Group 4 Medulloblastomas of Early Childhood Treated With High-Dose Chemotherapy- and Craniospinal Irradiation-Sparing Approach - PubMed

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Group 4 medulloblastoma (MB) is rare in young children, and data on craniospinal irradiation (CSI)-sparing approaches are limited.
A multicenter study reported outcomes for patients younger than 7 years old with Group 4 MB treated with high-dose chemotherapy (HDC) without adjuvant CSI.
38 patients were included, with a median age of 46.4 months at diagnosis; 24% were ≤36 months old.
63.2% had localized disease, while 36.8% presented with metastatic disease; 68.4% underwent gross total resection (GTR).
The most used HDC regimens were carboplatin/thiotepa (76.3%) and carboplatin/thiotepa/etoposide (21.1%).
52.6% of patients experienced relapses at a median of 21.9 months from diagnosis.
Patients with upfront GTR and/or three cycles of HDC had better progression-free survival (PFS).
Local relapse accounted for 45% of cases; 18 patients received salvage therapy with curative intent, all involving radiotherapy.
The 5-year post-relapse survival and overall survival (OS) were 60.3% and 72.7%, respectively.
The 5-year CSI-free OS was 69.7%, suggesting HDC may be an alternative to upfront CSI for young children with Group 4 MB.

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