Suboptimal outcome for patients with biliary rhabdomyosarcoma treated on low-risk clinical trials: A report from the Children’s Oncology Group

Aye JM, Xue W, Palmer JD, Walterhouse DO, Arnold MA, Heaton TE, Venkatramani R. Suboptimal outcome for patients with biliary rhabdomyosarcoma treated on low-risk clinical trials: A report from the Children’s Oncology Group. Pediatr Blood Cancer. 2021 Apr;68(4):e28914. doi: 10.1002/pbc.28914. Epub 2021 Jan 26. PubMed PMID: 33501771; PubMed Central PMCID: PMC8765674.

Biliary rhabdomyosarcoma (RMS) is the most common biliary tumor in children. The biliary tract is classified as a favorable primary site. Therefore, patients with localized biliary RMS were included in two consecutive low-risk studies, D9602 and ARST0331, by the Children’s Oncology Group (COG). The outcome for these patients treated with low-risk therapy has not been reported. Patients with biliary RMS enrolled on COG low-risk trials D9602 or ARST0331 were analyzed. All patients received systemic chemotherapy and those with Group II (microscopic residual) or Group III (macroscopic residual) disease received 36–50.4 Gy adjuvant radiotherapy (RT). Delayed primary excision (DPE) was allowed on both studies.