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. 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. Lancet Oncol. 2024 Jul;25(7):912-921. doi: 10.1016/S1470-2045(24)00255-9. PubMed PMID: 38936378; PubMed Central PMCID: PMC11550893.

The Children’s Oncology Group defines intermediate-risk rhabdomyosarcoma (IR-RMS) as patients who have unresected FOXO1 fusion negative disease arising at an unfavorable site or those with non-metastatic FOXO1 fusion positive disease. Temsirolimus (TEM) in combination with chemotherapy has shown promising activity in patients with relapsed and refractory RMS. The primary objective of this phase 3 trial was to compare the event-free survival (EFS) of patients with IR-RMS treated with vincristine, actinomycin, cyclophosphamide alternating with vincristine and irinotecan (VAC-VI) combined with TEM followed by maintenance therapy with vinorelbine plus low dose cyclophosphamide versus VAC-VI alone with maintenance.