Roadmap for the next generation of Children's Oncology Group rhabdomyosarcoma trials
Jonathan L. Metts, Sarcoma Department, Moffitt Cancer Center, Tampa, Florida, USA.
Jamie M. Aye, Division of Pediatric Hematology-Oncology, Department of Pediatrics, University of Alabama at Birmingham, Birmingham, Alabama, USA.
Jacquelyn N. Crane, Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Sapna Oberoi, Department of Pediatric Hematology/Oncology, Cancer Care Manitoba, Winnipeg, Manitoba, Canada.
Frank M. Balis, Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Smita Bhatia, Institute for Cancer Outcomes and Survivorship, Heersink School of Medicine, University of Alabama at Birmingham, Birmingham, Alabama, USA.
Kira Bona, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.
Bruce Carleton, Division of Translational Therapeutics, Department of Pediatrics, Faculty of Medicine, University of British Columbia, Vancouver, British Columbia, Canada.
Roshni Dasgupta, Division of Pediatric General and Thoracic Surgery, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA.
Filemon S. Dela Cruz, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
Katie A. Greenzang, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.
Jonathan L. Kaufman, Department of Hematology and Medical Oncology, Emory University, Atlanta, Georgia, USA.
Corinne M. Linardic, Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA.
Susan K. Parsons, Institute for Clinical Research and Health Policy Studies and Division of Hematology/Oncology, Tufts Medical Center, Boston, Massachusetts, USA.
Mark Robertson-Tessi, Integrated Mathematical Oncology Department, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida, USA.
Erin R. Rudzinski, Department of Laboratory Medicine and Pathology, Seattle Children's Hospital and University of Washington Medical Center, Seattle, Washington, USA.
Alice Soragni, Department of Orthopedic Surgery, University of California Los Angeles, Los Angeles, CA, USA.
Elizabeth Stewart, Department of Oncology, St Jude Children's Research Hospital, Memphis, Tennessee, USA.
Brenda J. Weigel, Division of Pediatric Hematology Oncology, University of Minnesota, Minneapolis, Minnesota, USA.
Suzanne L. Wolden, Department of Radiation Oncology, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
Aaron R. Weiss, Department of Pediatrics, Maine Medical Center, Portland, Maine, USA.
Rajkumar Venkatramani, Texas Children's Cancer Center, Baylor College of Medicine, Houston, Texas, USA.
Christine M. Heske, Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, Maryland, USA.
Abstract
Clinical trials conducted by the Intergroup Rhabdomyosarcoma (RMS) Study Group and the Children's Oncology Group 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, the authors outline the proposed strategic approach of the Children's Oncology Group Soft Tissue Sarcoma 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.
