Abstract
BackgroundCentral nervous system tumors remain the leading cause of cancer-related mortality amongst children with solid tumors, with medulloblastoma (MB) representing the most common pediatric brain malignancy. Despite best current therapies, patients with recurrent MB experience have an alarmingly high mortality rate and often have limited therapeutic options beyond inadequate chemotherapy or experimental clinical trials. Therefore, a systematic review of the literature regarding treatment strategies employed in recurrent pediatric MB will evaluate previous salvage therapies in order to guide future clinical trials. The aim of this systematic review will be to investigate the efficacy and safety of salvage therapies for the management of children with progressive, treatment-refractory, or recurrent MB.MethodsWe will conduct literature searches (from 1995 onwards) in MEDLINE, EMBASE, ClinicalTrials.gov, WHO International Clinical Trials Registry Platform, and Cochrane Central Register of Controlled Trials. Studies examining the survival and toxicity of therapies administered to treatment-refractory pediatric MB patients will be included. Two reviewers will independently assess the search results based on predefined selection criteria, complete data abstraction, and quality assessment. The primary outcomes of this review will be overall and progression-free survival. Secondary outcomes will include safety and toxicity of each therapy administered. The study methodological quality (or bias) will be appraised using an appropriate tool. Due to the nature of the research question and published literature, we expect large inter-study heterogeneity and therefore will use random effects regression analysis to extract the combined effect. In additional analyses, we will investigate the role of re-irradiation and mono- vs. poly-therapy in recurrent disease, and whether molecular subgrouping of MB influences salvage therapy.DiscussionThis systematic review will provide an overview of the current literature regarding salvage therapies for relapsed MB patients. Investigation of clinically tested therapies for children with recurrent MB has significant implications for clinical practice. By reviewing the efficacy and toxicity of MB salvage therapies, this study will identify effective therapeutic strategies administered to recurrent MB patients and can inform future clinical trials aimed to improve patient survivorship and quality of life.Systematic review registrationPROSPERO CRD42020167421
Highlights
Central nervous system tumors remain the leading cause of cancer-related mortality amongst children with solid tumors, with medulloblastoma (MB) representing the most common pediatric brain malignancy
This systematic review will provide an overview of the current literature regarding salvage therapies for relapsed MB patients
The present study aims to outline the methodology and analytical approaches that will be employed to detail a systematic review of salvage therapies for recurrent pediatric MB patients
Summary
Standard methodology of systematic reviews and metaanalyses will be conducted to ensure limited bias in study identification, selection, and extraction. Selection and data collection process Two reviewers (AAA, SKS) will independently assess the search results, conduct data abstraction, and complete quality assessment, as guided by the population, intervention, and study design eligibility criteria. A. Data items A standardized data extraction form will be used to collect relevant information from each study, which will include (but is not limited to) the following: study characteristics (i.e., citation, author details, participating center(s)), methodology (i.e., study design, randomization method, data extraction methods, quality assessment results), population (i.e., eligibility criteria, age, male/female proportion, prior treatment history), intervention (i.e., therapeutic(s) label, dosage, duration and frequency, delivery method), and outcome measures (i.e., definition, type and number of events/ type, toxicity/safety effects), as listed in Additional file 4. The finalized extraction form will be included in the meta-analysis
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