Treatments for Children With Newly Diagnosed Medulloblastoma: A Systematic Review Of Randomized Controlled Trials
Author(s)
Leticia Barcena, Ldo. Vet1, Musku Kumaraswamy, M.Pharma2, Anu Priya, M.Pharma2, Sunita Nair, PhD3.
1Clarivate, London, United Kingdom, 2Clarivate, Banglore, India, 3Clarivate, Mumbai, India.
1Clarivate, London, United Kingdom, 2Clarivate, Banglore, India, 3Clarivate, Mumbai, India.
OBJECTIVES: To provide a comprehensive overview of randomised controlled trials (RCTs) assessing therapies in newly diagnosed medulloblastoma (MB), which is the most common malignant central nervous system tumor in children.
METHODS: We conducted a systematic literature review (SLR) following the PRISMA guidelines to identify RCTs assessing treatments in children (aged 0 to 21 years) with newly diagnosed MB. Interventions included any type of surgery, radiotherapy, chemotherapy, gene therapy and oncolytic virus. Searches were run on 9 June 2025 in MEDLINE, Embase, and Cochrane library (via OVID). Searches were limited to publications (including conference abstracts) in the English language. Publications were screened and data extracted by two independent reviewers.
RESULTS: In total, 34 publications reporting on 20 RCTs were included in the SLR. Most of the RCTs assessed chemotherapy (n=17 [85%]) followed by radiotherapy (n=3 [15%]). There were no RCTs assessing surgery or gene therapies. The sample size ranged from 16 to 464 (median 183.5). Of the eight RCTs that specified population risk, four involved high-risk patients, three involved standard-risk patients, and one included both high-and low-risk patients. The SLR is ongoing and full results for outcomes (event-free survival, overall survival, response rates and adverse events) will be presented in the poster.
CONCLUSIONS: The evidence of clinical effectiveness and safety of interventions for the treatment of newly diagnosed MB in children arising from RCTs is very limited. More data, especially from RCTs are required to fill the evidence gap on effectiveness of therapies and management of adverse events.
METHODS: We conducted a systematic literature review (SLR) following the PRISMA guidelines to identify RCTs assessing treatments in children (aged 0 to 21 years) with newly diagnosed MB. Interventions included any type of surgery, radiotherapy, chemotherapy, gene therapy and oncolytic virus. Searches were run on 9 June 2025 in MEDLINE, Embase, and Cochrane library (via OVID). Searches were limited to publications (including conference abstracts) in the English language. Publications were screened and data extracted by two independent reviewers.
RESULTS: In total, 34 publications reporting on 20 RCTs were included in the SLR. Most of the RCTs assessed chemotherapy (n=17 [85%]) followed by radiotherapy (n=3 [15%]). There were no RCTs assessing surgery or gene therapies. The sample size ranged from 16 to 464 (median 183.5). Of the eight RCTs that specified population risk, four involved high-risk patients, three involved standard-risk patients, and one included both high-and low-risk patients. The SLR is ongoing and full results for outcomes (event-free survival, overall survival, response rates and adverse events) will be presented in the poster.
CONCLUSIONS: The evidence of clinical effectiveness and safety of interventions for the treatment of newly diagnosed MB in children arising from RCTs is very limited. More data, especially from RCTs are required to fill the evidence gap on effectiveness of therapies and management of adverse events.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO255
Topic
Clinical Outcomes, Methodological & Statistical Research
Topic Subcategory
Clinical Outcomes Assessment, Comparative Effectiveness or Efficacy
Disease
Neurological Disorders, Oncology, Pediatrics