Survival of patients with orbital and eyelid rhabdomyosarcoma treated on Children's Oncology Group studies from 1997 to 2013: A report from the Children's Oncology Group

• Published in: Cancer Vol. 129; no. 11; pp. 1735 - 1743
• Main Authors: Metts, Jonathan, Xue, Wei, Gao, Zhengya, Ermoian, Ralph, Bradley, Julie A., Arnold, Michael A., Dasgupta, Roshni, Venkatramani, Rajkumar, Walterhouse, David
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 01.06.2023
• Subjects: Cancer Survivors; Child; Child, Preschool; Children; Children & youth; Clinical trials; Eyelid; Eyelids - pathology; Female; Health risks; Humans; Infant; Kaplan-Meier Estimate; Lymph nodes; Male; Oncology; orbital; Orbital Neoplasms - mortality; pediatric; Rhabdomyosarcoma; Risk; Sarcoma; Subgroups; Survival; Survival Rate; Tumors; sarcoma; rhabdomyosarcoma; orbital; survival; pediatric
• ISSN: 0008-543X; 1097-0142
• DOI: 10.1002/cncr.34723

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. The authors analyzed the long‐term survival outcomes for orbital rhabdomyosarcoma on Children's Oncology Group trials. The results indicated that long‐term survival outcomes were comparable to those reported at 3 years and 5 years, and a significant portion of recurrent orbital rhabdomyosarcomas appeared to be salvageable.