Naila Makhani1, Teri Schreiner2. 1. Department of Pediatrics, Yale University School of Medicine, New Haven, Connecticut; Department of Neurology, Yale University School of Medicine, New Haven, Connecticut. Electronic address: naila.makhani@yale.edu. 2. Department of Pediatrics, Children's Hospital Colorado, University of Colorado, Aurora, Colorado; Department of Neurology, Children's Hospital Colorado, University of Colorado, Aurora, Colorado.
Abstract
BACKGROUND: First-line injectable therapies for multiple sclerosis in children may be ineffective or not well-tolerated. There is therefore an urgent need to explore oral medications for pediatric multiple sclerosis. We review our dual-center experience with oral dimethyl fumarate. METHODS: This study was a retrospective review of children 18 years of age or less with multiple sclerosis treated with dimethyl fumarate at Yale University and the University of Colorado. Clinical, demographic, and magnetic resonance imaging parameters were analyzed. RESULTS: We identified 13 children treated with oral dimethyl fumarate for a median of 15.0 months (range, 1 to 25). Dimethyl fumarate was utilized as first-line therapy in five children (38%). Ten children (77%) tolerated dose escalation to the usual adult dose of 240 mg twice daily. Nine children had ≥12 months of follow-up on treatment. Eight of nine (89%) displayed stabilized or reduced relapse rates and disability scores on treatment. Nine children underwent brain magnetic resonance imaging performed after 12 or more months of therapy. New T2 lesions were observed in three children (33%), one of whom had been nonadherent 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 because of side effects. No patients displayed laboratory abnormalities including lymphopenia or abnormal liver transaminases. There were no reported infections. CONCLUSIONS: Oral dimethyl fumarate appears to be safe and generally well tolerated in children with multiple sclerosis. Formal clinical trials to evaluate efficacy are ongoing.
BACKGROUND: First-line injectable therapies for multiple sclerosis in children may be ineffective or not well-tolerated. There is therefore an urgent need to explore oral medications for pediatric multiple sclerosis. We review our dual-center experience with oral dimethyl fumarate. METHODS: This study was a retrospective review of children 18 years of age or less with multiple sclerosis treated with dimethyl fumarate at Yale University and the University of Colorado. Clinical, demographic, and magnetic resonance imaging parameters were analyzed. RESULTS: We identified 13 children treated with oral dimethyl fumarate for a median of 15.0 months (range, 1 to 25). Dimethyl fumarate was utilized as first-line therapy in five children (38%). Ten children (77%) tolerated dose escalation to the usual adult dose of 240 mg twice daily. Nine children had ≥12 months of follow-up on treatment. Eight of nine (89%) displayed stabilized or reduced relapse rates and disability scores on treatment. Nine children underwent brain magnetic resonance imaging performed after 12 or more months of therapy. New T2 lesions were observed in three children (33%), one of whom had been nonadherent 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 because of side effects. No patients displayed laboratory abnormalities including lymphopenia or abnormal liver transaminases. There were no reported infections. CONCLUSIONS: Oral dimethyl fumarate appears to be safe and generally well tolerated in children with multiple sclerosis. Formal clinical trials to evaluate efficacy are ongoing.
Authors: Jasna Jancic; Blazo Nikolic; Nikola Ivancevic; Vesna Djuric; Ivan Zaletel; Dejan Stevanovic; Sasa Peric; John N van den Anker; Janko Samardzic Journal: Neurol Ther Date: 2016-09-17
Authors: Kristen M Krysko; Jennifer Graves; Mary Rensel; Bianca Weinstock-Guttman; Gregory Aaen; Leslie Benson; Tanuja Chitnis; Mark Gorman; Manu Goyal; Lauren Krupp; Timothy Lotze; Soe Mar; Moses Rodriguez; John Rose; Michael Waltz; T Charles Casper; Emmanuelle Waubant Journal: Neurology Date: 2018-10-17 Impact factor: 9.910