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Refractory Rhabdomyosarcoma

MedGen UID:
1645148
•Concept ID:
C4684864
•
Neoplastic Process

Definition

Rhabdomyosarcoma that does not respond to treatment. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVRefractory Rhabdomyosarcoma

Professional guidelines

PubMed

Ifosfamide and Etoposide Chemotherapy in the Treatment of Recurrent/Refractory Rhabdomyosarcoma in Adults.
Sasada S, Kodaira M, Shimoi T, Shimomura A, Yunokawa M, Yonemori K, Shimizu C, Fujiwara Y, Tamura K
Anticancer Res 2016 May;36(5):2429-32. PMID: 27127153

Recent clinical studies

Etiology

Addition of temsirolimus to chemotherapy in children, adolescents, and young adults with intermediate-risk rhabdomyosarcoma (ARST1431): a randomised, open-label, phase 3 trial from the Children's Oncology Group.
Gupta AA, Xue W, Harrison DJ, Hawkins DS, Dasgupta R, Wolden S, Shulkin B, Qumseya A, Routh JC, MacDonald T, Feinberg S, Crompton B, Rudzinski ER, Arnold M, Venkatramani R
Lancet Oncol 2024 Jul;25(7):912-921. doi: 10.1016/S1470-2045(24)00255-9. PMID: 38936378
Event-free survival in relapsed and refractory rhabdomyosarcoma treated on cooperative group phase II trials: A report from the Children's Oncology Group.
Metts J, Xue W, Gao Z, Oberoi S, Weiss AR, Venkatramani R, Harrison DJ
Pediatr Blood Cancer 2024 Jul;71(7):e31009. Epub 2024 Apr 16 doi: 10.1002/pbc.31009. PMID: 38627882Free PMC Article
A systematic review of combined surgery and brachytherapy approaches for children and young people with relapsed and refractory rhabdomyosarcoma (Local-REFoRMS).
Ballantyne E, Evans C, Shepherd L, Fulbright H, Wakeling S, Phillips B, Morgan JE
Pediatr Blood Cancer 2024 Jun;71(6):e30952. Epub 2024 Apr 2 doi: 10.1002/pbc.30952. PMID: 38566349
Randomized Phase II Trial of Vincristine-Irinotecan With or Without Temozolomide, in Children and Adults With Relapsed or Refractory Rhabdomyosarcoma: A European Paediatric Soft Tissue Sarcoma Study Group and Innovative Therapies for Children With Cancer Trial.
Defachelles AS, Bogart E, Casanova M, Merks JHM, Bisogno G, Calareso G, Gallego Melcon S, Gatz SA, Le Deley MC, McHugh K, Probst A, Rocourt N, van Rijn RR, Wheatley K, Minard-Colin V, Chisholm JC
J Clin Oncol 2021 Sep 20;39(27):2979-2990. Epub 2021 Aug 3 doi: 10.1200/JCO.21.00124. PMID: 34343032
Phase 2 trial of sorafenib in children and young adults with refractory solid tumors: A report from the Children's Oncology Group.
Kim A, Widemann BC, Krailo M, Jayaprakash N, Fox E, Weigel B, Blaney SM
Pediatr Blood Cancer 2015 Sep;62(9):1562-6. Epub 2015 Apr 27 doi: 10.1002/pbc.25548. PMID: 26207356Free PMC Article

Diagnosis

A phase 2 trial of R1507, a monoclonal antibody to the insulin-like growth factor-1 receptor (IGF-1R), in patients with recurrent or refractory rhabdomyosarcoma, osteosarcoma, synovial sarcoma, and other soft tissue sarcomas: results of a Sarcoma Alliance for Research Through Collaboration study.
Pappo AS, Vassal G, Crowley JJ, Bolejack V, Hogendoorn PC, Chugh R, Ladanyi M, Grippo JF, Dall G, Staddon AP, Chawla SP, Maki RG, Araujo DM, Geoerger B, Ganjoo K, Marina N, Blay JY, Schuetze SM, Chow WA, Helman LJ
Cancer 2014 Aug 15;120(16):2448-56. Epub 2014 May 2 doi: 10.1002/cncr.28728. PMID: 24797726Free PMC Article

Therapy

Addition of temsirolimus to chemotherapy in children, adolescents, and young adults with intermediate-risk rhabdomyosarcoma (ARST1431): a randomised, open-label, phase 3 trial from the Children's Oncology Group.
Gupta AA, Xue W, Harrison DJ, Hawkins DS, Dasgupta R, Wolden S, Shulkin B, Qumseya A, Routh JC, MacDonald T, Feinberg S, Crompton B, Rudzinski ER, Arnold M, Venkatramani R
Lancet Oncol 2024 Jul;25(7):912-921. doi: 10.1016/S1470-2045(24)00255-9. PMID: 38936378
Event-free survival in relapsed and refractory rhabdomyosarcoma treated on cooperative group phase II trials: A report from the Children's Oncology Group.
Metts J, Xue W, Gao Z, Oberoi S, Weiss AR, Venkatramani R, Harrison DJ
Pediatr Blood Cancer 2024 Jul;71(7):e31009. Epub 2024 Apr 16 doi: 10.1002/pbc.31009. PMID: 38627882Free PMC Article
Randomized Phase II Trial of Vincristine-Irinotecan With or Without Temozolomide, in Children and Adults With Relapsed or Refractory Rhabdomyosarcoma: A European Paediatric Soft Tissue Sarcoma Study Group and Innovative Therapies for Children With Cancer Trial.
Defachelles AS, Bogart E, Casanova M, Merks JHM, Bisogno G, Calareso G, Gallego Melcon S, Gatz SA, Le Deley MC, McHugh K, Probst A, Rocourt N, van Rijn RR, Wheatley K, Minard-Colin V, Chisholm JC
J Clin Oncol 2021 Sep 20;39(27):2979-2990. Epub 2021 Aug 3 doi: 10.1200/JCO.21.00124. PMID: 34343032
Ifosfamide and Etoposide Chemotherapy in the Treatment of Recurrent/Refractory Rhabdomyosarcoma in Adults.
Sasada S, Kodaira M, Shimoi T, Shimomura A, Yunokawa M, Yonemori K, Shimizu C, Fujiwara Y, Tamura K
Anticancer Res 2016 May;36(5):2429-32. PMID: 27127153
Efficacy of topotecan plus vincristine and doxorubicin in children with recurrent/refractory rhabdomyosarcoma.
Meazza C, Casanova M, Zaffignani E, Luksch R, Podda M, Favini F, Catania S, Biassoni V, Morosi C, Ferrari A
Med Oncol 2009;26(1):67-72. Epub 2008 Aug 5 doi: 10.1007/s12032-008-9085-8. PMID: 18679836

Prognosis

Event-free survival in relapsed and refractory rhabdomyosarcoma treated on cooperative group phase II trials: A report from the Children's Oncology Group.
Metts J, Xue W, Gao Z, Oberoi S, Weiss AR, Venkatramani R, Harrison DJ
Pediatr Blood Cancer 2024 Jul;71(7):e31009. Epub 2024 Apr 16 doi: 10.1002/pbc.31009. PMID: 38627882Free PMC Article
A systematic review of early phase studies for children and young people with relapsed and refractory rhabdomyosarcoma: The REFoRMS-SR project.
Evans C, Shepherd L, Bryan G, Fulbright H, Crowther S, Wakeling S, Stewart A, Stewart C, Chisholm J, Gibson F, Phillips B, Morgan JE
Int J Cancer 2024 Apr 1;154(7):1235-1260. Epub 2023 Dec 10 doi: 10.1002/ijc.34808. PMID: 38071594
A phase 2 trial of R1507, a monoclonal antibody to the insulin-like growth factor-1 receptor (IGF-1R), in patients with recurrent or refractory rhabdomyosarcoma, osteosarcoma, synovial sarcoma, and other soft tissue sarcomas: results of a Sarcoma Alliance for Research Through Collaboration study.
Pappo AS, Vassal G, Crowley JJ, Bolejack V, Hogendoorn PC, Chugh R, Ladanyi M, Grippo JF, Dall G, Staddon AP, Chawla SP, Maki RG, Araujo DM, Geoerger B, Ganjoo K, Marina N, Blay JY, Schuetze SM, Chow WA, Helman LJ
Cancer 2014 Aug 15;120(16):2448-56. Epub 2014 May 2 doi: 10.1002/cncr.28728. PMID: 24797726Free PMC Article
TRAIL-induced cytotoxicity in a melphalan-resistant rhabdomyosarcoma cell line via activation of caspase-2.
Klüttermann K, Banning U, Kachel M, Krause C, Körholz D, Mauz-Körholz C
Anticancer Res 2006 Jan-Feb;26(1A):351-6. PMID: 16475717
Regression of refractory rhabdomyosarcoma after allogeneic stem-cell transplantation.
Misawa A, Hosoi H, Tsuchiya K, Iehara T, Sawada T, Sugimoto T
Pediatr Hematol Oncol 2003 Mar;20(2):151-5. doi: 10.1080/0880010390158658. PMID: 12554526

Clinical prediction guides

Addition of temsirolimus to chemotherapy in children, adolescents, and young adults with intermediate-risk rhabdomyosarcoma (ARST1431): a randomised, open-label, phase 3 trial from the Children's Oncology Group.
Gupta AA, Xue W, Harrison DJ, Hawkins DS, Dasgupta R, Wolden S, Shulkin B, Qumseya A, Routh JC, MacDonald T, Feinberg S, Crompton B, Rudzinski ER, Arnold M, Venkatramani R
Lancet Oncol 2024 Jul;25(7):912-921. doi: 10.1016/S1470-2045(24)00255-9. PMID: 38936378
Phase 2 trial of sorafenib in children and young adults with refractory solid tumors: A report from the Children's Oncology Group.
Kim A, Widemann BC, Krailo M, Jayaprakash N, Fox E, Weigel B, Blaney SM
Pediatr Blood Cancer 2015 Sep;62(9):1562-6. Epub 2015 Apr 27 doi: 10.1002/pbc.25548. PMID: 26207356Free PMC Article
A phase 2 trial of R1507, a monoclonal antibody to the insulin-like growth factor-1 receptor (IGF-1R), in patients with recurrent or refractory rhabdomyosarcoma, osteosarcoma, synovial sarcoma, and other soft tissue sarcomas: results of a Sarcoma Alliance for Research Through Collaboration study.
Pappo AS, Vassal G, Crowley JJ, Bolejack V, Hogendoorn PC, Chugh R, Ladanyi M, Grippo JF, Dall G, Staddon AP, Chawla SP, Maki RG, Araujo DM, Geoerger B, Ganjoo K, Marina N, Blay JY, Schuetze SM, Chow WA, Helman LJ
Cancer 2014 Aug 15;120(16):2448-56. Epub 2014 May 2 doi: 10.1002/cncr.28728. PMID: 24797726Free PMC Article
Aberrant CDK4 amplification in refractory rhabdomyosarcoma as identified by genomic profiling.
Park S, Lee J, Do IG, Jang J, Rho K, Ahn S, Maruja L, Kim SJ, Kim KM, Mao M, Oh E, Kim YJ, Kim J, Choi YL
Sci Rep 2014 Jan 10;4:3623. doi: 10.1038/srep03623. PMID: 24406431Free PMC Article
Regression of refractory rhabdomyosarcoma after allogeneic stem-cell transplantation.
Misawa A, Hosoi H, Tsuchiya K, Iehara T, Sawada T, Sugimoto T
Pediatr Hematol Oncol 2003 Mar;20(2):151-5. doi: 10.1080/0880010390158658. PMID: 12554526

Recent systematic reviews

A systematic review of combined surgery and brachytherapy approaches for children and young people with relapsed and refractory rhabdomyosarcoma (Local-REFoRMS).
Ballantyne E, Evans C, Shepherd L, Fulbright H, Wakeling S, Phillips B, Morgan JE
Pediatr Blood Cancer 2024 Jun;71(6):e30952. Epub 2024 Apr 2 doi: 10.1002/pbc.30952. PMID: 38566349
A systematic review of early phase studies for children and young people with relapsed and refractory rhabdomyosarcoma: The REFoRMS-SR project.
Evans C, Shepherd L, Bryan G, Fulbright H, Crowther S, Wakeling S, Stewart A, Stewart C, Chisholm J, Gibson F, Phillips B, Morgan JE
Int J Cancer 2024 Apr 1;154(7):1235-1260. Epub 2023 Dec 10 doi: 10.1002/ijc.34808. PMID: 38071594

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