Brain tumors in infancy and early childhood: a retrospective cohort study from a low- and middle-income country

Abstract Background Brain tumors in infancy and early childhood exhibit distinct clinicopathologic features and outcomes compared to older children. Despite their clinical significance, no study in this region has specifically characterized brain tumors in this age group. This study aimed to describe the clinical presentation, anatomical distribution, histopathology, prognostic factors, and outcomes of brain tumors in children under three years of age. Methods We conducted a retrospective cohort study at Aga Khan University Hospital, Karachi, Pakistan, including children under three years diagnosed with brain tumors between 2013 and 2024. Demographic data, clinical presentation, tumor characteristics, treatment details, and outcomes were collected. Survival analyses were performed using Kaplan-Meier curves, log-rank tests, and Cox proportional hazards regression to identify independent prognostic factors. Statistical analysis was performed using SPSS and STATA, with a median follow-up of 60 months. Results Over 11.5 years, 50 children under three years were diagnosed with brain tumors. Vomiting (58%) was the most common presenting symptom. Tumors were predominantly infratentorial (62%), with ependymomas (25.5%) and medulloblastomas (23.4%) being the most frequent histological types. Gross total or near-total resection was achieved in 53.1% of cases, while temporary and permanent cerebrospinal fluid diversion were performed in 53.3% and 54% of patients, respectively. Intraoperative complications were rare (4.1%), whereas postoperative complications, including persistent hydrocephalus, occurred in 62% of patients. Adjuvant chemotherapy and radiotherapy were administered to 34% and 26.7% of patients, respectively. The overall mortality rate was 68%, with 12 patients alive at a median Lansky score of 95. Median overall survival (OS) was highest for pilocytic astrocytoma (71.38 months) and lowest in neonates (0–3 months). Kaplan-Meier analyses demonstrated improved OS and progression-free survival (PFS) with a greater extent of resection, absence of metastases, and receipt of adjuvant therapy. Univariate analyses demonstrated associations between survival and patient age, extent of resection, metastatic dissemination, and radiological characteristics. Conclusion Brain tumors in children under three years are rare and associated with substantial morbidity and mortality. Clinical presentation is non-specific and varies with age. Surgical resection remains the primary treatment, with more extensive resections associated with improved outcomes. Patients receiving adjuvant therapy demonstrated improved survival, although these findings should be interpreted with caution given the retrospective study design. Future research should investigate age-related variations in tumor biology, treatment response, and long-term survival and quality-of-life outcomes.