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Refractory Very High Risk Myelodysplastic Syndrome

MedGen UID:
1710710
•Concept ID:
C5237068
•
Neoplastic Process
Synonym: Refractory Very High-Risk Myelodysplastic Syndrome

Definition

Very high-risk myelodysplastic syndrome that is resistant to treatment. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVRefractory Very High Risk Myelodysplastic Syndrome

Professional guidelines

PubMed

Diagnosis and management of Aspergillus diseases: executive summary of the 2017 ESCMID-ECMM-ERS guideline.
Ullmann AJ, Aguado JM, Arikan-Akdagli S, Denning DW, Groll AH, Lagrou K, Lass-Flörl C, Lewis RE, Munoz P, Verweij PE, Warris A, Ader F, Akova M, Arendrup MC, Barnes RA, Beigelman-Aubry C, Blot S, Bouza E, Brüggemann RJM, Buchheidt D, Cadranel J, Castagnola E, Chakrabarti A, Cuenca-Estrella M, Dimopoulos G, Fortun J, Gangneux JP, Garbino J, Heinz WJ, Herbrecht R, Heussel CP, Kibbler CC, Klimko N, Kullberg BJ, Lange C, Lehrnbecher T, Löffler J, Lortholary O, Maertens J, Marchetti O, Meis JF, Pagano L, Ribaud P, Richardson M, Roilides E, Ruhnke M, Sanguinetti M, Sheppard DC, Sinkó J, Skiada A, Vehreschild MJGT, Viscoli C, Cornely OA
Clin Microbiol Infect 2018 May;24 Suppl 1:e1-e38. Epub 2018 Mar 12 doi: 10.1016/j.cmi.2018.01.002. PMID: 29544767
Primary myelofibrosis: 2013 update on diagnosis, risk-stratification, and management.
Tefferi A
Am J Hematol 2013 Feb;88(2):141-50. doi: 10.1002/ajh.23384. PMID: 23349007
Myelodysplastic syndromes: 2011 update on diagnosis, risk-stratification, and management.
Garcia-Manero G
Am J Hematol 2011 Jun;86(6):490-8. doi: 10.1002/ajh.22047. PMID: 21594886

Recent clinical studies

Etiology

Azacitidine plus venetoclax in patients with high-risk myelodysplastic syndromes or chronic myelomonocytic leukaemia: phase 1 results of a single-centre, dose-escalation, dose-expansion, phase 1-2 study.
Bazinet A, Darbaniyan F, Jabbour E, Montalban-Bravo G, Ohanian M, Chien K, Kadia T, Takahashi K, Masarova L, Short N, Alvarado Y, Yilmaz M, Ravandi F, Andreeff M, Kanagal-Shamanna R, Ganan-Gomez I, Colla S, Qiao W, Huang X, McCue D, Mirabella B, Kantarjian H, Garcia-Manero G
Lancet Haematol 2022 Oct;9(10):e756-e765. Epub 2022 Sep 2 doi: 10.1016/S2352-3026(22)00216-2. PMID: 36063832
How I treat warm autoimmune hemolytic anemia.
Barcellini W, Fattizzo B
Blood 2021 Mar 11;137(10):1283-1294. doi: 10.1182/blood.2019003808. PMID: 33512406
The Changing Landscape of Autoimmune Hemolytic Anemia.
Barcellini W, Fattizzo B
Front Immunol 2020;11:946. Epub 2020 Jun 3 doi: 10.3389/fimmu.2020.00946. PMID: 32655543Free PMC Article
Diagnosis and management of Aspergillus diseases: executive summary of the 2017 ESCMID-ECMM-ERS guideline.
Ullmann AJ, Aguado JM, Arikan-Akdagli S, Denning DW, Groll AH, Lagrou K, Lass-Flörl C, Lewis RE, Munoz P, Verweij PE, Warris A, Ader F, Akova M, Arendrup MC, Barnes RA, Beigelman-Aubry C, Blot S, Bouza E, Brüggemann RJM, Buchheidt D, Cadranel J, Castagnola E, Chakrabarti A, Cuenca-Estrella M, Dimopoulos G, Fortun J, Gangneux JP, Garbino J, Heinz WJ, Herbrecht R, Heussel CP, Kibbler CC, Klimko N, Kullberg BJ, Lange C, Lehrnbecher T, Löffler J, Lortholary O, Maertens J, Marchetti O, Meis JF, Pagano L, Ribaud P, Richardson M, Roilides E, Ruhnke M, Sanguinetti M, Sheppard DC, Sinkó J, Skiada A, Vehreschild MJGT, Viscoli C, Cornely OA
Clin Microbiol Infect 2018 May;24 Suppl 1:e1-e38. Epub 2018 Mar 12 doi: 10.1016/j.cmi.2018.01.002. PMID: 29544767
A Review of Chronic Granulomatous Disease.
Arnold DE, Heimall JR
Adv Ther 2017 Dec;34(12):2543-2557. Epub 2017 Nov 22 doi: 10.1007/s12325-017-0636-2. PMID: 29168144Free PMC Article

Diagnosis

How I treat warm autoimmune hemolytic anemia.
Barcellini W, Fattizzo B
Blood 2021 Mar 11;137(10):1283-1294. doi: 10.1182/blood.2019003808. PMID: 33512406
10-day decitabine with venetoclax for newly diagnosed intensive chemotherapy ineligible, and relapsed or refractory acute myeloid leukaemia: a single-centre, phase 2 trial.
DiNardo CD, Maiti A, Rausch CR, Pemmaraju N, Naqvi K, Daver NG, Kadia TM, Borthakur G, Ohanian M, Alvarado Y, Issa GC, Montalban-Bravo G, Short NJ, Yilmaz M, Bose P, Jabbour EJ, Takahashi K, Burger JA, Garcia-Manero G, Jain N, Kornblau SM, Thompson PA, Estrov Z, Masarova L, Sasaki K, Verstovsek S, Ferrajoli A, Weirda WG, Wang SA, Konoplev S, Chen Z, Pierce SA, Ning J, Qiao W, Ravandi F, Andreeff M, Welch JS, Kantarjian HM, Konopleva MY
Lancet Haematol 2020 Oct;7(10):e724-e736. Epub 2020 Sep 5 doi: 10.1016/S2352-3026(20)30210-6. PMID: 32896301Free PMC Article
The Changing Landscape of Autoimmune Hemolytic Anemia.
Barcellini W, Fattizzo B
Front Immunol 2020;11:946. Epub 2020 Jun 3 doi: 10.3389/fimmu.2020.00946. PMID: 32655543Free PMC Article
Diagnosis and management of Aspergillus diseases: executive summary of the 2017 ESCMID-ECMM-ERS guideline.
Ullmann AJ, Aguado JM, Arikan-Akdagli S, Denning DW, Groll AH, Lagrou K, Lass-Flörl C, Lewis RE, Munoz P, Verweij PE, Warris A, Ader F, Akova M, Arendrup MC, Barnes RA, Beigelman-Aubry C, Blot S, Bouza E, Brüggemann RJM, Buchheidt D, Cadranel J, Castagnola E, Chakrabarti A, Cuenca-Estrella M, Dimopoulos G, Fortun J, Gangneux JP, Garbino J, Heinz WJ, Herbrecht R, Heussel CP, Kibbler CC, Klimko N, Kullberg BJ, Lange C, Lehrnbecher T, Löffler J, Lortholary O, Maertens J, Marchetti O, Meis JF, Pagano L, Ribaud P, Richardson M, Roilides E, Ruhnke M, Sanguinetti M, Sheppard DC, Sinkó J, Skiada A, Vehreschild MJGT, Viscoli C, Cornely OA
Clin Microbiol Infect 2018 May;24 Suppl 1:e1-e38. Epub 2018 Mar 12 doi: 10.1016/j.cmi.2018.01.002. PMID: 29544767
A Review of Chronic Granulomatous Disease.
Arnold DE, Heimall JR
Adv Ther 2017 Dec;34(12):2543-2557. Epub 2017 Nov 22 doi: 10.1007/s12325-017-0636-2. PMID: 29168144Free PMC Article

Therapy

Olutasidenib alone or with azacitidine in IDH1-mutated acute myeloid leukaemia and myelodysplastic syndrome: phase 1 results of a phase 1/2 trial.
Watts JM, Baer MR, Yang J, Prebet T, Lee S, Schiller GJ, Dinner SN, Pigneux A, Montesinos P, Wang ES, Seiter KP, Wei AH, De Botton S, Arnan M, Donnellan W, Schwarer AP, Récher C, Jonas BA, Ferrell PB Jr, Marzac C, Kelly P, Sweeney J, Forsyth S, Guichard SM, Brevard J, Henrick P, Mohamed H, Cortes JE
Lancet Haematol 2023 Jan;10(1):e46-e58. Epub 2022 Nov 10 doi: 10.1016/S2352-3026(22)00292-7. PMID: 36370742
Azacitidine plus venetoclax in patients with high-risk myelodysplastic syndromes or chronic myelomonocytic leukaemia: phase 1 results of a single-centre, dose-escalation, dose-expansion, phase 1-2 study.
Bazinet A, Darbaniyan F, Jabbour E, Montalban-Bravo G, Ohanian M, Chien K, Kadia T, Takahashi K, Masarova L, Short N, Alvarado Y, Yilmaz M, Ravandi F, Andreeff M, Kanagal-Shamanna R, Ganan-Gomez I, Colla S, Qiao W, Huang X, McCue D, Mirabella B, Kantarjian H, Garcia-Manero G
Lancet Haematol 2022 Oct;9(10):e756-e765. Epub 2022 Sep 2 doi: 10.1016/S2352-3026(22)00216-2. PMID: 36063832
How I treat warm autoimmune hemolytic anemia.
Barcellini W, Fattizzo B
Blood 2021 Mar 11;137(10):1283-1294. doi: 10.1182/blood.2019003808. PMID: 33512406
Decitabine/Cedazuridine: First Approval.
Dhillon S
Drugs 2020 Sep;80(13):1373-1378. doi: 10.1007/s40265-020-01389-7. PMID: 32860582Free PMC Article
Diagnosis and management of Aspergillus diseases: executive summary of the 2017 ESCMID-ECMM-ERS guideline.
Ullmann AJ, Aguado JM, Arikan-Akdagli S, Denning DW, Groll AH, Lagrou K, Lass-Flörl C, Lewis RE, Munoz P, Verweij PE, Warris A, Ader F, Akova M, Arendrup MC, Barnes RA, Beigelman-Aubry C, Blot S, Bouza E, Brüggemann RJM, Buchheidt D, Cadranel J, Castagnola E, Chakrabarti A, Cuenca-Estrella M, Dimopoulos G, Fortun J, Gangneux JP, Garbino J, Heinz WJ, Herbrecht R, Heussel CP, Kibbler CC, Klimko N, Kullberg BJ, Lange C, Lehrnbecher T, Löffler J, Lortholary O, Maertens J, Marchetti O, Meis JF, Pagano L, Ribaud P, Richardson M, Roilides E, Ruhnke M, Sanguinetti M, Sheppard DC, Sinkó J, Skiada A, Vehreschild MJGT, Viscoli C, Cornely OA
Clin Microbiol Infect 2018 May;24 Suppl 1:e1-e38. Epub 2018 Mar 12 doi: 10.1016/j.cmi.2018.01.002. PMID: 29544767

Prognosis

Myelodysplastic Syndromes: A New Decade.
Volpe VO, Garcia-Manero G, Komrokji RS
Clin Lymphoma Myeloma Leuk 2022 Jan;22(1):1-16. Epub 2021 Aug 2 doi: 10.1016/j.clml.2021.07.031. PMID: 34544674
Summary of animal models of myelodysplastic syndrome.
Li W, Li M, Yang X, Zhang W, Cao L, Gao R
Animal Model Exp Med 2021 Mar;4(1):71-76. Epub 2021 Feb 3 doi: 10.1002/ame2.12144. PMID: 33738439Free PMC Article
How I treat warm autoimmune hemolytic anemia.
Barcellini W, Fattizzo B
Blood 2021 Mar 11;137(10):1283-1294. doi: 10.1182/blood.2019003808. PMID: 33512406
Decitabine/Cedazuridine: First Approval.
Dhillon S
Drugs 2020 Sep;80(13):1373-1378. doi: 10.1007/s40265-020-01389-7. PMID: 32860582Free PMC Article
The Changing Landscape of Autoimmune Hemolytic Anemia.
Barcellini W, Fattizzo B
Front Immunol 2020;11:946. Epub 2020 Jun 3 doi: 10.3389/fimmu.2020.00946. PMID: 32655543Free PMC Article

Clinical prediction guides

Long-Term Efficacy and Safety of Luspatercept for Anemia Treatment in Patients With Lower-Risk Myelodysplastic Syndromes: The Phase II PACE-MDS Study.
Platzbecker U, Götze KS, Kiewe P, Germing U, Mayer K, Radsak M, Wolff T, Chromik J, Sockel K, Oelschlägel U, Haase D, Illmer T, Al-Ali HK, Silling G, Reynolds JG, Zhang X, Attie KM, Shetty JK, Giagounidis A
J Clin Oncol 2022 Nov 20;40(33):3800-3807. Epub 2022 Aug 23 doi: 10.1200/JCO.21.02476. PMID: 35998303Free PMC Article
Myelodysplastic Syndromes: A New Decade.
Volpe VO, Garcia-Manero G, Komrokji RS
Clin Lymphoma Myeloma Leuk 2022 Jan;22(1):1-16. Epub 2021 Aug 2 doi: 10.1016/j.clml.2021.07.031. PMID: 34544674
Imetelstat Achieves Meaningful and Durable Transfusion Independence in High Transfusion-Burden Patients With Lower-Risk Myelodysplastic Syndromes in a Phase II Study.
Steensma DP, Fenaux P, Van Eygen K, Raza A, Santini V, Germing U, Font P, Diez-Campelo M, Thepot S, Vellenga E, Patnaik MM, Jang JH, Varsos H, Bussolari J, Rose E, Sherman L, Sun L, Wan Y, Dougherty S, Huang F, Feller F, Rizo A, Platzbecker U
J Clin Oncol 2021 Jan 1;39(1):48-56. Epub 2020 Oct 27 doi: 10.1200/JCO.20.01895. PMID: 33108243
Decitabine/Cedazuridine: First Approval.
Dhillon S
Drugs 2020 Sep;80(13):1373-1378. doi: 10.1007/s40265-020-01389-7. PMID: 32860582Free PMC Article
The Changing Landscape of Autoimmune Hemolytic Anemia.
Barcellini W, Fattizzo B
Front Immunol 2020;11:946. Epub 2020 Jun 3 doi: 10.3389/fimmu.2020.00946. PMID: 32655543Free PMC Article

Recent systematic reviews

Effects of all-trans retinoic acid (ATRA) in addition to chemotherapy for adults with acute myeloid leukaemia (AML) (non-acute promyelocytic leukaemia (non-APL)).
Küley-Bagheri Y, Kreuzer KA, Monsef I, Lübbert M, Skoetz N
Cochrane Database Syst Rev 2018 Aug 6;8(8):CD011960. doi: 10.1002/14651858.CD011960.pub2. PMID: 30080246Free PMC Article

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