COG ACNS0332
ACNS0332 found that giving carboplatin alongside radiotherapy improved survival for children with the most aggressive group 3 medulloblastomas, while isotretinoin, tested in the same trial, did nothing and was dropped.
Overview
ACNS0332 randomised children and young adults with high-risk medulloblastoma (metastatic, incompletely resected or with anaplasia) to 36 Gy craniospinal radiotherapy plus weekly vincristine with or without daily carboplatin as a radiosensitiser, followed by maintenance chemotherapy with or without isotretinoin. The isotretinoin randomisation closed early for futility.
Carboplatin during radiotherapy did not improve event-free survival in the whole cohort, but in the molecularly defined group 3 subgroup it improved event-free and overall survival, a finding that came from the trial's tumour profiling. The result made carboplatin radiosensitisation a standard for group 3 high-risk disease and was one of the first paediatric brain tumour trials to show benefit in a molecular subgroup.
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