作者: AA Shelat , M Remke , GW Robinson , S Rutkowski , T Schoep
DOI: 10.1007/S00401-013-1213-7
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摘要: Medulloblastoma is curable in approximately 70 % of patients. Over the past decade, progress improving survival using conventional therapies has stalled, resulting reduced quality life due to treatment-related side effects, which are a major concern survivors. The vast amount genomic and molecular data generated over last 5–10 years encourages optimism that improved risk stratification new targets will improve outcomes. It now clear medulloblastoma not single-disease entity, but instead consists at least four distinct subgroups: WNT/Wingless, Sonic Hedgehog, Group 3, 4. Down Under 2013 meeting, convened Bunker Bay, Australia, brought together 50 leading clinicians scientists. 2-day agenda included focused sessions on pathology stratification, genomics mouse models, high-throughput drug screening, clinical trial design. meeting established global action plan translate novel biologic insights targeting into treatment regimens A consensus was reached several key areas, with most important being classification scheme for based subgroups, as well histopathologic features, should be presented consideration upcoming fifth edition World Health Organization’s tumours central nervous system. Three other notable areas agreement were follows: (1) establish repository annotated models readily accessible freely available international research community; (2) institute common eligibility criteria between Children’s Oncology International Society Paediatric Europe initiate joint or parallel trials; (3) share preliminary screening across discovery labs hasten development therapeutics. an effective forum meaningful discussion, resulted enhancing collaborative translational this rare disease. This template could applied fields devise plans addressing all aspects disease, from disease classification, superior assessed cooperative trials.