Feiba global outcome study (FEIBA-GO): first demographic data from a real world study on FEIBA in patients with inhibitors
K. Steinitz-Trost1, C. Escuriola Ettingshausen2, C. Hermans3, P. A. Holme4, C. Negrier5, S. Rangarajan6, A. Rocino7, J. Windyga8, A. Gringeri1, R. Crea1, A. R. Cid9 (1Vienna, Austria, 2Frankfurt-Moerfelden, Germany, 3Brussels, Belgium, 4Oslo, Norway, 5Bron, France, 6Basingstoke, United Kingdom, 7Naples, Italy, 8Warsaw, Poland, 9Valencia, Spain)
Bleeding disorders, coagulation and fibrinolytic factors
Time: 17:15 - 18:15
Objective: Data on FEIBA´s effectiveness, safety and quality of life are relatively poor. The FEIBA-GO study is designed to capture long-term outcomes on effectiveness, safety and health-related quality of life (HR-QoL) in subjects with severe hemophilia A receiving FEIBA in different settings in routine clinical practice. Primary objective is to describe the hemostatic effectiveness of FEIBA; most relevant secondary objectives are joint functionality outcomes, safety, HR-QoL, daily activity level, acute and chronic pain associated with hemophilia, healthcare resources used.
Methods: The study is a prospective, non-interventional, multicenter cohort study in patients with hemophilia A or B and high-responding inhibitors treated with FEIBA. A hundred subjects are targeted for enrollment and observed for 4 years; treatment regimens are at the discretion of the attending physicians in accordance with routine clinical practice, either on early, secondary or tertiary prophylaxis or on demand, including patients treated with FEIBA during immune tolerance induction.
Results: As of September 20, 2016, 40 centres have been qualified in 14 countries and 16 initiated. 29 patients have been enrolled at 14 sites in 8 countries. Data are available for 28 patients: 23 caucasians, 1 african (4 missing). At the enrolment, 21 were on prophylaxis and 7 on demand. Overall median age is 24.1 years (min-max:3-71), 17 years in patients on prophylaxis (min-max:3-71) and 39 years in those on demand (min-max:5-65). Overall median inhibitor titer at screening was 10 BU (min-max:1-2410), 7.3 BU for prophylaxis (min-max:1-92) and 15 BU in on demand (min-max:3-2410). Six subjects (4 on prophylaxis, 2 on demand) have been reported to have overall 7 target joints. Gilbert Score was assessed in 6 patients at screening. Assessment for acute pain was performed in 12 patients and for chronic pain in 9. Two of 12 patients assessed reported acute pain (both on demand). Two of the 9 patients in whom the assessment was performed reported chronic pain (treatment regimen missing).
Conclusion: This study will further enhance the information on long-term effectiveness, HR-QoL and safety of FEIBA across and beyond Europe on hemophilia A and B patients with high-responding inhibitors in routine clinical practice. Preliminary effectiveness data will be available soon.