A multicentre, prospective, randomized, open-label pragmatic trial to compare the effectiveness and safety of interferon beta-1a and glatiramer-acetate in paediatric patients affected by Multiple Sclerosis

Background: Paediatric-onset Multiple Sclerosis (POMS) is a rare, highly active disease requiring timely disease-modifying therapy. Objectives: This trial compared the efficacy and safety of intramuscular (i.m.) Interferon beta-1a (IFN-beta 1a) and subcutaneous (s.c.) Glatiramer Acetate (GA). Methods: A 96-week, Phase IIIb, multicenter, open-label randomized trial enrolled 30 participants (ages 12–17). Patients were randomized into two groups: 15 received i.m. IFN-beta 1a, and 15 received s.c. GA. The primary endpoint was MRI disease activity-free status; secondary outcomes included annualized relapse rate (ARR), time to first relapse, Expanded Disability Status Scale (EDSS), cognitive and fatigue scores, and quality of life. Results: The groups differed by age but not sex. MRI disease activity-free status did not differ (p = 0.09). ARR was significantly lower in the GA group (0.20; 95% CI 0.08–0.42) than in the IFN-beta 1a group (0.57; 95% CI 0.31–0.95, p = 0.02). EDSS, cognitive, fatigue, and quality-of-life scores were comparable. Notably, 50% of GA patients and 30% of IFN-beta 1a patients switched to higher efficacy treatments. Conclusions: Both treatments showed similar efficacy and safety, but early high-efficacy therapy may be preferable for POMS management. Trial Registration: EudraCT Number: 2017–005129-18. https://www.clinicaltrialsregister.eu/ctr-search/trial/2017-005129-18/IT