carboplatin, and etoposide (ICE) reinduction chemotherapy in a large cohort of children and adolescents with recurrent/refractory sarcoma: the Children's Cancer Group (CCG) experience. Pediatr Blood Cancer. 2005 Apr;44(4):338–47. [PubMed: 15503297]
388.
Bernstein ML, Devidas M, Lafreniere D, et al. Intensive therapy with growth factor support for patients with Ewing tumor metastatic at diagnosis: Pediatric Oncology Group/Children's Cancer Group Phase II Study 9457--a report from the Children's Oncology Group. J Clin Oncol. 2006 Jan 1;24(1):152–9. [PubMed: 16382125]
389.
Kasper B, Lehnert T, Bernd L, et al. High-dose chemotherapy with autologous peripheral blood stem cell transplantation for bone and soft-tissue sarcomas. Bone Marrow Transplant. 2004 Jul;34(1):37–41. [PubMed: 15170176]
390.
National Cancer Institute Physician Data Query. Wilms Tumor and Other Childhood Kidney Tumors Treatment. 2010. [updated 11/15/2010]; www​.cancer.gov/cancertopics​/pdq/treatment​/wilms/healthprofessional/allpages.
391.
Breslow N, Olshan A, Beckwith J. Epidemiology of Wilms tumor. Medical Pediatric Oncology. 1993;21(3):172–81. [PubMed: 7680412]
392.
Kremens B, Gruhn B, Klingebiel T, et al. High-dose chemotherapy with autologous stem cell rescue in children with nephroblastoma. Bone Marrow Transplant. 2002 Dec;30(12):893–8. [PubMed: 12476282]
393.
Saarinen-Pihkala UM, Wikstrom S, Vettenranta K. Maximal preservation of renal function in patients with bilateral Wilms' tumor: therapeutic strategy of late kidney-sparing surgery and replacement of radiotherapy by high-dose melphalan and stem cell rescue. Bone Marrow Transplant. 1998 Jul;22(1):53–9. [PubMed: 9678796]
394.
Spreafico F, Bisogno G, Collini P, et al. Treatment of high-risk relapsed Wilms tumor with dose-intensive chemotherapy, marrow-ablative chemotherapy, and autologous hematopoietic stem cell support: experience by the Italian Association of Pediatric Hematology and Oncology. Pediatr Blood Cancer. 2008 Jul;51(1):23–8. [PubMed: 18293386]
395.
Campbell AD, Cohn SL, Reynolds M, et al. Treatment of relapsed Wilms' tumor with high-dose therapy and autologous hematopoietic stem-cell rescue: the experience at Children's Memorial Hospital. J Clin Oncol. 2004 Jul 15;22(14):2885–90. [PubMed: 15254057]
396.
Termuhlen AM, Grovas A, Klopfenstein K, et al. Autologous hematopoietic stem cell transplant with melphalan and thiotepa is safe and feasible in pediatric patients with low normalized glomerular filtration rate. Pediatr Transplant. 2006 Nov;10(7):830–4. [PubMed: 17032431]
397.
Kullendorff CM, Bekassy AN. Salvage treatment of relapsing Wilms' tumour by autologous bone marrow transplantation. Eur J Pediatr Surg. 1997 Jun;7(3):177–9. [PubMed: 9241510]
398.
Pein F, Michon J, Valteau-Couanet D, et al. High-dose melphalan, etoposide, and carboplatin followed by autologous stem-cell rescue in pediatric high-risk recurrent Wilms' tumor: a French Society of Pediatric Oncology study. J Clin Oncol. 1998 Oct;16(10):3295–301. [PubMed: 9779704]
399.
Valera ET, Cristofani L, Scrideli CA, et al. Megatherapy in the treatment of high-risk relapsed Wilms tumor. Pediatr Blood Cancer. 2004 Aug;43(2):186–8. [PubMed: 15236294]
400.
Hempel L, Kremens B, Weirich A, et al. High dose consolidation with autologous stem cell rescue (ASCR) for nephroblastoma initially treated according to the SIOP 9/GPOH trial and study. Klin Padiatr. 1996 Jul-Aug;208(4):186–9. [PubMed: 8776705]
401.
Park ES, Kang HJ, Shin HY, et al. Improved survival in patients with recurrent Wilms tumor: the experience of the Seoul National University Children's Hospital. J Korean Med Sci. 2006 Jun;21(3):436–40. [PMC free article: PMC2729947] [PubMed: 16778385]
402.
Abu-Ghosh AM, Krailo MD, Goldman SC, et al. Ifosfamide, carboplatin and etoposide in children with poor-risk relapsed Wilms' tumor: a Children's Cancer Group report. Ann Oncol. 2002 Mar;13(3):460–9. [PubMed: 11996479]
403.
Malogolowkin M, Cotton CA, Green DM, et al. Treatment of Wilms tumor relapsing after initial treatment with vincristine, actinomycin D, and doxorubicin. A report from the National Wilms Tumor Study Group. Pediatr Blood Cancer. 2008 Feb;50(2):236–41. [PubMed: 17539021]
404.
Hempel L, Patzer L, Misselwitz J, et al. Complete recovery of renal function in a Wilms' tumor patient after acute renal failure caused by autologous bone marrow transplantation (ABMT). Pediatr Hematol Oncol. 1998 May-Jun;15(3):255–60. [PubMed: 9615324]
405.
Maurer K, Heitger A, Schwaighofer H, et al. Double high-dose chemotherapy with autologous peripheral stem cell rescue in relapsed Wilms' tumor. Bone Marrow Transplant. 1997 Dec;20(12):1111–3. [PubMed: 9466289]
406.
Goldman SC, Bracho F, Davenport V, et al. Feasibility study of IL-11 and granulocyte colony-stimulating factor after myelosuppressive chemotherapy to mobilize peripheral blood stem cells from heavily pretreated patients. J Pediatr Hematol Oncol. 2001 Jun-Jul;23(5):300–5. [PubMed: 11464987]
407.
Dagher R, Kreissman S, Robertson KA, et al. High dose chemotherapy with autologous peripheral blood progenitor cell transplantation in an anephric child with multiply recurrent Wilms tumor. J Pediatr Hematol Oncol. 1998 Jul-Aug;20(4):357–60. [PubMed: 9703013]
408.
Brown E, Hebra A, Jenrette J, et al. Successful treatment of late, recurrent wilms tumor with high-dose chemotherapy and autologous stem cell rescue in third complete response. Journal of pediatric hematology/oncology : official journal of the American Society of Pediatric Hematology/Oncology. 2010 Aug;32(6):e241–3. [PubMed: 20628317]
409.
Lucas KG, Shapiro T, Freiberg A, et al. Matched unrelated umbilical cord blood transplantation for a patient with chemotherapy resistant Wilms tumor. Pediatric blood & cancer. 2010 Oct;55(4):763–5. [PubMed: 20589657]
410.
Tucci SJ, Cologna AJ, Suaid HJ, et al. Results of novel strategies for treatment of Wilms' tumor. Int Braz J Urol. 2007 Mar-Apr;33(2):195–201. discussion -3. [PubMed: 17488540]
411.
Dagher R, Helman L. Rhabdomyosarcoma: an overview. Oncologist. 1999;4(1):34–44. [PubMed: 10337369]
412.
Gurney JG, Young JL, Roffers SD, et al. Soft Tissue Sarcomas. In: Ries LAG, Smith MA, Gurney JG, Linet M, Tamra T, Young JL, et al., editors. Cancer Incidence and Survival among Children and Adolescents: United States SEER Program 1975-1995. NIH Pub. No. 99-4649 ed. Bethesda, MD: National Cancer Institute, SEER Program; 1999.
413.
Pappo AS, Anderson JR, Crist WM, et al. Survival after relapse in children and adolescents with rhabdomyosarcoma: A report from the Intergroup Rhabdomyosarcoma Study Group. J Clin Oncol. 1999 Nov;17(11):3487–93. [PubMed: 10550146]
414.
Carli M, Colombatti R, Oberlin O, et al. High-dose melphalan with autologous stem-cell rescue in metastatic rhabdomyosarcoma. J Clin Oncol. 1999 Sep;17(9):2796–803. [PubMed: 10561355]
415.
McDowell HP, Foot AB, Ellershaw C, et al. Outcomes in paediatric metastatic rhabdomyosarcoma: results of The International Society of Paediatric Oncology (SIOP) study MMT-98. Eur J Cancer. 2010 Jun;46(9):1588–95. [PubMed: 20338746]
416.
Breneman JC, Lyden E, Pappo AS, et al. Prognostic factors and clinical outcomes in children and adolescents with metastatic rhabdomyosarcoma--a report from the Intergroup Rhabdomyosarcoma Study IV. J Clin Oncol. 2003 Jan 1;21(1):78–84. [PubMed: 12506174]
417.
Carli M, Colombatti R, Oberlin O, et al. European intergroup studies (MMT4-89 and MMT4-91) on childhood metastatic rhabdomyosarcoma: final results and analysis of prognostic factors. J Clin Oncol. 2004 Dec 1;22(23):4787–94. [PubMed: 15570080]
418.
Williams BA, Williams KM, Doyle J, et al. Metastatic rhabdomyosarcoma: a retrospective review of patients treated at the hospital for sick children between 1989 and 1999. J Pediatr Hematol Oncol. 2004 Apr;26(4):243–7. [PubMed: 15087952]
419.
Bisogno G, Ferrari A, Prete A, et al. Sequential high-dose chemotherapy for children with metastatic rhabdomyosarcoma. Eur J Cancer. 2009 Nov;45(17):3035–41. [PubMed: 19783136]
420.
Koscielniak E, Klingebiel TH, Peters C, et al. Do patients with metastatic and recurrent rhabdomyosarcoma benefit from high-dose therapy with hematopoietic rescue? Report of the German/Austrian Pediatric Bone Marrow Transplantation Group. Bone Marrow Transplant. 1997 Feb;19(3):227–31. [PubMed: 9028550]
421.
Matsubara H, Makimoto A, Higa T, et al. Possible benefits of high-dose chemotherapy as intensive consolidation in patients with high-risk rhabdomyosarcoma who achieve complete remission with conventional chemotherapy. Pediatr Hematol Oncol. 2003 Apr-May;20(3):201–10. [PubMed: 12637216]
422.
Sato A, Imaizumi M, Saisho T, et al. Improved survival of children with advanced tumors by myeloablative chemotherapy and autologous peripheral blood stem cell transplantation in complete remission. Tohoku J Exp Med. 1998 Dec;186(4):255–65. [PubMed: 10328158]
423.
Shaw PJ, Pinkerton CR, Yaniv I. Melphalan combined with a carboplatin dose based on glomerular filtration rate followed by autologous stem cell rescue for children with solid tumours. Bone Marrow Transplant. 1996 Dec;18(6):1043–7. [PubMed: 8971371]
424.
Walterhouse DO, Hoover ML, Marymont MA, et al. High-dose chemotherapy followed by peripheral blood stem cell rescue for metastatic rhabdomyosarcoma: the experience at Chicago Children's Memorial Hospital. Med Pediatr Oncol. 1999 Feb;32(2):88–92. [PubMed: 9950194]
425.
Pappo AS, Lyden E, Breneman J, et al. Up-front window trial of topotecan in previously untreated children and adolescents with metastatic rhabdomyosarcoma: an intergroup rhabdomyosarcoma study. J Clin Oncol. 2001 Jan 1;19(1):213–9. [PubMed: 11134215]
426.
Raney B, Anderson J, Breneman J, et al. Results in patients with cranial parameningeal sarcoma and metastases (Stage 4) treated on Intergroup Rhabdomyosarcoma Study Group (IRSG) Protocols II-IV, 1978-1997: report from the Children's Oncology Group. Pediatr Blood Cancer. 2008 Jul;51(1):17–22. [PubMed: 18266224]
427.
Sandler E, Lyden E, Ruymann F, et al. Efficacy of ifosfamide and doxorubicin given as a phase II ‘window’ in children with newly diagnosed metastatic rhabdomyosarcoma: a report from the Intergroup Rhabdomyosarcoma Study Group. Med Pediatr Oncol. 2001 Nov;37(5):442–8. [PubMed: 11745872]
428.
Donker AE, Hoogerbrugge PM, Mavinkurve-Groothuis AM, et al. Metastatic rhabdomyosarcoma cured after chemotherapy and allogeneic SCT. INC, editor. 2009 [PubMed: 18762761]
429.
Grundy R, Anderson J, Gaze M, et al. Congenital alveolar rhabdomyosarcoma: clinical and molecular distinction from alveolar rhabdomyosarcoma in older children. Cancer. 2001 Feb 1;91(3):606–12. [PubMed: 11169945]
430.
Kuroiwa M, Sakamoto J, Shimada A, et al. Manifestation of alveolar rhabdomyosarcoma as primary cutaneous lesions in a neonate with Beckwith-Wiedemann syndrome. J Pediatr Surg. 2009 Mar;44(3):e31–5. [PubMed: 19302842]
431.
Kwan WH, Choi PH, Li CK, et al. Breast metastasis in adolescents with alveolar rhabdomyosarcoma of the extremities: report of two cases. Pediatr Hematol Oncol. 1996 May-Jun;13(3):277–85. [PubMed: 8735345]
432.
Misawa A, Hosoi H, Tsuchiya K, et al. Regression of refractory rhabdomyosarcoma after allogeneic stem-cell transplantation. Pediatr Hematol Oncol. 2003 Mar;20(2):151–5. [PubMed: 12554526]
433.
Moritake H, Ikuno Y, Tasaka H, et al. Donor leukocyte infusion after allogeneic bone marrow transplantation was not effective for relapsed rhabdomyosarcoma. Bone Marrow Transplant. 1998 Apr;21(7):725–6. [PubMed: 9578314]
434.
Oue T, Kubota A, Okuyama H, et al. Megatherapy with hematopoietic stem cell rescue as a preoperative treatment in unresectable pediatric malignancies. J Pediatr Surg. 2003 Jan;38(1):130–3. discussion -3. [PubMed: 12592635]
435.
Scully RE, Mark EJ, McNeely WF, et al. Case records of the Massachusetts General Hospital. Weekly clinicopathological exercises. Case 33-2000. A seven-year-old girl with the superior vena cava syndrome after treatment for a peripheral rhabdomyosarcoma. N Engl J Med. 2000 Oct 26;343(17):1249–57. [PubMed: 11071677]
436.
Doelken R, Weigel S, Schueler F, et al. Poor outcome of two children with relapsed state stage IV alveolar rhabdomyosarcoma after allogeneic stem cell transplantation. Pediatr Hematol Oncol. 2005 Dec;22(8):699–703. [PubMed: 16251176]
437.
Finger PT, Czechonska G, Demirci H, et al. Chemotherapy for retinoblastoma: a current topic. Drugs. 1999 Dec;58(6):983–96. [PubMed: 10651386]
438.
Dunkel IJ, Aledo A, Kernan NA, et al. Successful treatment of metastatic retinoblastoma. Cancer. 2000 Nov 15;89(10):2117–21. [PubMed: 11066053]
439.
Taguchi A, Suei Y, Ogawa I, et al. Metastatic retinoblastoma of the maxilla and mandible. Dentomaxillofac Radiol. 2005 Mar;34(2):126–31. [PubMed: 15829698]
440.
Moshfeghi DM, Wilson MW, Haik BG, et al. Retinoblastoma metastatic to the ovary in a patient with Waardenburg syndrome. Am J Ophthalmol. 2002 May;133(5):716–8. [PubMed: 11992879]
441.
Hertzberg H, Kremens B, Velten I, et al. Recurrent disseminated retinoblastoma in a 7-year-old girl treated successfully by high-dose chemotherapy and CD34-selected autologous peripheral blood stem cell transplantation. Bone Marrow Transplant. 2001 Mar;27(6):653–5. [PubMed: 11319597]
442.
Dai S, Dimaras H, Heon E, et al. Trilateral retinoblastoma with pituitary-hypothalamic dysfunction. Ophthalmic Genet. 2008 Sep;29(3):120–5. [PubMed: 18766991]
443.
Dimaras H, Heon E, Budning A, et al. Retinoblastoma CSF metastasis cured by multimodality chemotherapy without radiation. Ophthalmic Genet. 2009 Sep;30(3):121–6. [PubMed: 19941416]
444.
Dunkel IJ, Jubran RF, Gururangan S, et al. Trilateral retinoblastoma: potentially curable with intensive chemotherapy. Pediatr Blood Cancer. 2010 Mar;54(3):384–7. [PubMed: 19908299]
445.
Kremens B, Wieland R, Reinhard H, et al. High-dose chemotherapy with autologous stem cell rescue in children with retinoblastoma. Bone Marrow Transplant. 2003 Feb;31(4):281–4. [PubMed: 12621463]
446.
Matsubara H, Makimoto A, Higa T, et al. A multidisciplinary treatment strategy that includes high-dose chemotherapy for metastatic retinoblastoma without CNS involvement. Bone Marrow Transplant. 2005 Apr;35(8):763–6. [PubMed: 15750608]
447.
Namouni F, Doz F, Tanguy ML, et al. High-dose chemotherapy with carboplatin, etoposide and cyclophosphamide followed by a haematopoietic stem cell rescue in patients with high-risk retinoblastoma: a SFOP and SFGM study. Eur J Cancer. 1997 Dec;33(14):2368–75. [PubMed: 9616283]
448.
Rodriguez-Galindo C, Wilson MW, Haik BG, et al. Treatment of metastatic retinoblastoma. Ophthalmology. 2003 Jun;110(6):1237–40. [PubMed: 12799253]
449.
Dunkel IJ, Chan HS, Jubran R, et al. High-dose chemotherapy with autologous hematopoietic stem cell rescue for stage 4B retinoblastoma. Pediatr Blood Cancer. 2010 Jul 15;55(1):149–52. [PubMed: 20486181]
450.
Dunkel IJ, Khakoo Y, Kernan NA, et al. Intensive multimodality therapy for patients with stage 4a metastatic retinoblastoma. Pediatr Blood Cancer. 2010 Jul 15;55(1):55–9. [PubMed: 20486171]
451.
Antoneli CB, Steinhorst F, de Cassia Braga Ribeiro K, et al. Extraocular retinoblastoma: a 13-year experience. Cancer. 2003 Sep 15;98(6):1292–8. [PubMed: 12973854]
452.
Chang CY, Chiou TJ, Hwang B, et al. Retinoblastoma in Taiwan: survival rate and prognostic factors. Jpn J Ophthalmol. 2006 May-Jun;50(3):242–9. [PubMed: 16767380]
453.
Chantada GL, Fandino A, Mato G, et al. Phase II window of idarubicin in children with extraocular retinoblastoma. J Clin Oncol. 1999 Jun;17(6):1847–50. [PubMed: 10561224]
454.
Gunduz K, Muftuoglu O, Gunalp I, et al. Metastatic retinoblastoma clinical features, treatment, and prognosis. Ophthalmology. 2006 Sep;113(9):1558–66. [PubMed: 16828510]
455.
Schvartzman E, Chantada G, Fandino A, et al. Results of a stage-based protocol for the treatment of retinoblastoma. J Clin Oncol. 1996 May;14(5):1532–6. [PubMed: 8622068]
456.
Cozza R, De Ioris MA, Ilari I, et al. Metastatic retinoblastoma: single institution experience over two decades. Br J Ophthalmol. 2009 Sep;93(9):1163–6. [PubMed: 19556217]
457.
Jubran RF, Erdreich-Epstein A, Butturini A, et al. Approaches to treatment for extraocular retinoblastoma: Children's Hospital Los Angeles experience. J Pediatr Hematol Oncol. 2004 Jan;26(1):31–4. [PubMed: 14707710]
458.
Ladenstein R, Philip T, Lasset C, et al. Multivariate analysis of risk factors in stage 4 neuroblastoma patients over the age of one year treated with megatherapy and stem-cell transplantation: a report from the European Bone Marrow Transplantation Solid Tumor Registry. J Clin Oncol. 1998 Mar;16(3):953–65. [PubMed: 9508178]
459.
George RE, Li S, Medeiros-Nancarrow C, et al. High-risk neuroblastoma treated with tandem autologous peripheral-blood stem cell-supported transplantation: long-term survival update. J Clin Oncol. 2006 Jun 20;24(18):2891–6. [PubMed: 16782928]
460.
Grupp SA, Stern JW, Bunin N, et al. Rapid-sequence tandem transplant for children with high-risk neuroblastoma. Med Pediatr Oncol. 2000 Dec;35(6):696–700. [PubMed: 11107149]
461.
Grupp SA, Stern JW, Bunin N, et al. Tandem high-dose therapy in rapid sequence for children with high-risk neuroblastoma. J Clin Oncol. 2000 Jul;18(13):2567–75. [PubMed: 10893288]
462.
Hobbie WL, Moshang T, Carlson CA, et al. Late effects in survivors of tandem peripheral blood stem cell transplant for high-risk neuroblastoma. Pediatr Blood Cancer. 2008 Nov;51(5):679–83. [PMC free article: PMC2888471] [PubMed: 18623215]
463.
Marcus KJ, Shamberger R, Litman H, et al. Primary tumor control in patients with stage 3/4 unfavorable neuroblastoma treated with tandem double autologous stem cell transplants. J Pediatr Hematol Oncol. 2003 Dec;25(12):934–40. [PubMed: 14663275]
464.
Powell JL, Bunin NJ, Callahan C, et al. An unexpectedly high incidence of Epstein-Barr virus lymphoproliferative disease after CD34+ selected autologous peripheral blood stem cell transplant in neuroblastoma. Bone Marrow Transplant. 2004 Mar;33(6):651–7. [PMC free article: PMC7091929] [PubMed: 14730339]
465.
von Allmen D, Grupp S, Diller L, et al. Aggressive surgical therapy and radiotherapy for patients with high-risk neuroblastoma treated with rapid sequence tandem transplant. J Pediatr Surg. 2005 Jun;40(6):936–41. discussion 41. [PubMed: 15991174]
466.
Sung KW, Lee SH, Yoo KH, et al. Tandem high-dose chemotherapy and autologous stem cell rescue in patients over 1 year of age with stage 4 neuroblastoma. Bone Marrow Transplant. 2007 Jul;40(1):37–45. [PubMed: 17468771]
467.
Sung KW, Yoo KH, Chung EH, et al. Successive double high-dose chemotherapy with peripheral blood stem cell rescue collected during a single leukapheresis round in patients with high-risk pediatric solid tumors: a pilot study in a single center. Bone Marrow Transplant. 2003 Mar;31(6):447–52. [PubMed: 12665839]
468.
Kim EK, Kang HJ, Park JA, et al. Retrospective analysis of peripheral blood stem cell transplantation for the treatment of high-risk neuroblastoma. J Korean Med Sci. 2007 Sep;22(Suppl):S66–72. [PMC free article: PMC2694391] [PubMed: 17923758]
469.
Sung KW, Ahn HS, Cho B, et al. Efficacy of tandem high-dose chemotherapy and autologous stem cell rescue in patients over 1 year of age with stage 4 neuroblastoma: the Korean Society of Pediatric Hematology-Oncology experience over 6 years (2000-2005). J Korean Med Sci. 2010 May;25(5):691–7. [PMC free article: PMC2858826] [PubMed: 20436703]
470.
Einhorn LH, Williams SD, Chamness A, et al. High-dose chemotherapy and stem-cell rescue for metastatic germ-cell tumors. N Engl J Med. 2007 Jul 26;357(4):340–8. [PubMed: 17652649]
471.
Center for International Blood and Marrow Transplant Research (CIBMTR). Data on tandem and single autologous HSCT in GCT. Milwaukee, WI: 2010.
472.
Gidwani P, Levy A, Goodrich J, et al. Successful outcome with tandem myeloablative chemotherapy and autologous peripheral blood stem cell transplants in a patient with atypical teratoid/rhabdoid tumor of the central nervous system. J Neurooncol. 2008 Jun;88(2):211–5. [PubMed: 18317689]
473.
Fangusaro J, Finlay J, Sposto R, et al. Intensive chemotherapy followed by consolidative myeloablative chemotherapy with autologous hematopoietic cell rescue (AuHCR) in young children with newly diagnosed supratentorial primitive neuroectodermal tumors (sPNETs): report of the Head Start I and II experience. Pediatr Blood Cancer. 2008 Feb;50(2):312–8. [PubMed: 17668858]
474.
Dhall G, Grodman H, Ji L, et al. Outcome of children less than three years old at diagnosis with non-metastatic medulloblastoma treated with chemotherapy on the ‘Head Start’ I and II protocols. Pediatr Blood Cancer. 2008 Jun;50(6):1169–75. [PubMed: 18293379]
475.
Chi SN, Gardner SL, Levy AS, et al. Feasibility and response to induction chemotherapy intensified with high-dose methotrexate for young children with newly diagnosed high-risk disseminated medulloblastoma. J Clin Oncol. 2004 Dec 15;22(24):4881–7. [PubMed: 15611503]
476.
Gardner SL, Asgharzadeh S, Green A, et al. Intensive induction chemotherapy followed by high dose chemotherapy with autologous hematopoietic progenitor cell rescue in young children newly diagnosed with central nervous system atypical teratoid rhabdoid tumors. Pediatr Blood Cancer. 2008 Aug;51(2):235–40. [PubMed: 18381756]
477.
Perez-Martinez A, Lassaletta A, Gonzalez-Vicent M, et al. High-dose chemotherapy with autologous stem cell rescue for children with high risk and recurrent medulloblastoma and supratentorial primitive neuroectodermal tumors. J Neurooncol. 2005 Jan;71(1):33–8. 2005. [PubMed: 15719272]
478.
Taylor RE, Bailey CC, Robinson KJ, et al. Outcome for patients with metastatic (M2-3) medulloblastoma treated with SIOP/UKCCSG PNET-3 chemotherapy. Eur J Cancer. 2005 Mar;41(5):727–34. [PubMed: 15763649]
479.
Bandopadhayay P, Hassall TE, Rosenfeld JV, et al. ANZCCSG BabyBrain99; intensified systemic chemotherapy, second look surgery and involved field radiation in young children with central nervous system malignancy. Pediatr Blood Cancer. 2011 Jul 1;56(7):1055–61. [PubMed: 21298769]
480.
Aihara Y, Tsuruta T, Kawamata T, et al. Double high-dose chemotherapy followed by autologous peripheral blood stem cell transplantation for primary disseminated medulloblastoma: a report of 3 cases. J Pediatr Hematol Oncol. 2010 Mar;32(2):e70–4. [PubMed: 20168248]
481.
Geyer JR, Sposto R, Jennings M, et al. Multiagent chemotherapy and deferred radiotherapy in infants with malignant brain tumors: a report from the Children's Cancer Group. J Clin Oncol. 2005 Oct 20;23(30):7621–31. [PubMed: 16234523]
482.
Packer RJ, Gajjar A, Vezina G, et al. Phase III study of craniospinal radiation therapy followed by adjuvant chemotherapy for newly diagnosed average-risk medulloblastoma. J Clin Oncol. 2006 Sep 1;24(25):4202–8. [PubMed: 16943538]
483.
Fangusaro JR, Jubran RF, Allen J, et al. Brainstem primitive neuroectodermal tumors (bstPNET): results of treatment with intensive induction chemotherapy followed by consolidative chemotherapy with autologous hematopoietic cell rescue. Pediatr Blood Cancer. 2008 Mar;50(3):715–7. [PubMed: 17009232]
484.
Cohen KJ, Broniscer A, Glod J. Pediatric glial tumors. Curr Treat Options Oncol. 2001 Dec;2(6):529–36. [PubMed: 12057098]
485.
Finlay JL, Dhall G, Boyett JM, et al. Myeloablative chemotherapy with autologous bone marrow rescue in children and adolescents with recurrent malignant astrocytoma: outcome compared with conventional chemotherapy: a report from the Children's Oncology Group. Pediatr Blood Cancer. 2008 Dec;51(6):806–11. [PMC free article: PMC2844080] [PubMed: 18802947]
486.
Bay JO, Linassier C, Biron P, et al. Does high-dose carmustine increase overall survival in supratentorial high-grade malignant glioma? An EBMT retrospective study. Int J Cancer. 2007 Apr 15;120(8):1782–6. [PubMed: 17230505]
487.
DeVita VT, Hellman S, Rosenberg SA. Cancer, principles & practice of oncology. 7th ed. Philadelphia, PA: Lippincott Williams & Wilkins; 2005.
488.
Berger C, Thiesse P, Lellouch-Tubiana A, et al. Choroid plexus carcinomas in childhood: clinical features and prognostic factors. Neurosurgery. 1998 Mar;42(3):470–5. [PubMed: 9526979]
489.
Robertson PL, Zeltzer PM, Boyett JM, et al. Survival and prognostic factors following radiation therapy and chemotherapy for ependymomas in children: a report of the Children's Cancer Group. J Neurosurg. 1998 Apr;88(4):695–703. [PubMed: 9525716]
490.
Zacharoulis S, Levy A, Chi SN, et al. Outcome for young children newly diagnosed with ependymoma, treated with intensive induction chemotherapy followed by myeloablative chemotherapy and autologous stem cell rescue. Pediatr Blood Cancer. 2007 Jul;49(1):34–40. [PubMed: 16874765]
491.
Bertolone SJ, Yates AJ, Boyett JM, et al. Combined modality therapy for poorly differentiated gliomas of the posterior fossa in children: a Children's Cancer Group report. J Neurooncol. 2003 May;63(1):49–54. [PubMed: 12814254]
492.
Gilheeney SW, Khakoo Y, Souweidane M, et al. Thiotepa/topotecan/carboplatin with autologous stem cell rescue in recurrent/refractory/poor prognosis pediatric malignancies of the central nervous system. Pediatr Blood Cancer. 2010 Apr;54(4):591–5. [PubMed: 19998470]
493.
Bouffet E, Khelfaoui F, Philip I, et al. High-dose carmustine for high-grade gliomas in childhood. Cancer Chemother Pharmacol. 1997;39(4):376–9. [PubMed: 9025780]
494.
Bouffet E, Raquin M, Doz F, et al. Radiotherapy followed by high dose busulfan and thiotepa: a prospective assessment of high dose chemotherapy in children with diffuse pontine gliomas. Cancer. 2000 Feb 1;88(3):685–92. [PubMed: 10649264]
495.
Busca A, Miniero R, Besenzon L, et al. Etoposide-containing regimens with autologous bone marrow transplantation in children with malignant brain tumors. Childs Nerv Syst. 1997 Nov-Dec;13(11-12):572–7. [PubMed: 9454971]
496.
Dunkel IJ, Garvin JHJ, Goldman S, et al. High dose chemotherapy with autologous bone marrow rescue for children with diffuse pontine brain stem tumors. Children's Cancer Group. J Neurooncol. 1998 Mar;37(1):67–73. [PubMed: 9525840]
497.
Grill J, Kalifa C, Doz F, et al. A high-dose busulfan-thiotepa combination followed by autologous bone marrow transplantation in childhood recurrent ependymoma. A phase-II study. Pediatr Neurosurg. 1996 Jul;25(1):7–12. [PubMed: 9055328]
498.
Grovas AC, Boyett JM, Lindsley K, et al. Regimen-related toxicity of myeloablative chemotherapy with BCNU, thiotepa, and etoposide followed by autologous stem cell rescue for children with newly diagnosed glioblastoma multiforme: report from the Children's Cancer Group. Med Pediatr Oncol. 1999 Aug;33(2):83–7. [PubMed: 10398181]
499.
Gururangan S, Marina NM, Luo X, et al. Treatment of children with peripheral primitive neuroectodermal tumor or extraosseous Ewing's tumor with Ewing's-directed therapy. J Pediatr Hematol Oncol. 1998 Jan-Feb;20(1):55–61. [PubMed: 9482414]
500.
Jakacki RI, Siffert J, Jamison C, et al. Dose-intensive, time-compressed procarbazine, CCNU, vincristine (PCV) with peripheral blood stem cell support and concurrent radiation in patients with newly diagnosed high-grade gliomas. J Neurooncol. 1999 Aug;44(1):77–83. [PubMed: 10582673]
501.
Mahoney DHJ, Strother D, Camitta B, et al. High-dose melphalan and cyclophosphamide with autologous bone marrow rescue for recurrent/progressive malignant brain tumors in children: a pilot pediatric oncology group study. J Clin Oncol. 1996 Feb;14(2):382–8. [PubMed: 8636747]
502.
Massimino M, Gandola L, Luksch R, et al. Sequential chemotherapy, high-dose thiotepa, circulating progenitor cell rescue, and radiotherapy for childhood high-grade glioma. Neuro Oncol. 2005 Jan;7(1):41–8. [PMC free article: PMC1871624] [PubMed: 15701281]
503.
Thorarinsdottir HK, Rood B, Kamani N, et al. Outcome for children <4 years of age with malignant central nervous system tumors treated with high-dose chemotherapy and autologous stem cell rescue. Pediatr Blood Cancer. 2007 Mar;48(3):278–84. [PubMed: 16456857]
504.
Yule SM, Foreman NK, Mitchell C, et al. High-dose cyclophosphamide for poor-prognosis and recurrent pediatric brain tumors: a dose-escalation study. J Clin Oncol. 1997 Oct;15(10):3258–65. [PubMed: 9336363]
505.
Merchant TE, Zhu Y, Thompson SJ, et al. Preliminary results from a Phase II trial of conformal radiation therapy for pediatric patients with localised low-grade astrocytoma and ependymoma. Int J Radiat Oncol Biol Phys. 2002 Feb 1;52(2):325–32. [PubMed: 11872277]
506.
Mason WP, Goldman S, Yates AJ, et al. Survival following intensive chemotherapy with bone marrow reconstitution for children with recurrent intracranial ependymoma--a report of the Children's Cancer Group. J Neurooncol. 1998 Apr;37(2):135–43. [PubMed: 9524092]
507.
Ayan I, Darendeliler E, Kebudi R, et al. Evaluation of response to postradiation eight in one chemotherapy in childhood brain tumors. J Neurooncol. 1995 Oct;26(1):65–72. [PubMed: 8583246]
508.
Conter C, Carrie C, Bernier V, et al. Intracranial ependymomas in children: society of pediatric oncology experience with postoperative hyperfractionated local radiotherapy. Int J Radiat Oncol Biol Phys. 2009 Aug 1;74(5):1536–42. [PubMed: 19362789]
509.
De Sio L, Milano GM, Castellano A, et al. Temozolomide in resistant or relapsed pediatric solid tumors. Pediatr Blood Cancer. 2006 Jul;47(1):30–6. [PubMed: 16047361]
510.
Doireau V, Grill J, Zerah M, et al. Chemotherapy for unresectable and recurrent intramedullary glial tumours in children. Brain Tumours Subcommittee of the French Society of Paediatric Oncology (SFOP). Br J Cancer. 1999 Nov;81(5):835–40. [PMC free article: PMC2374296] [PubMed: 10555754]
511.
Grill J, Le Deley MC, Gambarelli D, et al. Postoperative chemotherapy without irradiation for ependymoma in children under 5 years of age: a multicenter trial of the French Society of Pediatric Oncology. J Clin Oncol. 2001 Mar 1;19(5):1288–96. [PubMed: 11230470]
512.
Grundy RG, Wilne SA, Weston CL, et al. Primary postoperative chemotherapy without radiotherapy for intracranial ependymoma in children: the UKCCSG/SIOP prospective study. Lancet Oncol. 2007 Aug;8(8):696–705. [PubMed: 17644039]
513.
Grundy RG, Wilne SH, Robinson KJ, et al. Primary postoperative chemotherapy without radiotherapy for treatment of brain tumours other than ependymoma in children under 3 years: results of the first UKCCSG/SIOP CNS 9204 trial. Eur J Cancer. 2010 Jan;46(1):120–33. [PubMed: 19818598]
514.
Horn B, Heideman R, Geyer R, et al. A multi-institutional retrospective study of intracranial ependymoma in children: identification of risk factors. J Pediatr Hematol Oncol. 1999 May-Jun;21(3):203–11. [PubMed: 10363853]
515.
Hurwitz CA, Strauss LC, Kepner J, et al. Paclitaxel for the treatment of progressive or recurrent childhood brain tumors: a pediatric oncology phase II study. J Pediatr Hematol Oncol. 2001 Jun-Jul;23(5):277–81. [PubMed: 11464982]
516.
Jaing TH, Wang HS, Tsay PK, et al. Multivariate analysis of clinical prognostic factors in children with intracranial ependymomas. J Neurooncol. 2004 Jul;68(3):255–61. [PubMed: 15332330]
517.
Kobrinsky NL, Packer RJ, Boyett JM, et al. Etoposide with or without mannitol for the treatment of recurrent or primarily unresponsive brain tumors: a Children's Cancer Group Study, CCG-9881. J Neurooncol. 1999;45(1):47–54. [PubMed: 10728909]
518.
Korones DN, Smith A, Foreman N, et al. Temozolomide and oral VP-16 for children and young adults with recurrent or treatment-induced malignant gliomas. Pediatr Blood Cancer. 2006 Jul;47(1):37–41. [PubMed: 16047359]
519.
White L, Kellie S, Gray E, et al. Postoperative chemotherapy in children less than 4 years of age with malignant brain tumors: promising initial response to a VETOPEC-based regimen. A Study of the Australian and New Zealand Children's Cancer Study Group (ANZCCSG). J Pediatr Hematol Oncol. 1998 Mar-Apr;20(2):125–30. [PubMed: 9544162]
520.
Kuhl J, Muller HL, Berthold F, et al. Preradiation chemotherapy of children and young adults with malignant brain tumors: results of the German pilot trial HIT'88/'89. Klin Padiatr. 1998 Jul-Aug;210(4):227–33. [PubMed: 9743957]
521.
MacDonald TJ, Arenson EB, Ater J, et al. Phase II study of high-dose chemotherapy before radiation in children with newly diagnosed high-grade astrocytoma: final analysis of Children's Cancer Group Study 9933. Cancer. 2005 Dec 15;104(12):2862–71. [PubMed: 16315242]
522.
Wrede B, Hasselblatt M, Peters O, et al. Atypical choroid plexus papilloma: clinical experience in the CPT-SIOP-2000 study. J Neurooncol. 2009 Dec;95(3):383–92. [PMC free article: PMC5637399] [PubMed: 19543851]
523.
Meikle PJ, Hopwood JJ, Clague AE, et al. Prevalence of lysosomal storage disorders. JAMA. 1999 Jan 20;281(3):249–54. [PubMed: 9918480]
524.
Poorthuis BJ, Wevers RA, Kleijer WJ, et al. The frequency of lysosomal storage diseases in The Netherlands. Hum Genet. 1999 Jul-Aug;105(1-2):151–6. [PubMed: 10480370]
525.
Sakata N, Kawa K, Kato K, et al. Unrelated donor marrow transplantation for congenital immunodeficiency and metabolic disease: an update of the experience of the Japan Marrow Donor Program. Int J Hematol. 2004 Aug;80(2):174–82. [PubMed: 15481448]
526.
McGovern MM, Desnick RJ. Lysosomal Storage Diseases. In: Cecil RL, Goldman L, Ausiello DA, editors. Cecil medicine. 23rd ed. Philadelphia: Saunders Elsevier; 2008. p. xxxiii. 3078 p.
527.
Gramatges MM, Dvorak CC, Regula DP, et al. Pathological evidence of Wolman's disease following hematopoietic stem cell transplantation despite correction of lysosomal acid lipase activity. Bone Marrow Transplant. 2009 Oct;44(7):449–50. [PubMed: 19308038]
528.
Tolar J, Grewal SS, Bjoraker KJ, et al. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome. Bone Marrow Transplant. 2008 Mar;41(6):531–5. [PubMed: 18037941]
529.
Stein J, Garty BZ, Dror Y, et al. Successful treatment of Wolman disease by unrelated umbilical cord blood transplantation. Eur J Pediatr. 2007 Jul;166(7):663–6. [PubMed: 17033804]
530.
Styczynski J, Tallamy B, Waxman I, et al. A pilot study of reduced toxicity conditioning with BU, fludarabine and alemtuzumab before the allogeneic hematopoietic SCT in children and adolescents. Bone Marrow Transplant. 2011 Jun;46(6):790–9. [PubMed: 20818441]
531.
Tolar J, Petryk A, Khan K, et al. Long-term metabolic, endocrine, and neuropsychological outcome of hematopoietic cell transplantation for Wolman disease. Bone Marrow Transplant. 2009 Jan;43(1):21–7. [PubMed: 18776925]
532.
Medicine UNLo. Niemann-Pick disease - Genetics Home Reference. National Library of Medicine; 2008. [cited 2011 January, 10, 2011] http://ghr​.nlm.nih.gov​/condition/niemann-pick-disease.
533.
Morel CF, Gassas A, Doyle J, et al. Unsuccessful treatment attempt: cord blood stem cell transplantation in a patient with Niemann-Pick disease type A. J Inherit Metab Dis. 2007 Nov;30(6):987. [PubMed: 17960492]
534.
Bayever E, August CS, Kamani N, et al. Allogeneic bone marrow transplantation for Niemann-Pick disease (type IA). Bone Marrow Transplant. 1992;10 Suppl 1:85–6. [PubMed: 1521097]
535.
McGovern MM, Desnick RJ. Mucolipidoses (Chapter 86.5). In: Kliegman R, Nelson WE, editors. Nelson textbook of pediatrics. 18th ed. Philadelphia: Saunders; 2007. p. lii. 3147 p.
536.
Li CK, Shing MM, Chik KW, et al. Unrelated umbilical cord blood transplantation in children: experience of the Hong Kong Red Cross Blood Transfusion Service. Hong Kong Med J. 2004 Apr;10(2):89–95. [PubMed: 15075428]
537.
Grewal S, Shapiro E, Braunlin E, et al. Continued neurocognitive development and prevention of cardiopulmonary complications after successful BMT for I-cell disease: a long-term follow-up report. Bone Marrow Transplant. 2003 Nov;32(9):957–60. [PubMed: 14561999]
538.
Imaizumi M, Gushi K, Kurobane I, et al. Long-term effects of bone marrow transplantation for inborn errors of metabolism: a study of four patients with lysosomal storage diseases. Acta Paediatr Jpn. 1994 Feb;36(1):30–6. [PubMed: 8165905]
539.
Bonnardeaux A, Bichet DG. Inherited Disorders of the Renal Tubule. In: Brenner BM, Rector FC, editors. Brenner & Rector's the kidney. 8th ed. Philadelphia: Saunders Elsevier; 2008. p. 2 v. (xxii, 2241, lxix p.)
540.
Dell KM, Avner ED. Renal Tubular Acidosis (Chapter 529). In: Kliegman R, Nelson WE, editors. Nelson textbook of pediatrics. 18th ed. Philadelphia: Saunders; 2007. p. lii.p. 3147.
541.
Aula P, Gahl WA. Disorders of Free Sialic Acid Storage. In: Scriver CR, editor. The metabolic and molecular bases of inherited disease. 8th ed. New York: McGraw-Hill; 2001. pp. 3635–68.
542.
Lemyre E, Russo P, Melancon SB, et al. Clinical spectrum of infantile free sialic acid storage disease. Am J Med Genet. 1999 Feb 19;82(5):385–91. [PubMed: 10069709]
543.
Ben-Simon-Schiff E, Zlotogora J, Abeliovich D, et al. Hunter syndrome among Jews in Israel. Biomed Pharmacother. 1994;48(8-9):381–4. [PubMed: 7858175]
544.
Martin RA. Mucopolysaccharidosis Type II. 2007. [updated 11/6/07]; www​.ncbi.nlm.nih.gov/bookshelf/br​.fcgi?book​=gene&part=hunter.
545.
Guffon N, Bertrand Y, Forest I, et al. Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years. J Pediatr. 2009 May;154(5):733–7. [PubMed: 19167723]
546.
McKinnis EJ, Sulzbacher S, Rutledge JC, et al. Bone marrow transplantation in Hunter syndrome. J Pediatr. 1996 Jul;129(1):145–8. [PubMed: 8757575]
547.
Takahashi Y, Sukegawa K, Aoki M, et al. Evaluation of accumulated mucopolysaccharides in the brain of patients with mucopolysaccharidoses by (1)H-magnetic resonance spectroscopy before and after bone marrow transplantation. Pediatr Res. 2001 Mar;49(3):349–55. [PubMed: 11228260]
548.
Li P, Thompson JN, Hug G, et al. Biochemical and molecular analysis in a patient with the severe form of Hunter syndrome after bone marrow transplantation. Am J Med Genet. 1996 Sep 6;64(4):531–5. [PubMed: 8870917]
549.
Vellodi A, Young E, Cooper A, et al. Long-term follow-up following bone marrow transplantation for Hunter disease. J Inherit Metab Dis. 1999 Jun;22(5):638–48. [PubMed: 10399096]
550.
Coppa GV, Gabrielli O, Zampini L, et al. Bone marrow transplantation in Hunter syndrome (mucopolysaccharidosis type II): two-year follow-up of the first Italian patient and review of the literature. Pediatr Med Chir. 1995 May-Jun;17(3):227–35. [PubMed: 7567644]
551.
Coppa GV, Gabrielli O, Cordiali R, et al. Bone marrow transplantation in a Hunter patient with P266H mutation. Int J Mol Med. 1999 Oct;4(4):433–6. [PubMed: 10493987]
552.
Bergstrom SK, Quinn JJ, Greenstein R, et al. Long-term follow-up of a patient transplanted for Hunter's disease type IIB: a case report and literature review. Bone Marrow Transplant. 1994 Oct;14(4):653–8. [PubMed: 7858546]
553.
Hoogerbrugge PM, Brouwer OF, Bordigoni P, et al. Allogeneic bone marrow transplantation for lysosomal storage diseases. The European Group for Bone Marrow Transplantation. Lancet. 1995 Jun 3;345(8962):1398–402. [PubMed: 7760610]
554.
Muenzer J, Gucsavas-Calikoglu M, McCandless SE, et al. A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Mol Genet Metab. 2007 Mar;90(3):329–37. [PubMed: 17185020]
555.
Muenzer J, Wraith JE, Beck M, et al. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med. 2006 Aug;8(8):465–73. [PubMed: 16912578]
556.
Page KM, Mendizabal AM, Prasad VK, et al. Posttransplant autoimmune hemolytic anemia and other autoimmune cytopenias are increased in very young infants undergoing unrelated donor umbilical cord blood transplantation. Biol Blood Marrow Transplant. 2008 Oct;14(10):1108–17. [PMC free article: PMC3735356] [PubMed: 18804040]
557.
Tokimasa S, Ohta H, Takizawa S, et al. Umbilical cord-blood transplantations from unrelated donors in patients with inherited metabolic diseases: Single-institute experience. Pediatr Transplant. 2008 Sep;12(6):672–6. [PubMed: 18798361]
558.
Seto T, Kono K, Morimoto K, et al. Brain magnetic resonance imaging in 23 patients with mucopolysaccharidoses and the effect of bone marrow transplantation. Ann Neurol. 2001 Jul;50(1):79–92. [PubMed: 11456314]
559.
Mullen CA, Thompson JN, Richard LA, et al. Unrelated umbilical cord blood transplantation in infancy for mucopolysaccharidosis type IIB (Hunter syndrome) complicated by autoimmune hemolytic anemia. Bone Marrow Transplant. 2000 May;25(10):1093–7. [PubMed: 10828871]
560.
Defendi GL, Varma S. Mucopolysaccharidosis Type III. 2009. [updated May 19, 2009; cited 2010 November]; http://emedicine​.medscape​.com/article/948540-overview.
561.
Ringden O, Remberger M, Svahn BM, et al. Allogeneic hematopoietic stem cell transplantation for inherited disorders: experience in a single center. Transplantation. 2006 Mar 15;81(5):718–25. [PubMed: 16534474]
562.
Lange MC, Teive HA, Troiano AR, et al. Bone marrow transplantation in patients with storage diseases: a developing country experience. Arq Neuropsiquiatr. 2006 Mar;64(1):1–4. [PubMed: 16622543]
563.
Sivakumur P, Wraith JE. Bone marrow transplantation in mucopolysaccharidosis type IIIA: a comparison of an early treated patient with his untreated sibling. J Inherit Metab Dis. 1999 Oct;22(7):849–50. [PubMed: 10518291]
564.
Vellodi A, Young E, New M, et al. Bone marrow transplantation for Sanfilippo disease type B. J Inherit Metab Dis. 1992;15(6):911–8. [PubMed: 1293388]
565.
Northover H, Cowie RA, Wraith JE. Mucopolysaccharidosis type IVA (Morquio syndrome): a clinical review. J Inherit Metab Dis. 1996;19(3):357–65. [PubMed: 8803780]
566.
Gatzoulis MA, Vellodi A, Redington AN. Cardiac involvement in mucopolysaccharidoses: effects of allogeneic bone marrow transplantation. Arch Dis Child. 1995 Sep;73(3):259–60. [PMC free article: PMC1511298] [PubMed: 7492172]
567.
Goker-Alpan O, Wiggs EA, Eblan MJ, et al. Cognitive outcome in treated patients with chronic neuronopathic Gaucher disease. J Pediatr. 2008 Jul;153(1):89–94. [PubMed: 18571543]
568.
Chen RL, Hou JW, Chang PY, et al. Matched unrelated bone marrow transplantation without splenectomy for a child with Gaucher disease caused by homozygosity of the L444P mutation, who also suffered from schizencephaly. J Pediatr Hematol Oncol. 2007 Jan;29(1):57–9. [PubMed: 17230068]
569.
Tsai P, Lipton JM, Sahdev I, et al. Allogenic bone marrow transplantation in severe Gaucher disease. Pediatr Res. 1992 May;31(5):503–7. [PubMed: 1603628]
570.
Schiffmann R, Fitzgibbon EJ, Harris C, et al. Randomized, controlled trial of miglustat in Gaucher's disease type 3. Ann Neurol. 2008 Nov;64(5):514–22. [PMC free article: PMC2605167] [PubMed: 19067373]
571.
El-Beshlawy A, Ragab L, Youssry I, et al. Enzyme replacement therapy and bony changes in Egyptian paediatric Gaucher disease patients. J Inherit Metab Dis. 2006 Feb;29(1):92–8. [PubMed: 16601874]
572.
Chan LL, Lin HP. Enzyme replacement therapy for Gaucher Disease: the only experience in Malaysia. Med J Malaysia. 2002 Sep;57(3):348–52. [PubMed: 12440275]
573.
Banjar H. Pulmonary involvement of Gaucher's disease in children: a common presentation in Saudi Arabia. Ann Trop Paediatr. 1998 Mar;18(1):55–9. [PubMed: 9692003]
574.
Schiffmann R, Heyes MP, Aerts JM, et al. Prospective study of neurological responses to treatment with macrophage-targeted glucocerebrosidase in patients with type 3 Gaucher's disease. Ann Neurol. 1997 Oct;42(4):613–21. [PubMed: 9382473]
575.
Erikson A, Astrom M, Mansson JE. Enzyme infusion therapy of the Norrbottnian (type 3) Gaucher disease. Neuropediatrics. 1995 Aug;26(4):203–7. [PubMed: 8544959]
576.
McGovern MM, Desnick RJ. Disorders of Glycoprotein Degradation and Structure (Chapter 87.6). In: Kliegman R, Nelson WE, editors. Nelson textbook of pediatrics. 18th ed. Philadelphia: Saunders; 2007. p. lii. 3147 p.
577.
Malm G, Mansson JE, Winiarski J, et al. Five-year follow-up of two siblings with aspartylglucosaminuria undergoing allogeneic stem-cell transplantation from unrelated donors. Transplantation. 2004 Aug 15;78(3):415–9. [PubMed: 15316370]
578.
Arvio M, Sauna-Aho O, Peippo M. Bone marrow transplantation for aspartylglucosaminuria: follow-up study of transplanted and non-transplanted patients. J Pediatr. 2001 Feb;138(2):288–90. [PubMed: 11174635]
579.
Autti T, Rapola J, Santavuori P, et al. Bone marrow transplantation in aspartylglucosaminuria--histopathological and MRI study. Neuropediatrics. 1999 Dec;30(6):283–8. [PubMed: 10706021]
580.
Laitinen A, Hietala M, Haworth JC, et al. Two novel mutations in a Canadian family with aspartylglucosaminuria and early outcome post bone marrow transplantation. Clin Genet. 1997 Mar;51(3):174–8. [PubMed: 9137882]
581.
Bedilu R, Nummy KA, Cooper A, et al. Variable clinical presentation of lysosomal beta-mannosidosis in patients with null mutations. Mol Genet Metab. 2002 Dec;77(4):282–90. [PubMed: 12468273]
582.
Altarescu G, Sun M, Moore DF, et al. The neurogenetics of mucolipidosis type IV. Neurology. 2002 Aug 13;59(3):306–13. [PubMed: 12182165]
583.
Bonney DK, O'Meara A, Shabani A, et al. Successful allogeneic bone marrow transplant for Niemann-Pick disease type C2 is likely to be associated with a severe ‘graft versus substrate’ effect. J Inherit Metab Dis. 2010 Apr 15 [PubMed: 20393800]
584.
Hsu YS, Hwu WL, Huang SF, et al. Niemann-Pick disease type C (a cellular cholesterol lipidosis) treated by bone marrow transplantation. Bone Marrow Transplant. 1999 Jul;24(1):103–7. [PubMed: 10435744]
585.
Patterson MC, Vecchio D, Jacklin E, et al. Long-term miglustat therapy in children with Niemann-Pick disease type C. J Child Neurol. 2010 Mar;25(3):300–5. [PubMed: 19822772]
586.
Pineda M, Wraith JE, Mengel E, et al. Miglustat in patients with Niemann-Pick disease Type C (NP-C): a multicenter observational retrospective cohort study. Mol Genet Metab. 2009 Nov;98(3):243–9. [PubMed: 19656703]
587.
Paciorkowski AR, Westwell M, Ounpuu S, et al. Motion analysis of a child with Niemann-Pick disease type C treated with miglustat. Mov Disord. 2008 Jan;23(1):124–8. [PubMed: 17973331]
588.
Patterson MC, Vecchio D, Prady H, et al. Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Lancet Neurol. 2007 Sep;6(9):765–72. [PubMed: 17689147]
589.
Kishnani PS, Chen YT. Defects in Metabolism of Carbohydrates (Chapter 87). In: Kliegman R, Nelson WE, editors. Nelson textbook of pediatrics. 18th ed. Philadelphia: Saunders; 2007. p. lii. 3147 p.
590.
Amalfitano A, Bengur AR, Morse RP, et al. Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial. Genet Med. 2001 Mar-Apr;3(2):132–8. [PubMed: 11286229]
591.
Klinge L, Straub V, Neudorf U, et al. Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial. Neuromuscul Disord. 2005 Jan;15(1):24–31. [PubMed: 15639117]
592.
Murray B, Mitsumoto H. Disorders of Upper and Lower Motor Neurons. In: Bradley WG, editor. Neurology in clinical practice. 5th ed. Philadelphia, PA: Butterworth-Heinemann/Elsevier; 2008. p. 2 v. xvi, 2488, lxxx p.
593.
Johnston MV. Adrenoleukodystrophy (Chapter 599.3). In: Kliegman R, Nelson WE, editors. Nelson textbook of pediatrics. 18th ed. Philadelphia: Saunders; 2007. p. lii. 3147 p.
594.
Bissonnette B. Syndromes : rapid recognition and perioperative management. 1st ed. New York: McGraw-Hill; 2005.
595.
Moser HW, Linke T, Fensom AH, et al. Acid ceramidase deficiency: Farber lipogranulomatosis. In: Scriver CR, Sly WS, Childs B, Beaudet AL, editors. The metabolic and molecular bases of inherited disease. 8th ed. New York: McGraw-Hill; 2001. pp. 3573–85.
596.
Ehlert K, Roth J, Frosch M, et al. Farber's disease without central nervous system involvement: bone-marrow transplantation provides a promising new approach. 2006 [PMC free article: PMC1798467] [PubMed: 17105855]
597.
Vormoor J, Ehlert K, Groll AH, et al. Successful hematopoietic stem cell transplantation in Farber disease. J Pediatr. 2004 Jan;144(1):132–4. [PubMed: 14722533]
598.
Yeager AM, Uhas KA, Coles CD, et al. Bone marrow transplantation for infantile ceramidase deficiency (Farber disease). Bone Marrow Transplant. 2000 Aug;26(3):357–63. [PubMed: 10967581]
599.
Brunetti-Pierri N, Scaglia F. GM1 gangliosidosis: review of clinical, molecular, and therapeutic aspects. Mol Genet Metab. 2008 Aug;94(4):391–6. [PubMed: 18524657]
600.
Johnston MV. Neurodegenerative Disorders of Childhood (Chapter 599). In: Kliegman R, Nelson WE, editors. Nelson textbook of pediatrics. 18th ed. Philadelphia: Saunders; 2007. p. lii. 3147 p.
601.
Shield JP, Stone J, Steward CG. Bone marrow transplantation correcting beta-galactosidase activity does not influence neurological outcome in juvenile GM1-gangliosidosis. J Inherit Metab Dis. 2005;28(5):797–8. [PubMed: 16151914]
602.
Jacobs JF, Willemsen MA, Groot-Loonen JJ, et al. Allogeneic BMT followed by substrate reduction therapy in a child with subacute Tay-Sachs disease. 2005 [PubMed: 16151419]
603.
Maegawa GH, Banwell BL, Blaser S, et al. Substrate reduction therapy in juvenile GM2 gangliosidosis. Mol Genet Metab. 2009 Sep-Oct;98(1-2):215–24. 2009. [PubMed: 19595619]
604.
Sieving PA, Caruso RC. Retinitis Pigmentosa and Related Disorders. In: Yanoff M, Duker JS, editors. Ophthalmology. 3rd ed. St. Louis, MO: Mosby; 2008.
605.
Lonnqvist T, Vanhanen SL, Vettenranta K, et al. Hematopoietic stem cell transplantation in infantile neuronal ceroid lipofuscinosis. Neurology. 2001 Oct 23;57(8):1411–6. [PubMed: 11673581]
606.
Sakuraba H, Suzuki Y, Akagi M, et al. beta-Galactosidase-neuraminidase deficiency (galactosialidosis): clinical, pathological, and enzymatic studies in a postmortem case. Ann Neurol. 1983 May;13(5):497–503. [PubMed: 6408977]
607.
Milanetti F, Abinun M, Voltarelli JC, et al. Autologous hematopoietic stem cell transplantation for childhood autoimmune disease. Pediatr Clin North Am. 2010 Feb;57(1):239–71. [PubMed: 20307720]
608.
Gale EA. The rise of childhood type 1 diabetes in the 20th century. Diabetes. 2002 Dec;51(12):3353–61. [PubMed: 12453886]
609.
Notkins AL, Lernmark A. Autoimmune type 1 diabetes: resolved and unresolved issues. J Clin Invest. 2001 Nov;108(9):1247–52. [PMC free article: PMC209446] [PubMed: 11696564]
610.
Nathan DM. Long-term complications of diabetes mellitus. N Engl J Med. 1993 Jun 10;328(23):1676–85. [PubMed: 8487827]
611.
Genuth S. Insights from the diabetes control and complications trial/epidemiology of diabetes interventions and complications study on the use of intensive glycemic treatment to reduce the risk of complications of type 1 diabetes. Endocr Pract. 2006 Jan-Feb;12 Suppl 1:34–41. [PubMed: 16627378]
612.
Couri CE, Oliveira MC, Stracieri AB, et al. C-peptide levels and insulin independence following autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA. 2009 Apr 15;301(15):1573–9. [PubMed: 19366777]
613.
Crino A, Schiaffini R, Ciampalini P, et al. A two year observational study of nicotinamide and intensive insulin therapy in patients with recent onset type 1 diabetes mellitus. J Pediatr Endocrinol Metab. 2005 Aug;18(8):749–54. [PubMed: 16200840]
614.
Mastrandrea L, Yu J, Behrens T, et al. Etanercept treatment in children with new-onset type 1 diabetes: pilot randomized, placebo-controlled, double-blind study. Diabetes Care. 2009 Jul;32(7):1244–9. [PMC free article: PMC2699714] [PubMed: 19366957]
615.
Hochberg MC. Updating the American College of Rheumatology revised criteria for the classification of systemic lupus erythematosus. Arthritis Rheum. 1997 Sep;40(9):1725. [PubMed: 9324032]
616.
Klein-Gitelman M, Reiff A, Silverman ED. Systemic lupus erythematosus in childhood. Rheum Dis Clin North Am. 2002 Aug;28(3):561–77. vi–vii. [PubMed: 12380370]
617.
Ravelli A, Ruperto N, Martini A. Outcome in juvenile onset systemic lupus erythematosus. Curr Opin Rheumatol. 2005 Sep;17(5):568–73. [PubMed: 16093835]
618.
Bernatsky S, Clarke A, Gladman DD, et al. Mortality related to cerebrovascular disease in systemic lupus erythematosus. Lupus. 2006;15(12):835–9. [PubMed: 17211987]
619.
Tucker LB. Making the diagnosis of systemic lupus erythematosus in children and adolescents. Lupus. 2007;16(8):546–9. [PubMed: 17711886]
620.
Musso M, Porretto F, Crescimanno A, et al. Intense immunosuppressive therapy followed by autologous peripheral blood selected progenitor cell reinfusion for severe autoimmune disease. Am J Hematol. 2001 Feb;66(2):75–9. [PubMed: 11421302]
621.
Statkute L, Traynor A, Oyama Y, et al. Antiphospholipid syndrome in patients with systemic lupus erythematosus treated by autologous hematopoietic stem cell transplantation. Blood. 2005 Oct 15;106(8):2700–9. [PubMed: 15870182]
622.
Chen J, Wang Y, Kunkel G, et al. Use of CD34+ autologous stem cell transplantation in the treatment of children with refractory systemic lupus erythematosus. Clin Rheumatol. 2005 Sep;24(5):464–8. [PubMed: 15662487]
623.
Lisukov IA, Sizikova SA, Kulagin AD, et al. High-dose immunosuppression with autologous stem cell transplantation in severe refractory systemic lupus erythematosus. Lupus. 2004;13(2):89–94. [PubMed: 14995000]
624.
Wulffraat NM, Sanders EA, Kamphuis SS, et al. Prolonged remission without treatment after autologous stem cell transplantation for refractory childhood systemic lupus erythematosus. Arthritis Rheum. 2001 Mar;44(3):728–31. [PubMed: 11263789]
625.
Brunner M, Greinix HT, Redlich K, et al. Autologous blood stem cell transplantation in refractory systemic lupus erythematosus with severe pulmonary impairment: a case report. Arthritis Rheum. 2002 Jun;46(6):1580–4. PMID: [PubMed: 12115189]
626.
Trysberg E, Lindgren I, Tarkowski A. Autologous stem cell transplantation in a case of treatment resistant central nervous system lupus. Ann Rheum Dis. 2000 Mar;59(3):236–8. [PMC free article: PMC1753087] [PubMed: 10700436]
627.
Ravelli A, Martini A. Juvenile idiopathic arthritis. Lancet. 2007 Mar 3;369(9563):767–78. [PubMed: 17336654]
628.
de Kleer I, Vastert B, Klein M, et al. Autologous stem cell transplantation for autoimmunity induces immunologic self-tolerance by reprogramming autoreactive T cells and restoring the CD4+CD25+ immune regulatory network. Blood. 2006 Feb 15;107(4):1696–702. [PubMed: 16263787]
629.
Roord ST, de Jager W, Boon L, et al. Autologous bone marrow transplantation in autoimmune arthritis restores immune homeostasis through CD4+CD25+Foxp3+ regulatory T cells. Blood. 2008 May 15;111(10):5233–41. [PubMed: 18256318]
630.
De Kleer IM, Brinkman DM, Ferster A, et al. Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality, and transplant related morbidity. Ann Rheum Dis. 2004 Oct;63(10):1318–26. [PMC free article: PMC1754760] [PubMed: 15361393]
631.
Kishimoto T, Hamazaki T, Yasui M, et al. Autologous hematopoietic stem cell transplantation for 3 patients with severe juvenile rheumatoid arthritis. Int J Hematol. 2003 Dec;78(5):453–6. [PubMed: 14704040]
632.
Nakagawa R, Kawano Y, Yoshimura E, et al. Intense immunosuppression followed by purified blood CD34+ cell autografting in a patient with refractory juvenile rheumatoid arthritis. Bone Marrow Transplant. 2001 Feb;27(3):333–6. [PubMed: 11277183]
633.
Rabusin M, Andolina M, Maximova N, et al. Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease. Haematologica. 2000 Nov;85(11 Suppl):81–5. [PubMed: 11268330]
634.
Annaloro C, Onida F, Lambertenghi Deliliers G. Autologous hematopoietic stem cell transplantation in autoimmune diseases. Expert Rev Hematol. 2009 Dec;2(6):699–715. [PubMed: 21082959]
635.
Altman RD, Medsger TA Jr, Bloch DA, et al. Predictors of survival in systemic sclerosis (scleroderma). Arthritis Rheum. 1991 Apr;34(4):403–13. [PubMed: 1901491]
636.
Bryan C, Knight C, Black CM, et al. Prediction of five-year survival following presentation with scleroderma: development of a simple model using three disease factors at first visit. Arthritis Rheum. 1999 Dec;42(12):2660–5. [PubMed: 10616015]
637.
Bulpitt KJ, Clements PJ, Lachenbruch PA, et al. Early undifferentiated connective tissue disease: III. Outcome and prognostic indicators in early scleroderma (systemic sclerosis). Ann Intern Med. 1993 Apr 15;118(8):602–9. [PubMed: 8452326]
638.
Ferri C, Valentini G, Cozzi F, et al. Systemic sclerosis: demographic, clinical, and serologic features and survival in 1,012 Italian patients. Medicine (Baltimore). 2002 Mar;81(2):139–53. [PubMed: 11889413]
639.
Scussel-Lonzetti L, Joyal F, Raynauld JP, et al. Predicting mortality in systemic sclerosis: analysis of a cohort of 309 French Canadian patients with emphasis on features at diagnosis as predictive factors for survival. Medicine (Baltimore). 2002 Mar;81(2):154–67. [PubMed: 11889414]
640.
Steen VD, Medsger TA Jr. Severe organ involvement in systemic sclerosis with diffuse scleroderma. Arthritis Rheum. 2000 Nov;43(11):2437–44. [PubMed: 11083266]
641.
Davas EM, Peppas C, Maragou M, et al. Intravenous cyclophosphamide pulse therapy for the treatment of lung disease associated with scleroderma. Clin Rheumatol. 1999;18(6):455–61. [PubMed: 10638770]
642.
Griffiths B, Miles S, Moss H, et al. Systemic sclerosis and interstitial lung disease: a pilot study using pulse intravenous methylprednisolone and cyclophosphamide to assess the effect on high resolution computed tomography scan and lung function. J Rheumatol. 2002 Nov;29(11):2371–8. [PubMed: 12415594]
643.
Varai G, Earle L, Jimenez SA, et al. A pilot study of intermittent intravenous cyclophosphamide for the treatment of systemic sclerosis associated lung disease. J Rheumatol. 1998 Jul;25(7):1325–9. [PubMed: 9676764]
644.
Farge D, Passweg J, van Laar JM, et al. Autologous stem cell transplantation in the treatment of systemic sclerosis: report from the EBMT/EULAR Registry. Ann Rheum Dis. 2004 Aug;63(8):974–81. [PMC free article: PMC1755096] [PubMed: 15249325]
645.
Leary SM, Thompson AJ. Primary progressive multiple sclerosis : current and future treatment options. CNS Drugs. 2005;19(5):369–76. [PubMed: 15907149]
646.
Krupp LB, Banwell B, Tenembaum S. Consensus definitions proposed for pediatric multiple sclerosis and related disorders. Neurology. 2007 Apr 17;68(16 Suppl 2):S7–12. [PubMed: 17438241]
647.
Yeh EA, Chitnis T, Krupp L, et al. Pediatric multiple sclerosis. Nat Rev Neurol. 2009 Nov;5(11):621–31. [PubMed: 19826402]
648.
Boiko A, Vorobeychik G, Paty D, et al. Early onset multiple sclerosis: a longitudinal study. Neurology. 2002 Oct 8;59(7):1006–10. [PubMed: 12370453]
649.
Chitnis T, Glanz B, Jaffin S, et al. Demographics of pediatric-onset multiple sclerosis in an MS center population from the Northeastern United States. Mult Scler. 2009 May;15(5):627–31. [PubMed: 19299440]
650.
Ghezzi A, Deplano V, Faroni J, et al. Multiple sclerosis in childhood: clinical features of 149 cases. Mult Scler. 1997 Feb;3(1):43–6. [PubMed: 9160345]
651.
Banwell B, Kennedy J, Sadovnick D, et al. Incidence of acquired demyelination of the CNS in Canadian children. Neurology. 2009 Jan 20;72(3):232–9. [PubMed: 19153370]
652.
Poser CM, Brinar VV. The nature of multiple sclerosis. Clin Neurol Neurosurg. 2004 Jun;106(3):159–71. [PubMed: 15177764]
653.
Fagius J, Lundgren J, Oberg G. Early highly aggressive MS successfully treated by hematopoietic stem cell transplantation. Mult Scler. 2009 Feb;15(2):229–37. [PubMed: 18805841]
654.
Kimiskidis V, Sakellari I, Tsimourtou V, et al. Autologous stem-cell transplantation in malignant multiple sclerosis: a case with a favorable long-term outcome. Mult Scler. 2008 Mar;14(2):278–83. [PubMed: 17942513]
655.
Mancardi GL, Murialdo A, Rossi P, et al. Autologous stem cell transplantation as rescue therapy in malignant forms of multiple sclerosis. Mult Scler. 2005 Jun;11(3):367–71. [PubMed: 15957523]
656.
Loftus EV Jr, Schoenfeld P, Sandborn WJ. The epidemiology and natural history of Crohn's disease in population-based patient cohorts from North America: a systematic review. Aliment Pharmacol Ther. 2002 Jan;16(1):51–60. [PubMed: 11856078]
657.
Vermeire S, van Assche G, Rutgeerts P. Review article: Altering the natural history of Crohn's disease--evidence for and against current therapies. Aliment Pharmacol Ther. 2007 Jan 1;25(1):3–12. [PubMed: 17229216]
658.
Oyama Y, Craig RM, Traynor AE, et al. Autologous hematopoietic stem cell transplantation in patients with refractory Crohn's disease. Gastroenterology. 2005 Mar;128(3):552–63. [PubMed: 15765390]
659.
Burt RK, Craig RM, Milanetti F, et al. Autologous nonmyeloablative hematopoietic stem cell transplantation in patients with severe anti-TNF refractory Crohn disease: long-term follow-up. Blood. 2010 Dec 23;116(26):6123–32. [PubMed: 20837778]
660.
Christadoss P, Poussin M, Deng C. Animal models of myasthenia gravis. Clin Immunol. 2000 Feb;94(2):75–87. [PubMed: 10637092]
661.
Scadding GK, Havard CW. Pathogenesis and treatment of myasthenia gravis. Br Med J (Clin Res Ed). 1981 Oct 17;283(6298):1008–12. [PMC free article: PMC1507269] [PubMed: 6794741]
662.
Drachman DB, Adams RN, Hu R, et al. Rebooting the immune system with high-dose cyclophosphamide for treatment of refractory myasthenia gravis. Ann N Y Acad Sci. 2008;1132:305–14. [PMC free article: PMC3390145] [PubMed: 18567882]
663.
Miteva L, Pramatarov K, Vassileva S. Calcinosis cutis in childhood systemic lupus erythematosus. J Eur Acad Dermatol Venereol. 2003 Sep;17(5):611–2. [PubMed: 12941115]
664.
Yun SJ, Lee JB, Kim SJ, et al. Calcinosis cutis universalis with joint contractures complicating juvenile dermatomyositis. Dermatology. 2006;212(4):401–3. [PubMed: 16707900]
665.
Maddison PJ. Overlap syndromes and mixed connective tissue disease. Curr Opin Rheumatol. 1991 Dec;3(6):995–1000. [PubMed: 1772755]
666.
Kim P, Grossman JM. Treatment of mixed connective tissue disease. Rheum Dis Clin North Am. 2005 Aug;31(3):549–65. viii. [PubMed: 16084325]
667.
Strober J, Cowan MJ, Horn BN. Allogeneic hematopoietic cell transplantation for refractory myasthenia gravis. Arch Neurol. 2009 May;66(5):659–61. [PubMed: 19433668]
668.
Jones OY, Good RA, Cahill RA. Nonmyeloablative allogeneic bone marrow transplantation for treatment of childhood overlap syndrome and small vessel vasculitis. Bone Marrow Transplant. 2004 May;33(10):1061–3. [PubMed: 15048144]
669.
Elhasid R, Rowe JM, Berkowitz D, et al. Disappearance of diffuse calcinosis following autologous stem cell transplantation in a child with autoimmune disease. Bone Marrow Transplant. 2004 Jun;33(12):1257–9. 2004. [PubMed: 15077134]
670.
Evans ER. Diagnosis of the hemolytic anemias. Calif Med. 1951 Oct;75(4):271–5. [PMC free article: PMC1520966] [PubMed: 14879272]
671.
Savasan S, Warrier I, Ravindranath Y. The spectrum of Evans' syndrome. Arch Dis Child. 1997 Sep;77(3):245–8. [PMC free article: PMC1717303] [PubMed: 9370906]
672.
Wang WC. Evans syndrome in childhood: pathophysiology, clinical course, and treatment. Am J Pediatr Hematol Oncol. 1988 Winter;10(4):330–8. [PubMed: 3071168]
673.
Teachey DT, Manno CS, Axsom KM, et al. Unmasking Evans syndrome: T-cell phenotype and apoptotic response reveal autoimmune lymphoproliferative syndrome (ALPS). Blood. 2005 Mar 15;105(6):2443–8. [PubMed: 15542578]
674.
Mathew P, Chen G, Wang W. Evans syndrome: results of a national survey. J Pediatr Hematol Oncol. 1997 Sep-Oct;19(5):433–7. [PubMed: 9329465]
675.
Norton A, Roberts I. Management of Evans syndrome. Br J Haematol. 2006 Jan;132(2):125–37. [PubMed: 16398647]
676.
Sokol RJ, Hewitt S, Stamps BK. Autoimmune haemolysis: an 18-year study of 865 cases referred to a regional transfusion centre. Br Med J (Clin Res Ed). 1981 Jun 20;282(6281):2023–7. [PMC free article: PMC1505955] [PubMed: 6788179]
677.
McMillan R. The pathogenesis of chronic immune (idiopathic) thrombocytopenic purpura. Semin Hematol. 2000 Jan;37(1 Suppl 1):5–9. [PubMed: 10676917]
678.
George JN, el-Harake MA, Raskob GE. Chronic idiopathic thrombocytopenic purpura. N Engl J Med. 1994 Nov 3;331(18):1207–11. [PubMed: 7935660]
679.
Daikeler T, Hugle T, Farge D, et al. Allogeneic hematopoietic SCT for patients with autoimmune diseases. Bone Marrow Transplant. 2009 Jul;44(1):27–33. [PubMed: 19139739]
680.
Connor P, Veys P, Amrolia P, et al. Pulmonary hypertension in children with Evans syndrome. Pediatr Hematol Oncol. 2008 Mar;25(2):93–8. [PubMed: 18363174]
681.
Urban C, Lackner H, Sovinz P, et al. Successful unrelated cord blood transplantation in a 7-year-old boy with Evans syndrome refractory to immunosuppression and double autologous stem cell transplantation. Eur J Haematol. 2006 Jun;76(6):526–30. [PubMed: 16529601]
682.
Raetz E, Beatty PG, Adams RH. Treatment of severe Evans syndrome with an allogeneic cord blood transplant. Bone Marrow Transplant. 1997 Sep;20(5):427–9. [PubMed: 9339762]
683.
Paillard C, Kanold J, Halle P, et al. Two-step immunoablative treatment with autologous peripheral blood CD34(+) cell transplantation in an 8-year-old boy with autoimmune haemolytic anaemia. Br J Haematol. 2000 Sep;110(4):900–2. [PubMed: 11054079]
684.
De Stefano P, Zecca M, Giorgiani G, et al. Resolution of immune haemolytic anaemia with allogeneic bone marrow transplantation after an unsuccessful autograft. Br J Haematol. 1999 Sep;106(4):1063–4. [PubMed: 10520015]
685.
Huhn RD, Fogarty PF, Nakamura R, et al. High-dose cyclophosphamide with autologous lymphocyte-depleted peripheral blood stem cell (PBSC) support for treatment of refractory chronic autoimmune thrombocytopenia. Blood. 2003 Jan 1;101(1):71–7. [PubMed: 12393623]
686.
Altman DG. Systematic reviews of evaluations of prognostic variables. BMJ. 2001 Jul 28;323(7306):224–8. [PMC free article: PMC1120839] [PubMed: 11473921]
687.
Altman DG, Lyman GH. Methodological challenges in the evaluation of prognostic factors in breast cancer. Breast Cancer Res Treat. 1998;52(1-3):289–303. [PubMed: 10066088]
688.
Altman DG, Riley RD. Primer: an evidence-based approach to prognostic markers. Nat Clin Pract Oncol. 2005 Sep;2(9):466–72. [PubMed: 16265015]
689.
Brocklehurst P, French R. The association between maternal HIV infection and perinatal outcome: a systematic review of the literature and meta-analysis. Br J Obstet Gynaecol. 1998 Aug;105(8):836–48. [PubMed: 9746375]
690.
Gould Rothberg BE, Bracken MB. E-cadherin immunohistochemical expression as a prognostic factor in infiltrating ductal carcinoma of the breast: a systematic review and meta-analysis. Breast Cancer Res Treat. 2006 Nov;100(2):139–48. [PubMed: 16791476]
691.
McShane LM, Altman DG, Sauerbrei W, et al. Reporting recommendations for tumor marker prognostic studies (REMARK). J Natl Cancer Inst. 2005 Aug 17;97(16):1180–4. [PubMed: 16106022]
692.
Simon R, Altman DG. Statistical aspects of prognostic factor studies in oncology. Br J Cancer. 1994 Jun;69(6):979–85. [PMC free article: PMC1969431] [PubMed: 8198989]
693.
Deeks JJ, Dinnes J, D'Amico R, et al. Evaluating non-randomised intervention studies. Health Technol Assess. 2003;7(27):iii–x. 1–173. [PubMed: 14499048]

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