An update on rhabdomyosarcoma risk stratification and the rationale for current and future Children's Oncology Group clinical trials

Josephine H Haduong; Christine M Heske; Wendy Allen-Rhoades; Wei Xue; Lisa A Teot; David A Rodeberg; Sarah S Donaldson; Aaron Weiss; Douglas S Hawkins; Rajkumar Venkatramani

doi:10.1002/pbc.29511

An update on rhabdomyosarcoma risk stratification and the rationale for current and future Children's Oncology Group clinical trials

Pediatr Blood Cancer. 2022 Apr;69(4):e29511. doi: 10.1002/pbc.29511. Epub 2022 Feb 7.

Authors

Affiliations

¹ Division of Oncology, Hyundai Cancer Institute, Children's Hospital Orange County, Orange, California, USA.
² Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, Maryland, USA.
³ Division of Pediatric Oncology, Mayo Clinic, Rochester, Minnesota, USA.
⁴ Department of Biostatistics, College of Public Health and Health Professions and College of Medicine, University of Florida, Gainesville, Florida, USA.
⁵ Department of Pathology, Boston Children's Hospital/Harvard Medical School, Boston, Massachusetts, USA.
⁶ Division of Pediatric Surgery, East Carolina University, Greenville, North Carolina, USA.
⁷ Department of Radiation Oncology, Stanford University, Stanford, California, USA.
⁸ Division of Pediatric Hematology-Oncology, Maine Medical Center, Portland, Maine, USA.
⁹ Division of Hematology/Oncology, Seattle Children's Hospital, University of Washington, Seattle, Washington, USA.
¹⁰ Department of Pediatrics, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.

Abstract

Children and adolescents with rhabdomyosarcoma (RMS) comprise a heterogeneous population with variable overall survival rates ranging between approximately 6% and 100% depending on defined risk factors. Although the risk stratification of patients has been refined across five decades of collaborative group studies, molecular prognostic biomarkers beyond FOXO1 fusion status have yet to be incorporated prospectively in upfront risk-based therapy assignments. This review describes the evolution of risk-based therapy and the current risk stratification, defines a new risk stratification incorporating novel biomarkers, and provides the rationale for the current and upcoming Children's Oncology Group RMS studies.

Keywords: MYOD1; TP53; rhabdomyosarcoma.

Publication types

Review
Research Support, N.I.H., Extramural

MeSH terms

Adolescent
Child
Gene Fusion
Humans
Rhabdomyosarcoma* / therapy
Rhabdomyosarcoma, Embryonal*
Risk Assessment
Risk Factors

Abstract

Publication types

MeSH terms

Grants and funding