Contributions
Abstract: P293
Type: Poster
Abstract Category: Clinical aspects of MS - Paediatric MS
Background: BETAPAEDIC is the first prospective, international, multicentre, observational study to assess the safety and tolerability of interferon beta-1b (IFNB-1b) in paediatric patients with relapsing-remitting MS (RRMS).
Objective: To present 2-year results from BETAPAEDIC.
Methods: Treatment-naïve patients (12-16 years) diagnosed with RRMS according to McDonald (2005) or Poser criteria and scheduled by the investigator to be treated with IFNB-1b were enrolled. Follow-up visits were planned for every 6 months for 2 years. Clinical efficacy was evaluated by annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) progression (>=1.0-step increase relative to baseline), and MRI. Neuropsychological function was measured by WISC-IV, SPM+, Beery VMI, and d2 at baseline and Year 2. Fatigue was assessed by the Fatigue Severity Scale (FSS), with scores >4 indicating fatigue. Enrollment is complete and outcomes after 2 years in patients with available data are reported.
Results: 68 patients were enrolled (mean age: 14.2 years; female:male 3.5:1). Mean time since disease onset was 0.98 years with a mean time since diagnosis of 0.52 years. 67.6% of patients were diagnosed by both McDonald and Poser criteria and 32.4% by McDonald only. In the 2 years prior to entry, 98.5% reported >=1 clinical event and EDSS progression (mean ARR: 1.1). Mean ARR in the 2-year observation period was 0.65 (n=59). Based on available data, 50.8% (30/59) had no relapses and 74.1% (40/54) of patients had no EDSS progression while 43.1% (25/58) were relapse- and progression-free from baseline to last follow-up visit. MRI scans indicated the presence of new T2 or Gd+ lesions (relative to baseline) in 77.2% (44/57) or 52.6% (30/57) of patients, respectively. 15.5% of patients with MRI assessment had NEDA-3 (no relapses, disease progression, or MRI activity [new T2 and Gd+ lesions]). Cognitive performance was within normal ranges at baseline and at the last follow-up visit. Mean total FSS score was 2.9, mean FSS VAS was 3.9 (n=29), indicating no fatigue; however, in 26 patients the mean total FSS score was >4 at some point during the study. Preliminary analyses revealed no unexpected safety signals. The most frequent adverse events were flu-like symptoms, headache, injection site reactions, and elevations of liver transaminases.
Conclusions: 2-year results from BETAPAEDIC suggest that IFNB-1b is an effective treatment with a favourable safety profile for paediatric patients.
Disclosure:
W Brück has received honoraria for lectures from Bayer Vital, Teva Pharma, Sanofi-Aventis, Genzyme, Novartis, Biogen and Merck-Serono and is member of Advisory Boards for Teva, Genzyme, Novartis and Biogen. W Brück has received research grants from Teva Pharma, Novartis and Biogen.
A Weddige has received consultancy fees from Bayer Pharma AG.
H Hummel has received honoraria from Bayer Pharma AG for a lecture.
C Norenberg is a salaried employee of Bayer Pharma AG.
JP Bugge is a salaried employee of Bayer Pharma AG.
Abstract: P293
Type: Poster
Abstract Category: Clinical aspects of MS - Paediatric MS
Background: BETAPAEDIC is the first prospective, international, multicentre, observational study to assess the safety and tolerability of interferon beta-1b (IFNB-1b) in paediatric patients with relapsing-remitting MS (RRMS).
Objective: To present 2-year results from BETAPAEDIC.
Methods: Treatment-naïve patients (12-16 years) diagnosed with RRMS according to McDonald (2005) or Poser criteria and scheduled by the investigator to be treated with IFNB-1b were enrolled. Follow-up visits were planned for every 6 months for 2 years. Clinical efficacy was evaluated by annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) progression (>=1.0-step increase relative to baseline), and MRI. Neuropsychological function was measured by WISC-IV, SPM+, Beery VMI, and d2 at baseline and Year 2. Fatigue was assessed by the Fatigue Severity Scale (FSS), with scores >4 indicating fatigue. Enrollment is complete and outcomes after 2 years in patients with available data are reported.
Results: 68 patients were enrolled (mean age: 14.2 years; female:male 3.5:1). Mean time since disease onset was 0.98 years with a mean time since diagnosis of 0.52 years. 67.6% of patients were diagnosed by both McDonald and Poser criteria and 32.4% by McDonald only. In the 2 years prior to entry, 98.5% reported >=1 clinical event and EDSS progression (mean ARR: 1.1). Mean ARR in the 2-year observation period was 0.65 (n=59). Based on available data, 50.8% (30/59) had no relapses and 74.1% (40/54) of patients had no EDSS progression while 43.1% (25/58) were relapse- and progression-free from baseline to last follow-up visit. MRI scans indicated the presence of new T2 or Gd+ lesions (relative to baseline) in 77.2% (44/57) or 52.6% (30/57) of patients, respectively. 15.5% of patients with MRI assessment had NEDA-3 (no relapses, disease progression, or MRI activity [new T2 and Gd+ lesions]). Cognitive performance was within normal ranges at baseline and at the last follow-up visit. Mean total FSS score was 2.9, mean FSS VAS was 3.9 (n=29), indicating no fatigue; however, in 26 patients the mean total FSS score was >4 at some point during the study. Preliminary analyses revealed no unexpected safety signals. The most frequent adverse events were flu-like symptoms, headache, injection site reactions, and elevations of liver transaminases.
Conclusions: 2-year results from BETAPAEDIC suggest that IFNB-1b is an effective treatment with a favourable safety profile for paediatric patients.
Disclosure:
W Brück has received honoraria for lectures from Bayer Vital, Teva Pharma, Sanofi-Aventis, Genzyme, Novartis, Biogen and Merck-Serono and is member of Advisory Boards for Teva, Genzyme, Novartis and Biogen. W Brück has received research grants from Teva Pharma, Novartis and Biogen.
A Weddige has received consultancy fees from Bayer Pharma AG.
H Hummel has received honoraria from Bayer Pharma AG for a lecture.
C Norenberg is a salaried employee of Bayer Pharma AG.
JP Bugge is a salaried employee of Bayer Pharma AG.