Isotretinoin has no effect on event-free survival across high-risk medulloblastoma molecular groups when added to maintenance: A secondary analysis of the Children’s Oncology Group ACNS0332 data

Raskin S, de Blank P, Billups CA, Li Y, Olson JM, Leary SES. Isotretinoin has no effect on event-free survival across high-risk medulloblastoma molecular groups when added to maintenance: A secondary analysis of the Children’s Oncology Group ACNS0332 data. Neurooncol Adv. 2025 Mar 4;7(1):vdaf054. doi: 10.1093/noajnl/vdaf054. PMID: 40276374; PMCID: PMC12019958.

The Children’s Oncology Group (COG) study ACNS0332 examined the effect of adding carboplatin and isotretinoin to high-risk medulloblastoma therapy. Isotretinoin arms were closed early due to futility, but the effect of carboplatin was shown to vary by individual medulloblastoma subgroups. Because isotretinoin arms were closed before subgroup classification was available, a differential effect of isotretinoin among various subgroups was not examined. Here, we conduct a secondary analysis of ACNS0332 data examining the effect of isotretinoin on event-free survival (EFS) among individual medulloblastoma subgroups.