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Cancer Health Center

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Childhood Rhabdomyosarcoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Recurrent Childhood Rhabdomyosarcoma


The following standard chemotherapy regimens have been used to treat recurrent rhabdomyosarcoma:

  • Carboplatin/etoposide.[10]
  • Ifosfamide, carboplatin, and etoposide.[11,12]
  • Cyclophosphamide/topotecan.[13]
  • Irinotecan with or without vincristine.[14,15,16,17] A Children's Oncology Group (COG) prospective, randomized, up-front window trial, COG-ARST0121, showed no difference between vincristine plus irinotecan (20 mg/m2 /d) daily × 5 days for 4 weeks per 6-week treatment cycle (Regimen 1A) and irinotecan (50 mg/m2 /d) daily × 5 days for 2 weeks per 6-week treatment cycle (Regimen 1B) in poor-risk patients with relapsed or progressive rhabdomyosarcoma. At 1 year after initiation of treatment for recurrence, the failure-free survival (FFS) rate was 37% and the overall survival rate (OS) was 55% for Regimen 1A; the FFS rate was 38% and OS rate was 60% for Regimen 1B. The Soft Tissue Sarcoma Committee of the COG recommended the more convenient Regimen 1B for further investigation.[18][Level of evidence: 1iiA]
  • Single-agent vinorelbine. In one phase II trial, four of eleven patients with recurrent rhabdomyosarcoma responded to single-agent vinorelbine.[19] In another trial, 6 of 12 young patients (aged 9-29 years) had a partial response.[20]
  • Vinorelbine and cyclophosphamide. In a pilot study, three of nine patients with rhabdomyosarcoma had an objective response.[21] In a phase II study in France (N = 50), children with recurrent or refractory rhabdomyosarcoma were treated with vinorelbine and low-dose oral cyclophosphamide. Four complete responses and 14 partial responses were observed, for an objective response rate of 36%.[22][Level of evidence: 3iiiDiv]
  • Gemcitabine and docetaxel. In a single institution trial, two patients (N = 5) with recurrent rhabdomyosarcoma achieved an objective response.[23]
  • Rapamycin.[24]
  • Topotecan, vincristine, and doxorubicin.[25][Level of evidence: 3iiiDiv]
  • Vincristine, irinotecan, and temozolomide. One of four patients with recurrent alveolar rhabdomyosarcoma had a complete radiographic response sustained for 27 weeks with no grade 3 or 4 toxicities.[26]; [27][Level of evidence: 3iiiDiii]

Treatment options under clinical evaluation for recurrent rhabdomyosarcoma:

The following are examples of national and/or institutional clinical trials that are currently being conducted. Information about ongoing clinical trials is available from the NCI Web site.

  • Intensive chemotherapy followed by autologous bone marrow transplantation. Very intensive chemotherapy followed by autologous bone marrow reinfusion is also under investigation for patients with recurrent rhabdomyosarcoma. However, a review of the published data did not determine a significant benefit for patients who underwent this salvage treatment approach.[28,29,30]
  • New agents under clinical evaluation in phase I and phase II trials should be considered for relapsed patients.
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