title: Diffuse brainstem glioma in children: critical review of clinical trials.
abstract: Diffuse intrinsic brainstem gliomas constitute 15-20% of all CNS tumours in children, and are the main cause of death in children with brain tumours. Many clinical trials have been done over the past three decades, but survival has remained static. More than 90% of children die within 2 years of diagnosis, and conventional fractionated radiation remains the standard treatment. However, median survival differs substantially between clinical trials, suggesting a survival benefit with some strategies. We appraised the consistency between protocols in terms of eligibility criteria, definition and assessment of response and progression, statistical design, and endpoints. Study designs varied substantially, which could explain the differences in outcome, and no treatment has shown a benefit over conventional radiotherapy. However, consistency between protocols (eg, eligibility criteria and outcome measures) is important to measure the progress in management of diffuse pontine gliomas.
authors.affiliation: Department of Paediatric Oncology, Royal Marsden Hospital, Sutton, Surrey, UK.
authors.forename: Darren
authors.initials: D
authors.lastname: Hargrave
authors.forename: Ute
authors.initials: U
authors.lastname: Bartels
authors.forename: Eric
authors.initials: E
authors.lastname: Bouffet
mesh_terms: D020295:Brain Stem Neoplasms / Q000473:pathology / Q000532:radiotherapy*; D002648:Child; D002986:Clinical Trials as Topic; D023381:Endpoint Determination; D005910:Glioma / Q000473:pathology / Q000532:radiotherapy*; D006801:Humans; D008279:Magnetic Resonance Imaging; D012107:Research Design; D016019:Survival Analysis; D016896:Treatment Outcome