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Novel Pharmacological Treatment Options in Pediatric Glioblastoma-A Systematic Review
Johanna Wyss1,2, Nicole Alexandra Frank3, Jehuda Soleman3,4,5
1Division of Oncology-Hematology, Department of Pediatrics, Kantonsspital Aarau, 5001 Aarau, Switzerland.
Background:
Pediatric glioblastoma (GBM) is an aggressive central nervous system tumor in children that has dismal prognosis. Standard of care is surgery with subsequent irradiation and temozolomide. We aimed to outline currently available data on novel pharmacological treatments for pediatric GBM.
Methods:
We conducted a systematic literature search in PubMed and Embase, including reports published in English from 2010 to 2021. We included randomized trials, cohort studies and case series. Phase I trials were not analyzed. We followed PRISMA guidelines, assessed the quality of the eligible reports using the Newcastle-Ottawa scale (NOS) and the RoB-2 tool and registered the protocol on PROSPERO.
Results:
We included 6 out of 1122 screened reports. All six selected reports were prospective, multicenter phase II trials (five single-arm and one randomized controlled trial). None of the investigated novel treatment modalities showed any benefit regarding overall or progression free survival.
Conclusions:
To date, the role of pharmacological approaches regarding pediatric GBM remains unclear, since no novel treatment approach could provide a significant impact on overall or progression free survival. Further research should aim to combine different treatment strategies in large international multicenter trials with central comprehensive diagnostics regarding subgrouping. These novel treatment approaches should include targeted and immunotherapeutic treatments, potentially leading to a more successful outcome.
Insights
Novel treatments for pediatric glioblastoma (GBM) show no survival benefit. Further research is needed to explore targeted and immunotherapy combinations for better outcomes in pediatric central nervous system tumors.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Pharmacological Research
Background:
- Pediatric glioblastoma (GBM) is a highly aggressive brain tumor with poor prognosis in children.
- Current standard care involves surgery, radiation, and temozolomide, with limited success.
- There is a critical need for effective novel treatments for pediatric GBM.
Purpose of the Study:
- To systematically review and outline current data on novel pharmacological treatments for pediatric GBM.
- To assess the efficacy of emerging therapies in improving survival outcomes.
- To identify gaps in research and suggest future directions.
Main Methods:
- Systematic literature search of PubMed and Embase (2010-2021).
- Inclusion of randomized trials, cohort studies, and case series (Phase II trials).
- PRISMA guidelines followed; study quality assessed using NOS and RoB-2 tools.
Main Results:
- Six reports were included from 1122 screened studies.
- All included studies were prospective, multicenter phase II trials.
- No investigated novel treatment demonstrated significant improvement in overall or progression-free survival.
Conclusions:
- Current novel pharmacological approaches for pediatric GBM lack proven efficacy.
- Further research requires large international trials combining targeted and immunotherapies.
- Future strategies should focus on personalized treatment based on comprehensive diagnostics and subgrouping.
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