Characteristics and Outcome of Patients With Refractory Orbital Rhabdomyosarcoma.

Abstract

Orbital rhabdomyosarcoma (oRMS) is a rare pediatric soft-tissue sarcoma with generally excellent outcomes; however, a subset of patients relapse, and evidence on optimal salvage strategies and long-term survival after recurrence remains limited. Pediatric patients with oRMS enrolled in the cooperative protocols RMS79, RMS88, RMS96, and RMS2005 between 1979 and 2016. Among 117 enrolled patients, 30 relapsed, predominantly with local recurrence. Relapse was associated with age < 1 year, female sex, RMS88 protocol, incomplete initial surgery, and omission of radiotherapy. Salvage treatments included multi-agent chemotherapy, radiotherapy, and surgery, with orbit exenteration performed in a total of 13 cases. After a median follow-up of 12.7 years from relapse, 10-year OS and EFS were 56.0% and 43.3%, respectively. Although oRMS usually has an excellent prognosis, relapse remains challenging. Multidisciplinary salvage therapy can yield long-term survival in over half of relapsed patients. Better initial treatment selection is needed to reduce relapse and late toxicity.