Orbital rhabdomyosarcoma in children: a favorable primary suitable for a less-invasive treatment strategy.

ABSTRACT

BACKGROUND: Orbital rhabdomyosarcoma (ORMS) treatment is based on combination chemotherapy associated with best local therapy, sometimes surgery but more often radiation therapy. A retrospective single-center analysis was conducted to more clearly define the long-term outcome of patients with ORMS, to identify patients in whom aggressive first-line local therapy can be avoided. POPULATION A total of 95 patients with localized parameningeal (PM) or nonparameningeal (NPM) ORMS, treated at the Institut Curie between 1975 and 2010, were analyzed. RESULTS: Median age at diagnosis was 6 years (range, 8 mo to 19.5 y), and median follow-up was 8.5 years (range, 7 mo to 24 y). A total of 25 patients presented PM extension. Radiation therapy was part of primary therapy for 78 patients. Five-year event-free survival and overall survival rates were 65.4%±5.2% and 85.6%±3.9%, respectively. On multivariate analysis, initial tumor size was identified as a significant prognostic factor. Event-free survival was similar for PM and NPM tumors (60.3%±10.4% vs. 62.7%±5.9%, P=0.57), whereas there was a trend for overall survival to be better for NPM tumors (90%±3.9% vs. 72.7%±9.6%, P=0.07). CONCLUSIONS: Localized ORMS has a favorable outcome despite the current trend toward less aggressive and more limited indications of local therapy. Patients with a favorable pattern of strictly ORMS can be treated without first-line radiation therapy.