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First-line injectable therapies for multiple sclerosis (MS) in children may be ineffective or not well-tolerated. There is therefore an urgent need to explore oral medications for pediatric MS. We review our dual-center experience with oral dimethyl fumarate (DMF).
Retrospective chart review of children with MS < 18 years treated with DMF at Yale University and the University of Colorado. Clinical, demographic, and MRI parameters were analyzed.
We identified 13 children treated with oral DMF for a median of 15.0 months (range 1-25). DMF was utilized first-line in five children (38%). Ten children (77%) tolerated dose escalation to usual adult dose of 240 mg BID. Nine children had > 12 months of follow-up on treatment. Eight of nine (89%) displayed either stabilized or reduced relapse rates and disability scores on treatment. Nine children had brain MR imaging performed after > 12 months of therapy. New T2 lesions were observed in three children (33%), one of whom had been non-adherent to treatment. Common side effects included facial flushing (8/13, 62%), gastrointestinal discomfort (7/13, 54%), rash (3/13, 23%), and malaise (2/13, 15%). Three children (23%) discontinued treatment due to side effects. No patients displayed laboratory abnormalities including lymphopenia or abnormal liver transaminases. There were no reported infections.
Oral DMF appears to be safe and generally well-tolerated in children with MS. Formal clinical trials to evaluate efficacy are ongoing.
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