Characteristics and Outcome of Patients With Refractory Orbital Rhabdomyosarcoma

Area of Science:

• Pediatric Oncology
• Sarcoma Research
• Clinical Outcomes

Background:

• Orbital rhabdomyosarcoma (oRMS) is a rare pediatric soft-tissue sarcoma with generally good outcomes.
• A subset of patients experience relapse, necessitating effective salvage strategies.
• Limited evidence exists on optimal treatment and long-term survival post-recurrence.

Purpose of the Study:

• To analyze outcomes and identify factors associated with relapse in pediatric orbital rhabdomyosarcoma.
• To evaluate the effectiveness of salvage therapies for recurrent oRMS.
• To inform strategies for reducing relapse and late toxicities.

Main Methods:

• Retrospective analysis of pediatric patients with oRMS.
• Data collected from cooperative protocols (RMS79, RMS88, RMS96, RMS2005) between 1979-2016.
• Evaluation of relapse risk factors and salvage treatment outcomes.

Main Results:

• 30 out of 117 patients relapsed, primarily with local recurrence.
• Relapse was linked to younger age, female sex, specific protocols, incomplete initial surgery, and lack of radiotherapy.
• Salvage options included chemotherapy, radiotherapy, and surgery; 13 patients underwent orbit exenteration.
• Median follow-up of 12.7 years post-relapse; 10-year overall survival (OS) was 56.0%, and event-free survival (EFS) was 43.3%.

Conclusions:

• Recurrence of oRMS presents a significant challenge despite generally favorable initial prognosis.
• Multidisciplinary salvage therapy demonstrates potential for achieving long-term survival in over half of relapsed cases.
• Improved initial treatment selection is crucial to minimize relapse rates and long-term side effects.