EPT-07
PARTICIPATION OF CHILDREN AND ADOLESCENTS WITH CENTRAL NERVOUS SYSTEM TUMOURS IN PHASE I TRIALS WITHIN THE ITCC EUROPEAN CONSORTIUM

BACKGROUND: Early phase trials are crucial in the evaluation of novel anti-cancer agents for children, including those with central nervous system (CNS) tumours. Optimal patient selection is necessary to maximise clinical benefit for the patient, whilst ensuring evaluability to determine trial objectives. Data from children/adolescents participating in phase I trials across the Innovative Therapies for Children with Cancer (ITCC) European consortium was analysed to determine prognostic factors of survival, including two scores previously validated in adult cancer patients: the Royal Marsden Hospital (RMH) and MD Anderson Cancer Centre (MDACC) scores. METHODS: Patients <18 years at enrolment in their first phase I trial between 2000-2014 were eligible. Descriptive and uni/multivariate analyses of overall survival (OS) were conducted. RESULTS: Among 257 patients treated across 8 institutions, 123 (48%) had CNS tumours. Main diagnoses: medulloblastoma/PNET (30%), DIPG (24%) and high-grade gliomas (22%). Median age: 10.0 years (range, 1.0-17.9). Objective response was reported in 10 patients (8%), stable disease in 30 (24%). Median OS was 5.9 months (95%CI 4.2-7.5). In the univariate analysis, performance status (PS) ≤80%, no school/work attendance, ALT or AST above upper limit of normal significantly correlated with worse OS. In the multivariate analysis, only PS ≤ 80% correlated with worse OS: HR (95%CI) 1.674 (1.0-2.8); p = 0.05. The RMH and MDACC scores did not correlate with survival. CONCLUSIONS: CNS tumours represent a significant population in paediatric phase I trials. Evaluation of new agents in these patients is feasible and PS at enrolment may help in selecting the patient population for early clinical trials.