A Systematic Review of Dimethyl Fumarate (DMF) in Pediatric Patients with Multiple Sclerosis (MS)
Amr Mahmoud Yousef1, Sara Hosny El-Farargy1, Ammar Elsayed1, Ahmed Mamdoh Mahmoud1, Hossam Tharwat Ali2, Mostafa Meshref3, Ahmed M. Afifi4
1Benha Faculty of Medicine, Benha University, Benha, Egypt, 2Qena Faculty of Medicine, South Valley University, Qena, Egypt, 3Department of Neurology, Faculty of Medicine, Al-Azhar university, Cairo, 4University of Toledo Medical Center, Toledo, USA
Objective:
We conducted a review of the clinical trials assessing the efficacy and/or safety of DMF in MS pediatric patients.
Background:
Pediatric MS is rare but associated with cognitive decline, disabilities, and higher rates of relapse. There are limited approved options for pediatric MS with only fingolimod approved in 2018 by the FDA and teriflunomide by the EMA in 2021. DMF is an oral disease-modifying medication approved for adults with MS but there is limited data regarding its use in pediatric patients.
Design/Methods:
We conducted a systematic search of PubMed, Cochrane, Scopus, and Web of Science databases until October 2024 using the relevant keywords. Two authors independently screened the results, and a third author resolved the conflicts. We extracted data on patients’ demographics (age, gender), MS-related data (disease duration, type, EDSS, relapse rate, and MRI lesions), control group, outcome measures, and the main findings of the study.
Results:
We included three reports from two distinct clinical trials with a total of 172 patients aged 10-17 (66.8% females) and administered DMF 240mg BID. The first 24-week single-arm trial showed a reduction of new or newly enlarging T2 hyperintense lesions with DMF. A 96-week extension of this trial showed continued decrease in relapses and an 84.5% relative reduction of the annualized relapse rate (ARR). The recent randomized clinical trial revealed a reduced number of T2 lesions, relapses, and ARR with DMF compared to interferon beta (IFN-β). All studies showed that DMF was well-tolerated with gastrointestinal symptoms being the commonest adverse events.
Conclusions:
Despite the scarcity of trials, DMF was efficacious in improving clinical and imaging parameters and it was well-tolerated. The long-term efficacy and safety of DMF in pediatric patients were consistent with adults which suggests DMF as potential option in pediatric patients with MS.
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