Dimethyl fumarate bests interferon β-1a in paediatric-onset multiple sclerosis

The use of dimethyl fumarate (DMF) in the treatment of children with paediatric-onset multiple sclerosis (POMS) appears to produce more favourable effects than interferon β-1a (IFNβ-1a), with those on DMF being less likely to have new or newly enlarging lesions and experience relapse, according to data from the open-label CONNECT study.

The 96-week CONNECT trial randomized 150 patients with POMS (median age 15 years, 67.3 percent female) to receive either DMF (n=78) or IFNβ-1a (n=72). Patients on DMF received treatment at 120 mg orally twice daily for the first 7 days and 240 mg twice daily thereafter. Patients in the active-control group self-administered (or administered via proxy) IFNβ-1a 7.5 μg once by intramuscular injection on day 1, with subsequent doses increased by 7.5 μg each week for 3 weeks until the recommended dose of 30 μg once weekly was achieved.

At week 96, the primary endpoint of the proportion of patients free of new or newly enlarging (N or NE) T2 hyperintense lesions among 103 trial completers was greater in the DMF than in the IFNβ-1a group (16.1 percent, 95 percent confidence interval [CI], 8.0–27.7 vs 4.9 percent, 95 percent CI, 0.6–16.5). The result was consistent in a sensitivity analysis (12.8 percent vs 2.8 percent).

The estimated proportion of patients who remained relapse free at week 96 was 66.2 percent in the DMF group and 52.3 percent in the IFNβ-1a group. The corresponding adjusted annualized relapse rates were 0.24 (95 percent CI, 0.15–0.39) and 0.53 (95 percent CI, 0.33–0.84).

Compared with IFNβ-1a, DMF significantly lowered the risk of relapse (rate ratio, 0.46, 95 percent CI, 0.26–0.80; p=0.006).

The number of treatment-emergent adverse events (TEAEs; 74 patients [94.9 percent] vs 69 patients [95.8 percent]), serious TEAEs (18 patients [23.1 percent] vs 21 patients [29.2 percent]), and treatment discontinuations due to TEAEs (5 patients [6.4 percent] vs 8 patients [11.1 percent]) was similar for DMF vs IFNβ-1a.