Phase II window of irinotecan plus carboplatin in newly diagnosed patients with rhabdomyosarcoma: Results of Memorial Sloan-Kettering Cancer Center (MSKCC) Protocol IRB 03-099.

Abstract

9533 Background: There has been little improvement in outcome for patients (pts) with intermediate (INT)- and high-risk (HR) rhabdomyosarcoma (RMS) in nearly 2 decades. Methods: From November 2003 through December 2009, we enrolled 56 newly diagnosed pts with INT- (28) and HR (28) RMS on IRB 03-099, a 7-drug regimen that commenced with 2 courses of irinotecan + carboplatin (I+C) given 3 weeks apart. I (20 mg/m2 IV over 1 hour) was given on days 1-5 and 8-12; C (560 mg/m2 IV over 1 hour) was given on day 1. Results: Median age at diagnosis was 11.5 yrs (4 months – 42 years); median tumor size was 6 cm. There were 29 males and 27 females; 30 pts (15 HR) had alveolar RMS (ARMS); 24 pts (12 HR) had embryonal RMS (ERMS); one each had sclerosing RMS and pleomorphic RMS (HR RMS). Of 28 pts with INT-RMS, 26 had invasive (T2) and/or 16 large (Tb) tumors, and 15 were N1. 11 of 28 pts with HR RMS had more than 1 site of metastatic disease. Pts were restaged after 2 cycles using RECIST and WHO response criteria. 4 pt...