Characteristics and Outcome of Patients With Refractory Orbital Rhabdomyosarcoma.
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BACKGROUND: 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. METHODS: Pediatric patients with oRMS enrolled in the cooperative protocols RMS79, RMS88, RMS96, and RMS2005 between 1979 and 2016. RESULTS: 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. CONCLUSIONS: 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.