Efficacy and safety of a recombinant factor IX (Bax326) in previously treated patients with severe or moderately severe haemophilia B undergoing surgical or other invasive procedures: a prospective, open-label, uncontrolled, multicentre, phase III study

Haemophilia. 2014 Sep;20(5):651-8. doi: 10.1111/hae.12419. Epub 2014 Apr 3.

Abstract

Haemostatic management of haemophilia B patients undergoing surgery is critical to patient safety. The aim of this ongoing prospective trial was to investigate the haemostatic efficacy and safety of a recombinant factor IX (rFIX) (Bax326) in previously treated subjects (12-65 years, without history of FIX inhibitors) with severe or moderately severe haemophilia B, undergoing surgical, dental or other invasive procedures. Haemostatic efficacy was assessed according to a predefined scale. Blood loss was compared to the average and maximum blood loss predicted preoperatively. Haemostatic FIX levels were achieved peri- and postoperatively in 100% of subjects (n = 14). Haemostasis was 'excellent' intraoperatively in all patients and postoperatively in those without a drain, and 'excellent' or 'good' at the time of drain removal and day of discharge in those with a drain employed. Following the initial dose, the mean FIX activity level rose from 6.55% to 107.58% for major surgeries and from 3.60% to 81.4% for minor surgeries. Actual vs. predicted blood loss matched predicted intraoperative blood loss but was equal to or higher than (but less than 150%) the maximum predicted postoperative blood loss reflecting the severity of procedure and FIX requirements. There were no related adverse events, severe allergic reactions or thrombotic events. There was no evidence that BAX326 increased the risk of inhibitor or binding antibody development to FIX. BAX326 was safe and effective for peri-operative management of 14 subjects with severe and moderately severe haemophilia B.

Keywords: haemophilia B; previously treated patients; rFIX (Rixubis); surgical procedures.

Publication types

  • Clinical Trial, Phase III
  • Controlled Clinical Trial
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Blood Coagulation Factor Inhibitors / immunology
  • Blood Loss, Surgical / prevention & control*
  • Case-Control Studies
  • Child
  • Coagulants / adverse effects
  • Coagulants / therapeutic use*
  • Factor IX / adverse effects
  • Factor IX / therapeutic use*
  • Female
  • Hemophilia B / drug therapy*
  • Hemophilia B / immunology
  • Hemorrhage / prevention & control
  • Hemostasis, Surgical / methods*
  • Humans
  • Male
  • Middle Aged
  • Perioperative Care
  • Postoperative Care
  • Prospective Studies
  • Recombinant Proteins / adverse effects
  • Recombinant Proteins / therapeutic use
  • Surgical Procedures, Operative / methods*
  • Young Adult

Substances

  • Blood Coagulation Factor Inhibitors
  • Coagulants
  • Recombinant Proteins
  • Factor IX