Long-term outcomes of pediatric medulloblastoma before routine molecular classification: a 20-year single-center cohort study.

purposeMedulloblastoma is the most common malignant brain tumor of childhood. Although molecular classification has refined prognostication, long-term real-world outcomes from periods or settings without routine molecular subtyping remain clinically relevant. We report a 20-year single-center experience from a tertiary pediatric oncology center in Turkey.

methodsWe retrospectively analyzed 97 children diagnosed with Medulloblastoma at Uludag University between 2000 and 2020. Survival status was updated through 2025. Overall survival (OS) and event-free survival (EFS) were estimated using Kaplan-Meier analysis. Prognostic factors were assessed using log-rank tests and multivariate Cox regression for OS and EFS. Late effects among survivors were recorded.

resultsThe cohort included 56 males and 41 females, with a median age of 7.0 years. Five-year OS and EFS were 73.9% and 69.9%, respectively, with a median follow-up of 70.2 months. Five-year OS improved from 57.1% in 2000-2010 to 83.6% in 2011-2020. Later treatment era independently predicted improved OS (HR, 0.30; 95% CI, 0.14-0.65; p = 0.002) and EFS (HR, 0.38; 95% CI, 0.20-0.75; p = 0.005). Anaplastic/large-cell histology independently predicted poorer OS and EFS. Baseline characteristics were largely similar between eras, although median age differed. Late effects were documented in 56 of 68 survivors (82%), most commonly motor or cerebellar dysfunction, growth failure, neurocognitive impairment, and hypothyroidism.

conclusionSurvival improved across treatment eras and was not readily explained by a more favorable measured case mix. These findings provide pre-molecular era real-world benchmarks and highlight the importance of treatment standardization and structured survivorship care.