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- Title
Outcome of children and adolescents with central nervous system tumors in phase I trials.
- Authors
Carceller, Fernando; Bautista, Francisco; Jiménez, Irene; Hladun-Álvaro, Raquel; Giraud, Cécile; Bergamaschi, Luca; Dandapani, Madhumita; Aerts, Isabelle; Doz, François; Frappaz, Didier; Casanova, Michela; Morland, Bruce; Hargrave, Darren R.; Vassal, Gilles; Pearson, Andrew D. J.; Geoerger, Birgit; Moreno, Lucas; Marshall, Lynley V.
- Abstract
Central nervous system (CNS) tumors are a leading cause of death in pediatric oncology. New drugs are desperately needed to improve survival. We evaluated the outcome of children and adolescents with CNS tumors participating in phase I trials within the Innovative Therapies for Children with Cancer (ITCC) consortium. Patients with solid tumors aged < 18 years at enrollment in their first dose-finding trial between 2000 and 2014 at eight ITCC centers were included retrospectively. Survival was evaluated using univariate/multivariate analyses. Overall, 114 patients were included (109 evaluable for efficacy). Median age was 10.2 years (range 1.0–17.9). Main diagnoses included: medulloblastoma/primitive neuroectodermal tumors (32.5%) and high-grade gliomas (23.7%). Complete/partial responses (CR/PR) were reported in 7.3% patients and stable disease (SD) in 23.9%. Performance status of 90–100%, school/work attendance, normal ALT/AST and CR/PR/SD correlated with better overall survival (OS) in the univariate analysis. No variables assessable at screening/enrollment were associated with OS in the multivariate analysis. Five patients (4.5%) were discontinued from study due to toxicity. No toxic deaths occurred. Median OS was 11.9 months with CR/PR, 14.5 months with SD and 3.7 months with progressive disease (p < 0.001). The enrollment of children and adolescents with CNS tumors in phase I trials is feasible, safe and offers potential benefit for the patients. Sustained disease stabilization has a promising role as a marker of anti-tumor activity in children with CNS tumors participating in phase I trials.
- Subjects
ANTINEOPLASTIC agents; TARGETED drug delivery; CLINICAL trials; TUMORS in children
- Publication
Journal of Neuro-Oncology, 2018, Vol 137, Issue 1, p83
- ISSN
0167-594X
- Publication type
Article
- DOI
10.1007/s11060-017-2698-z