Results of a prospective multicentre open observational study of nonacog alfa in patients with severe and moderate hemophilia B: 616.151.514-056.7

Abstract

Summary. Objectives: to analyze the results of the nonacog alfa (Innonafactor) use in patients aged 12 years and older with severe and moderate hemophilia B in routine clinical practice. Patients/Methods. A prospective multicenter open, observational study included 51 patients aged 16 to 59 (32.9 ± 9.5) years. Among them 39 (76.5%) patients had severe hemophilia B and 12 (23.5%) patients had moderate hemophilia B. Treatment efficacy was assessed by the number of spontaneous bleedings occurring within 72–96 hours after nonacog alfa administration, bleeding severity and number of injections to stop one bleeding episode. Safety was assessed by the frequency and type of adverse events (AEs). Results. Forty one (63%) spontaneous and 24 (37%) post-trau- matic bleedings were registered in patients completed the study according to protocol. The spontaneous bleedings incidence was 2 (Me; IQR=6) within 72–96 hours after nonacog alfa administration. The mean prophylactic dose was 28.25±12.24 IU/kg (Me = 26.3 IU/kg; IQR = 24.4 IU/kg). About 1.9 ± 1.8 injections were required to stop a bleeding appeared during prophylactic treatment, an average single dose was of 2137 ± 790 IU (Me = 2000 IU; IQR = 1000 IU). Basing on World Federation of Hemophilia (WFH) scale the response to treatment of acute hemarthrosis was considered as “excellent” in 16 (32.0%), “good” in 31 (62.0%), and “moderate” in 3 (6.0%) for patients with prophylactic treatment, and as “good” in 10 (100%) in the on-demand treated group. AEs incidence was as 26 in 14 patients, of which only patient performed 2 AEs (dysgeusia and cough) associated directly with the nonacog alfa admin- istration. Conclusions. The results lets know nonacog alfa as effective and safe for prevention and to stop bleedings in patients with severe and moderate hemophilia B in routine clinical practice.

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