Although combination therapies have improved outcomes in rhabdomyosarcoma (RMS) patients, prognosis remains poor for those who relapse, emphasizing a need for new targeted therapies. The multi-tyrosine kinase inhibitor (TKI) regorafenib targets several kinases associated with RMS, including VEGFR, FGFR, and PDGFR, and has demonstrated promising preclinical antitumor activity in Ewing sarcoma (EWS), neuroblastoma, and RMS models. In this phase I trial, Casanova and colleagues establish a maximum tolerated dose for regorafenib and evaluate the efficacy of regorafenib combined with vincristine and irinotecan in pediatric patients with recurrent/refractory RMS, EWS, neuroblastoma, and Wilms tumor. Treatment was well tolerated, with less than 20% of patients experiencing grade 3/4 adverse events. The authors report an overall response rate of 48% and a disease control rate of 86%, with 2 of 21 of patients achieving complete response, 8 patients demonstrating partial response, and 7 patients maintaining stable disease. Median progression-free survival was 7.0 months,...

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