Risk-based treatment for patients with first relapse or progression of rhabdomyosarcoma: A report from the Children’s Oncology Group

Mascarenhas L, Lyden ER, Breitfeld PP, Walterhouse DO, Donaldson SS, Rodeberg DA, Parham DM, Anderson JR, Meyer WH, Hawkins DS. Risk-based treatment for patients with first relapse or progression of rhabdomyosarcoma: A report from the Children’s Oncology Group. Cancer. 2019 Aug 1;125(15):2602-2609. doi: 10.1002/cncr.32122. Epub 2019 May 8. PMID: 31067356; PMCID: PMC7069123.

Patients with RMS and measurable disease at first relapse with unfavorable risk (UR) features were randomized to a six-week Phase 2 window with one of two treatment schedules of irinotecan with vincristine (VI) (previously reported). Those with at least a partial response to VI continued to receive 44 weeks of multi-agent chemotherapy including the assigned VI regimen. UR patients without measurable disease at study entry, without radiographic response after the VI window, or who declined VI window therapy received 31 weeks of multi-agent chemotherapy including tirapazamine (TPZ) at weeks 1, 4, 10, 19 and 28. Favorable risk (FR) patients received 31 weeks of the same multi-agent chemotherapy without VI and TPZ.