Fingolimod for pediatric multiple sclerosis: The Ped-MS group systematic review and meta-analysis

Selecting the optimum disease-modifying therapy (DMT) for pediatric multiple sclerosis (Ped-MS) poses a significant challenge and requires special attention. If untreated or treated inappropriately, PED-MS can lead to cognitive impairment affecting the patient's whole life and causing irreversible sequelae.

To comprehensively review the efficacy and safety of fingolimod, the only FDA-approved DMT in Ped-MS.

Our PRISMA-based review included a systematic search of PubMed, Embase, Web of Science, and Scopus. The primary endpoint for meta-analysis was the mean difference (MD) in annualized relapse rate (ARR) after fingolimod compared to before treatment. Secondary outcomes were the MD of Expanded Disability Status Scale (EDSS) and the proportion of Ped-MS patients persisting on fingolimod.

A total of 2997 articles were identified from databases, and after screening and selection, 15 studies encompassing 585 patients were included in our systematic review. Fingolimod was associated with a significant ARR change of -1.17 relapses per patient-year (95% CI -1.76 to -0.59) from baseline. Fingolimod was not able to significantly decrease EDSS (p = 0.24) and showed a - 0.27 change from baseline (95% CI -0.86 to 0.32). After almost 24 months of fingolimod therapy, 79.8% of Ped-MS patients were still on fingolimod and did not switch their DMT. Overall adverse events ranged from 10.8% to 88%, with leukopenia/lymphopenia, headache, cough, and infections being the most reported.

Fingolimod, with a convenient oral route of administration, showed a significant effect on ARR and stabilized EDSS. Most patients remained on this DMT after 24 months, which shows relatively favorable efficacy and acceptable safety.