Clinical features and prognostic factors in paediatric medulloblastoma: a retrospective cohort study

To evaluate the clinical characteristics, outcomes, and prognostic factors associated with paediatric medulloblastoma (MB). We retrospectively analysed paediatric MB patients treated at Xiangya Hospital Central South University from January 2015 to July 2023. Overall survival (OS) and progression-free survival (PFS) were estimated using the Kaplan‒Meier method, and prognostic factors were assessed via univariate and multivariate Cox regression. A total of 68 patients (median age 84 months, IQR 61–132) were included; all of them underwent surgery and received chemotherapy. Gross-total/near-total resection (GTR/NTR) was achieved in 62 (91.2%) patients, and 59 (86.8%) patients received adjuvant radiotherapy. At a median follow-up of 85 months, 17 deaths and 22 recurrences were documented, with 15 deaths occurring after relapse. The 5-year PFS and OS rates were 51.5%±9.1% and 72.8%±6.3%, respectively. Multivariate analysis revealed M+ stage as an independent predictor of worse OS (HR = 6.42, P = 0.017). Subtotal resection (STR) was independently associated with poor OS (HR = 4.11, P = 0.043) and PFS (HR = 4.80, P = 0.013), whereas chromosome 17q gain (chr17q +) independently predicted inferior PFS (HR = 3.44, P = 0.032). Among relapsed patients (n=22), early relapse (< 12 months) (HR = 5.07, P = 0.018) and metastatic (M +) status (HR=4.42, P =0.013) emerged as significant predictors of post-relapse mortality. This single-centre real-world study validated the prognostic value of the Chang stage and extent of resection in paediatric MB. Early relapse and M+ status were key determinants of post-relapse outcomes, while chr17q+ merits further exploration as a potential prognostic biomarker.