Metts JL, Aye JM, Crane JN, Oberoi S, Balis FM, Bhatia S, Bona K, Carleton B, Dasgupta R, Dela Cruz FS, Greenzang KA, Kaufman JL, Linardic CM, Parsons SK, Robertson-Tessi M, Rudzinski ER, Soragni A, Stewart E, Weigel BJ, Wolden SL, Weiss AR, Venkatramani R, Heske CM. Roadmap for the next generation of Children’s Oncology Group rhabdomyosarcoma trials. Cancer. 2024 Nov 15;130(22):3785-3796. doi: 10.1002/cncr.35457. Epub 2024 Jun 28. PubMed PMID: 38941509; PubMed Central PMCID: PMC11511643.
Study ID Citation
Abstract
Clinical trials conducted by the Intergroup Rhabdomyosarcoma (RMS) Study Group (IRSG) and Children’s Oncology Group (COG) have been pivotal to establishing current standards for diagnosis and therapy for RMS. Recent advancements in understanding the biology and clinical behavior of RMS have led to more nuanced approaches to diagnosis, risk stratification, and treatment. The complexities introduced by these advancements, coupled with the rarity of RMS, pose challenges to conducting large-scale phase 3 clinical trials to evaluate new treatment strategies for RMS. Given these challenges, systematic planning of future clinical trials in RMS is paramount to address pertinent questions regarding the therapeutic efficacy of drugs, biomarkers of response, treatment-related toxicity, and patient quality of life. Herein, we outline the proposed strategic approach of the COG Soft Tissue Sarcoma (STS) Committee to the next generation of RMS clinical trials, focusing on five themes: improved novel agent identification and preclinical to clinical translation, more efficient trial development and implementation, expanded opportunities for knowledge generation during trials, therapeutic toxicity reduction and quality of life, and patient engagement.