EPT-07PARTICIPATION OF CHILDREN AND ADOLESCENTS WITH CENTRAL NERVOUS SYSTEM TUMOURS IN PHASE I TRIALS WITHIN THE ITCC EUROPEAN CONSORTIUM
Authors: Fernando Carceller, Francisco Bautista, Irene Jiménez, Raquel Hladun-Alvaro, Cécile Giraud, Luca Bergamaschi, Madhumita Dandapani, Isabelle Aerts, François Doz, Didier Frappaz, Michela Casanova, Bruce Morland, Darren Hargrave, Lynley Marshall, Gilles Vassal, Andrew Pearson, Birgit Geoerger, Lucas Moreno
Journal: Neuro-Oncology (2016)

EPT-07. PARTICIPATION OF CHILDREN AND ADOLESCENTS WITH CENTRAL NERVOUS SYSTEM TUMOURS IN PHASE I TRIALS WITHIN THE ITCC EUROPEAN CONSORTIUM Fernando Carceller1,2, Francisco Bautista3,4, Irene Jimenez5, Raquel Hladun-Alvaro3, Cecile Giraud6, Luca Bergamaschi7, Madhumita Dandapani8, Isabelle Aerts5, Francois Doz5, Didier Frappaz6, Michela Casanova7, Bruce Morland8, Darren Hargrave9, Lynley Marshall1,2, GillesVassal3,AndrewPearson1,2,Birgit Geoerger3, andLucas Moreno4; The RoyalMarsden NHS Foundation Trust, London, UK; The Institute of Cancer Research, London, UK; Goustave Roussy, Villejuif, France; Hospital Nino Jesus,Madrid, Spain; InstitutCurie, Paris,France; InstitutHematologiqueet d’Oncologie Pediatrique, Lyon, France; IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Birmingham Children’s Hospital, Birmingham, UK; Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK 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 1⁄4 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. Neuro-Oncology 18:iii24–iii29, 2016. doi:10.1093/neuonc/now069.6 #The Author(s) 2016. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.