Fingolimod Effective for Treating Pediatric Patients with Multiple Sclerosis
Complete results from landmark phase 3 PARADIGMS clinical trial (NCT01892722) were published in the New England Journal of Medicine (September 12, 2018). The PARADIGMS trial is the first-ever completed global, controlled, randomized study for children and adolescents with relapsing-remitting multiple sclerosis (RRMS). Children and adolescents with RRMS experience more frequent and often more severe relapses than those seen in adults with MS.
Patients with RRMS, age 10-17 years (mean 15.3), were randomly assigned to receive oral fingolimod (Gilenya; Novartis, East Hanover, NJ) (0.5 mg per day or 0.25 mg per day for patients with a body weight of ≤ 40 kg; n = 107) or intramuscular injections of interferon β1a (IFNβ-1a) (30 mcg/week; n = 108) for up to 2 years. The primary end point was the annualized relapse rate.
Patients had a mean relapse rate of 2.4 relapses during the previous 2 years with adjusted annualized relapse rates of 0.12 for patients treated with fingolimod and 0.67 with IFNβ-1a, an absolute difference of 0.55 relapses and a relative difference of 82% (P < .001).
The annualized rate of new or newly enlarged lesions on T2-weighted MRI was 4.39 for patients treated with fingolimod and 9.27 for those treated with IFNβ-1a (absolute difference, 4.88 lesions; relative difference, 53%; P < .001). Serious adverse events occurred in 18 patients (16.8%) in the fingolimod group including infection (in 4 patients), leukopenia (in 2 patients), and convulsions (in 6 patients). Serious adverse events occurred in 7 patients (6.5%) in the IFNβ-1a group including infection (in 2 patients) and supraventricular tachycardia (in 1 patient).
“I’d like to thank all the children who participated in the PARADIGMS study, and their families, who have helped transform the outlook for pediatric patients living with relapsing MS,” said Dr. Tanuja Chitnis, Principal Investigator for PARADIGMS and Director of the Partners Pediatric Multiple Sclerosis Center, Massachusetts General Hospital, Boston, US, and Scientist, Ann Romney Center, Brigham and Women’s Hospital, Boston, MA.