Dimethyl Fumarate May Be Safe, Effective for Pediatric Multiple Sclerosis
The primary end point of the study was the change in T2-hyperintense lesion incidence from baseline to the last 8 weeks of the treatment period.
According to an international, multicenter, open label, phase 2 study published in Pediatric Neurology, delayed-release dimethyl fumarate (DMF) may be used to manage pediatric relapsing-remitting multiple sclerosis (RRMS), as suggested by decreased magnetic resonance imaging activity from treatment and a favorable side effect profile.
Previous studies have shown that delayed-release DMF is safe and effective in treating RRMS in adult patients. The goal of this study was to extend these analyses to evaluate DMF in pediatric patients with RRMS.
This study included 20 patients between the ages of 10 and 17 years with RRMS. The baseline component of the study lasted 8 weeks, followed by a 24-week treatment period. All patients were treated with DMF 120 mg twice a day for week 1 of the treatment period, followed by 240 mg twice a day for the remainder of the study. Magnetic resonance imaging data were collected.
The primary end point of the study was the change in T2-hyperintense lesion incidence from baseline to the last 8 weeks of the treatment period. A significant reduction — approximately 3-fold — was found (median change -2.0; 90% CI, -8.0 to -1.5; P =.0009).
Secondary end points included pharmacokinetic parameters and adverse event incidence. Pharmacokinetics for pediatric patients (Cmax: 2.00±1.29 mg/L; daily area under the curve 7.24 hour·mg/L) were comparable to those seen in adults (Cmax: 1.87 mg/L; daily area under the curve 8.21 hour·mg/L).
Treatment-emergent adverse events included gastrointestinal symptoms, flushing, and relapse. Adverse events were reported in 91% of patients and were deemed to be caused by the study drug in 73% of those patients. No serious adverse events were reported.
“These results provide indirect support for an overlap between pediatric and adult MS in pathophysiology and DMT response,” the authors wrote. Although limited by its small sample size and absence of a control group, “[t]his study suggests DMF may prove to be a well-tolerated treatment option for pediatric patients with RRMS. Further clinical controlled studies are warranted.”
Disclosures: Funding for the study was provided by Biogen. Several authors declare various affiliations with the pharmaceutical industry.
Alroughani R, Das R, Penner N, Pultz J, Taylor C, Eraly S. Safety and efficacy of delayed-release dimethyl fumarate in pediatric patients with relapsing multiple sclerosis (FOCUS) [published online March 22, 2018]. Pediatr Neurol. doi:10.1016/j.pediatrneurol.2018.03.007