Related Experiment Video
Updated: Oct 17, 2025
Live-3D-Cell Immunocytochemistry Assays of Pediatric Diffuse Midline Glioma
Published on: November 11, 2021
Distinct survival and clinical profile of infantile glioblastoma: insights from a national database
Victor M Lu1, Daniel G Eichberg2, Evan M Luther2
1Department of Neurological Surgery, University of Miami Miller School of Medicine, 1095 NW 14th Terrace, Miami, FL, 33136, USA. victor.lu@jhsmiami.org.
Insights
Infantile glioblastoma (GBM) is rare and distinct, with a longer survival than other pediatric and adult forms. Chemotherapy significantly improves outcomes for these young patients.
Area of Science:
- Oncology
- Pediatric Oncology
- Cancer Research
Background:
- Infantile glioblastoma (GBM) is exceptionally rare.
- Its characteristics and survival outcomes are poorly understood compared to adult GBM.
- This study addresses the knowledge gap by analyzing infantile GBM demographics.
Purpose of the Study:
- To investigate the clinical profile and survival patterns of infants diagnosed with glioblastoma.
- To compare infantile GBM with other age groups using a national cancer database.
Main Methods:
- Retrospective review of the U.S. National Cancer Database (NCDB) from 2005-2016.
- Inclusion criteria: GBM patients aged 1 year or younger.
- Survival analysis using Kaplan-Meier and Cox regression models.
Main Results:
- 86 infants met the criteria, representing 0.08% of all GBM diagnoses.
- Median overall survival (OS) was 67.3 months, significantly longer than other groups.
- Chemotherapy independently predicted longer OS (HR=0.18, P<0.01); Hispanic status and comorbidity predicted shorter OS.
Conclusions:
- Infantile GBM is a distinct entity with a favorable survival prognosis.
- Chemotherapy is a critical treatment component for improving outcomes in this population.
- Further research into the biological and genetic underpinnings of infantile GBM is warranted.
Background:
The diagnosis of glioblastoma (GBM) in infants aged ≤ 1 year is extremely rare, and its comparability to the more common adult diagnosis is underexplored. Correspondingly, the objective of this study was to interrogate a national cancer database to elucidate the typical survival and clinical profile of this demographic.
Methods:
All GBM patients aged ≤ 1 year in the U.S. National Cancer Database (NCDB) between 2005 and 2016 were retrospectively reviewed. Data were summarized, and overall survival (OS) was modeled using Kaplan-Meier and Cox regression analyses.
Results:
A total of 86 patients satisfied criteria for entry into study, making up 0.08% of all GBM diagnoses in the database. There were 32 (37%) females and 54 (63%) males. Irrespective of treatment, median OS was 67.3 months (95% CI, 46-91), which was distinct from all other ages and pediatric age groups. There were 74 (86%) treated by surgery, 51 (59%) treated by chemotherapy, and 17 (20%) treated by radiation therapy. Multivariable analysis demonstrated that Hispanic status (HR = 3.41, P = 0.02) and the presence of comorbidity (HR = 3.24, P = 0.01) independently predicted shorter OS, whereas treatment with chemotherapy (HR = 0.18, P < 0.01) independently predicted longer OS. Neither extent of surgery nor radiation therapy demonstrated independent statistical significance.
Conclusion:
Infantile GBM should be viewed as a distinct GBM entity with a longer OS than other pediatric and adult patients. Chemotherapy is a statistically significant component in the treatment of this demographic, and the value of surgical treatment is likely universal. Future studies into understanding the biological and genetic profile of infantile GBM are needed to advance both pediatric and adult fields.
