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Abstract
Background:
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).
Methods:
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.
Results:
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.
Conclusions:
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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