Abstract
BACKGROUND: Orbital rhabdomyosarcoma (ORMS) commonly presents as low-risk disease (stage 1, group I-III, embryonal RMS) with excellent outcome. Long-term follow-up of patients with low-risk ORMS and outcomes of less common subgroups of ORMS treated on recent Children's Oncology Group (COG) trials have not been reported. METHODS: Patients with ORMS enrolled on COG trials from 1997 to 2013 were identified. Demographic information and disease characteristics were collected. Outcomes were determined for the following subgroups: 1) low-risk ORMS, 2) resected (group I/II) low-risk ORMS, 3) non-low-risk ORMS, and 4) recurrent ORMS. Event-free survival (EFS) and overall survival (OS) were estimated using the Kaplan-Meier method. ResultsThe authors identified 218 patients with ORMS. Most tumors were embryonal/botryoid (n = 169; 77.5%), <5 cm (n =" 213;" 97.7%), group iii (n =" 170;" 78.0%), and without lymph node involvement (n0; n =" 215;" 98.6%). for 192 patients with low-risk orms, the 10-year efs and os rates were 85.5% (95% confidence interval [ci], 77.0%-94.0%) and 95.6% (95% ci, 90.8%-100.0%), respectively. those with group i ii low-risk orms (n="5" in group i; n="39" in group iia) had 10-year efs and os rates of 88.0% (95% ci, 72.6%-100.0%) and 97.6% (95% ci, 90.0%-100.0%), respectively. twenty-six patients with non-low-risk orms had 5-year efs and os rates of 88.5% (95% ci, 75.6%-100.0%) and 95.8% (95% ci, 87.7%-100.0%), respectively. for patients with recurrent orms, the 10-year os rate from the time of recurrence was 69.4% (95% ci, 50.0%-88.8%). conclusions: patients with orms had favorable long-term survival outcomes on cog studies from 1997 to 2013, including those who had both low-risk and non-low-risk disease. a significant proportion of patients with recurrent orms may achieve long-term survival.>
