Integrated biomarker and treatment correlates of prognosis in infant non-WNT/non-SHH medulloblastoma: a multinational retrospective cohort study - PubMed
5 hours ago
- #infant medulloblastoma
- #retrospective cohort study
- #biomarker-driven prognosis
- Infant non-WNT/non-SHH medulloblastoma accounts for ~60% of infant medulloblastoma cases and is currently considered uniformly high-risk.
- This study retrospectively analyzed an international cohort of 375 infants with non-WNT/non-SHH medulloblastoma, categorized into MBGroup3 (66%) and MBGroup4 (34%).
- Upfront craniospinal irradiation significantly improved 5-year progression-free survival (62%) compared to non-craniospinal irradiation approaches (33%).
- When using chemotherapy-only upfront, high-dose chemotherapy yielded better 5-year overall survival (60%) than standard-dose chemotherapy (27%).
- MBGroup3 subgroup 4 tumors represented a novel chemosensitive favorable-risk group with 5-year progression-free survival of 64% when treated upfront with high-dose chemotherapy only.
- MBGroup3 subgroup 2 or 3 tumors with MYC amplification constituted a very-high-risk group, with 0% 5-year survival when treated with chemotherapy only.
- The study provides a foundation for biomarker-driven clinical trials by identifying distinct prognostic subgroups based on molecular pathology and treatment.