Abstract
A subset of patients with initially unresected (Clinical Group III) rhabdomyosarcoma achieve less than a complete response (CR) despite multimodal therapy. We assessed outcome based upon tumor response at the completion of all planned therapy. We studied 601 Clinical Group III participants who completed all protocol therapy without developing progressive disease on two Children's Oncology Group studies ARST0531 (n = 285) and D9803 (n = 316). Response was defined by imaging and categorized by response; complete resolution (CR), partial response (PR) or no response (NR). Failure-free survival (FFS) and overall survival (OS) between response groups were compared using the log-rank test. We found that radiographic response was CR in 393 (65.4%) and PR/NR in 208 (34.6%) patients. Achieving CR status was associated with study D9803, nonparameningeal (PM) primary sites, tumors ≤5 cm, noninvasive tumors and alveolar histology/FOXO fusion-positive tumors. The overall 5-year FFS was 75% for those achieving CR and 66.5% in those with PR/NR (adj. p = 0.094). Patients with PM primary site who achieved CR had significantly improved FFS (adj. p = 0.037) while those with non-PM primary sites had similar outcomes (adj. p = 0.47). Radiographic response was not associated with OS (adj. p = 0.21). Resection of the end-of-therapy mass did not improve FFS (p = 0.12) or OS (p = 0.37). In conclusion, CR status at the end of protocol therapy in patients with PM Clinical Group III RMS was associated with improved FFS but not OS. Efforts to understand the biology and treatment response in patients with PM primary site are under investigation.