Work

Ept-06outcomes for Patients With Recurrent Medulloblastoma or Ependymoma Enrolled on Phase Ii Clinical Trials: an Analysis of Contemporary Children's Oncology Group (cog) and Pediatric Brain Tumor Consortium (pbtc) Trials

Year: 2016

Type: article

Abstract: BACKGROUND: Well-defined historical controls are not available to design Phase II trials with appropriate endpoints in children with recurrent medulloblastoma (rMB) or ependymoma (rEP). This study sought to examine whether PFS across COG and PBTC Phase II studies could provide suitable parameters for future study design. METHODS: Six contemporary COG and PBTC Phase II trials with similar eligibility and assessment criteria enrolled 73 children with rMB and 49 with rEP. Patients with rMB (67% male) enrolled at a median of 10.3y (range,0.8-23.5), after prior XRT (83%) and/or chemotherapy (100%). Patients with rEP (55% male) enrolled at a median of 8.8y (range,1.2-21.4), were 53% grade 3, 62% infratentorial, after prior XRT (97%) and/or chemotherapy (77%). Median functional score was 90 (range, 60-100 and 50-100, respectively). RESULTS: Partial responses were noted in two subjects with rMB (2.7%) and one with rEP (2.0%); median PFS across trials was 55 days (range,45-66), and 60 days (range,44-64), respectively. Improved PFS for rMB was associated with prior XRT (p < 0.001) and older age at diagnosis (p = 0.024) or enrollment (p = 0.026). No association was detected for rEP between PFS and the examined variables. In rMB, 4-month PFS (PFS4) was homogeneous with a median of 18%; future trials with PFS4 significantly larger than 38% (90% upper bound) could be considered successful. For rEP no parameters were homogeneous enough for future trial design. CONCLUSIONS: Our analysis provides an overview of historical controls and suggests that PFS4 may be a reliable endpoint for design of future phase II trials in rMB.

Source: Neuro-Oncology

DOI: 10.1093/neuonc/now069.05