Final results from the BETAPAEDIC study of interferon beta-1b for treatment-naïve pediatric patients with relapsing-remitting MS (P1.346)

Objective: To present final results from the post-authorization safety study BETAPAEDIC Background: BETAPAEDIC is the first prospective, international, multicenter, observational study to assess the tolerability and effectiveness of interferon beta-1b (IFNB-1b) in pediatric patients with relapsing-remitting MS (RRMS). Design/Methods: Treatment-naive patients (12–16 years) diagnosed with RRMS who started treatment with IFNB-1b were enrolled. Follow-up visits were planned for 6, 12, 18, and 24 months. Tolerability was evaluated as the proportion of patients with adverse events (AEs). Effectiveness was evaluated by annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) progression (>=1.0-step increase from baseline), and MRI. Neuropsychological function was measured by WISC-IV, SPM+, Beery VMI, and d2. Fatigue was assessed by the Fatigue Severity Scale (FSS). Results: 68 patients were enrolled (mean age: 14.2 years; 50 females). Mean time since disease onset was 11 months. At baseline, mean (SD) EDSS was 0.6 (1.0) and number of T2 lesions was 18.3 (15.1). Mean ARR in the 2-year observation period was 0.7 (n=57). 49.1% (28/57) had no relapse and 76.9% (40/52) of patients had no EDSS progression while 41.1% (23/56) were relapse- and progression-free to last follow-up visit. New T2 or Gd+ lesions were seen in 43 and 29 patients, respectively, out of 55 with data. 10.7% (6/56) of patients with MRI assessment had NEDA-3 (no relapses, disease progression, or MRI activity). Cognitive performance was within normal ranges at baseline and last follow-up. Mean total FSS score was 3.0 (n=29); 31.6% of patients (18/57) had a total score >4 at some point. 76% of patients (51/67) reported drug-related AEs. The most frequent drug-related AEs were flu-like symptoms (46.3%), headache (19.4%), injection site reactions (16.4%), and abnormal liver function tests (11.9%). Conclusions: Final results from BETAPAEDIC suggest that IFNB-1b is an effective treatment with a favorable tolerability profile for pediatric patients with RRMS. Study Supported by: Bayer Healthcare Disclosure: Dr. Gaertner has received personal compensation from Bayer Vital, Biogen, Merck Serono, Teva, and Novartis as a consultant and for honoraria. Dr. Gaertner has received research support from Novartis and Biogen. Dr. Brueck has received personal compensation for activities with Bayer Vital, Biogen, Merck Serono, Teva Pharma, Genzyme, Sanofi Aventis, and Novartis as a lecturer or member of scientific advisory boards. Dr. Weddige has received personal compensation for activities with Bayer Pharma AG. Dr. Hummel has received personal compensation for activities with Bayer Pharma AG as an speaker. Dr. Norenberg has received personal compensation for activities with Bayer Pharma AG. Dr. Bugge has received personal compensation for activities with Bayer Pharma AG as an employee.