High-grade glioma in infants and very young children: characteristics, treatment, and outcomes

Abstract

Abstract Purpose High-grade gliomas (HGG) in infants and very young children (less than 3 to 5 years old) pose significant challenges due to the limited scientific literature available and high risks associated with treatments. This study aims to investigate the characteristics, treatment, and outcomes of this cohort. Methods A retrospective cohort study was conducted from 2007 to 2022 at Children’s Cancer Hospital, Egypt (CCHE-57357). Cases included children aged < 5 years old with pathologically confirmed CNS glioma grades III-IV. Baseline clinical and radiological characteristics, besides prognostic factors associated with survival, were assessed. Results In total, 76 cases were identified, 7 of them (9.2%) were < 1 year old, and 25 (32.9%) were aged 1 to 3 years. Most tumors were cerebral (55.3%). Gross- or near-total resection (GTR/NTR) was achieved in 32.9% of all cases. Of the tested cases, H3K27M-alteration was present in 5 subjects only. The 3-year OS and EFS for all cases were 26.9% and 15.4%, respectively. Extent of resection was the most important prognostic factor, as those achieving GTR/NTR experienced more than double the survival compared to those who do not (1-year EFS = 46.6% vs 21.4%, p=0.05). Age had a “bimodal” effect on EFS, with those aged 1 to 3 years old (1-year EFS = 40%) faring better than younger (EFS = 0%; p=0.02) and older age groups (1-year EFS = 28.1%; p=0.05). Subjects with midline tumors had worse survival compared to non-midline tumors (1-year EFS = 18.5% vs 35%, respectively, p=0.02). Conclusion This study in a large cohort of HGG in infants and very young children offers insights into the characteristics and treatment challenges. Extent of resection, age group, and tumor localization are important prognostic factors. Further research with larger sample size is warranted to refine treatment approaches and improve outcomes.