Improved outcome for patients with middle ear rhabdomyosarcoma: A children's oncology group study

Purpose: The goal of this study was to define the clinical features and opt imal therapy for children and adolescents with middle ear (ME) rhabdomyosar coma (RMS). Patients and Methods: We reviewed demographic data, clinical features, ther apy (including chemotherapy, surgery, and radiation), and outcome for the 1 79 eligible patients with ME RMS who were enrolled onto Intergroup Rhabdomy osarcoma Studies (IRS) I through IV or pilot studies between November 1972 and December 1997, Results: Most patients were younger than 10 years old (90%), and 63% were m ale. Because of the parameningeal location, most tumors were not resected b efore chemotherapy (group I, < 1%; group II, 4%; group III, 84%; group IV, 12%). Although most tumors were locally invasive (T2, 89%), the majority we re small (<less than or equal to> 5 cm, 66%), lacked nodal metastases (N0, 86%), and had embryonal histology (85%). The 5-year failure-free survival ( FFS) and overall survival (OS) estimates were 67% and 72%, respectively. Bo th FFS and OS improved significantly over the course of IRS I through IV (3 -year FFS and OS: IRS-I, 42% and 42%; IRS-II, 70% and 74%; IRS-III, 65% and 72%; IRS-IV pilot, 81% and 96%; IRS-IV, 88% and 88%, P < .001). Lower clin ical group or stage and smaller tumor size were associated with better outc ome. Age, sex, tumor invasiveness, and nodal metastases were not predictive of outcome. Conclusion: Patients with ME RMS generally present with small, unresectable , invasive tumors at a site traditionally considered prognostically unfavor able. Nevertheless, such patients have benefited markedly from improvements in multimodal, risk-based therapy during the course of IRS I through IV, a nd with contemporary therapy, most are cured. J Clin Oncol 19:3073-3079. (C ) 200l by American Society of Clinical Oncology.