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.