Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors.

Abstract

Factor replacement therapy for the treatment of moderate to severe haemophilia A and B can be complicated by the production of inhibitory alloantibodies to factor VIII (FVIII) or factor IX. Treatment with the nanofiltered anti-inhibitor coagulant complex, Factor Eight Inhibitor Bypassing Activity (FEIBA NF), is a key therapeutic option for controlling acute haemorrhages in patients with high-titre inhibitors or low-titre inhibitors refractory to replacement therapy. Given the high risk for morbidity and mortality in haemophilia patients with inhibitors to FVIII or FIX, we conducted this Phase 3 prospective study to evaluate whether prophylaxis with FEIBA NF is a safe and effective treatment option. Over a 1-year period, 17 subjects were treated prophylactically (85 ± 15 U kg(-1) every other day) while 19 subjects were treated on demand. The median (IQR) annualized bleeding rate (ABR) during prophylaxis was 7.9 (8.1), compared to 28.7 (32.3) during on-demand treatment, which amounts to a 72.5% reduction and a statistically significant difference in ABRs between arms (P = 0.0003). Three (17.6%) subjects (ITT) on prophylaxis experienced no bleeding episodes, whereas none treated on demand were bleeding episode-free. Total utilization of FEIBA NF for the treatment of bleeding episodes was significantly higher during on-demand therapy than prophylaxis (P = 0.0067). There were no differences in the rates of related adverse events between arms. This study demonstrates that FEIBA prophylaxis significantly reduces all types of bleeding compared with on-demand treatment, and the safety of prophylaxis is comparable to that of on-demand treatment.

• Authors: S V Antunes, S Tangada, O Stasyshyn, V Mamonov, J Phillips, N Guzman-Becerra, A Grigorian, B Ewenstein, W-Y Wong
• Journal: Haemophilia : the official journal of the World Federation of Hemophilia (Haemophilia) Vol. 20 Issue 1 Pg. 65-72 (Jan 2014) ISSN: 1365-2516 [Electronic] England
• PMID: 23910578 (Publication Type: Clinical Trial, Journal Article, Multicenter Study, Randomized Controlled Trial)
• Copyright: © 2013 John Wiley & Sons Ltd.

Chemical References

• Blood Coagulation Factor Inhibitors
• Blood Coagulation Factors
• Factor VIII
• Factor IX
• anti-inhibitor coagulant complex

Topics

• Adolescent
• Adult
• Blood Coagulation Factor Inhibitors
• Blood Coagulation Factors (administration & dosage, adverse effects, therapeutic use)
• Child
• Factor IX (administration & dosage, adverse effects, therapeutic use)
• Factor VIII (administration & dosage, adverse effects, therapeutic use)
• Hemophilia A (blood, complications, drug therapy)
• Hemophilia B (blood, complications, drug therapy)
• Hemorrhage (etiology, therapy)
• Humans
• Male
• Middle Aged
• Premedication
• Quality of Life
• Treatment Outcome
• Young Adult