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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.
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.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
379 enrolled.
Isotretinoin randomisation closed early for futility
Source| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Event-free survival at 5 yearsprimary | Carboplatin with craniospinal radiotherapy | - | 66.4% | - | 0.11 | link |
| No carboplatin | - | 59.2% | ||||
| Event-free survival at 5 years, group 3 medulloblastoma | Carboplatin with craniospinal radiotherapy | - | 73.2% | - | 0.047 | link |
| No carboplatin | - | 53.7% | ||||
| Overall survival at 5 years, all evaluable participants | All evaluable participants | 261 | 73.4% | - | - | link |
Shares Children's Oncology Group (COG), Vincristine, Cytotoxic chemotherapy and the tag subtype-trials.
Shares Platinum agents, Cisplatin, Cytotoxic chemotherapy and the tag subtype-trials.
Shares Cyclophosphamide, Platinum agents, Carboplatin, Cytotoxic chemotherapy and the tag subtype-trials.
Shares Children's Oncology Group (COG), Cytotoxic chemotherapy and the tag subtype-trials.
Shares Vincristine, Cytotoxic chemotherapy and the tag subtype-trials.
Shares Cisplatin, Carboplatin and the tag subtype-trials.
Shares Carboplatin, Cytotoxic chemotherapy and the tag subtype-trials.
Shares Vincristine, Cyclophosphamide and the tag subtype-trials.