Multimodal management and outcome of pediatric and adolescent malignant central nervous system tumors: A single-center retrospective study

Objective

There is a paucity of real-world evidence in the Indian context to address the outcome of primary brain tumors (PBTs) in children. This study aimed to describe the demographic profile, clinical characteristics, and histological features of PBTs based on the 2016 World Health Organization classification, assess the efficacy of treatment methods, and identify the factors that influence the outcome.

Methodology

This is a single-institution, hospital-based study. Data were collected for pediatric patients aged 0−19 years, from September 2001 to May 2023 (22 years), who were diagnosed with malignant PBTs. Patients with radiologically or histologically proven tumors were included. Those with metastatic disease to the central nervous system were excluded. The overall survival (OS) and recurrence-free survival (RFS) were estimated using the Kaplan–Meier method.

Results

A total of 251 patients with pediatric brain tumors were included in this analysis. The mean age was 9.10 ± 5.54 years. The male-to-female ratio was 1.20:1. In this cohort, the most common histologies were medulloblastoma and astrocytoma. The mean survival of all patients with PBTs was 141.00 ± 7.90 months with 1-, 3-, and 8-year OS rates of 79.00%, 67.00%, and 60.00%, respectively. Medulloblastoma had 1-, 3-, and 8-year OS rates of 81.00%, 72.00%, and 65.00%, respectively. The 1-year OS rates for glioblastoma and brainstem glioma were 46.00% and 45.00%, respectively. Complete tumoral resection showed longer survival than lesser degrees of resection (p = 0.001). Embryonal tumors (ETs) had a better RFS of 133.60 ± 12.70 months (p ≤ 0.001).

Conclusion

ETs have a better prognosis than glial tumors. With an improved OS, the surgical resection extent has a favorable outcome. As a chemosensitive tumor, medulloblastoma benefits most from systemic treatment and responds well to a multimodal approach.