Phase 3 Clinical Trial: Perioperative Use of Nonacog Gamma, a Recombinant Factor IX, in Previously Treated Patients With Moderate/Severe Hemophilia B.

Abstract

Hemostatic management is essential for ensuring the safety of patients with hemophilia during surgery. This phase 3, prospective, uncontrolled trial, evaluated hemostatic efficacy, consumption, and safety of a recombinant factor IX concentrate, nonacog gamma (BAX 326, Rixubis® [Baxalta US Inc., a Takeda company, Lexington, MA, USA]), in intraoperative and postoperative settings in previously treated patients (PTPs) with severe or moderately severe hemophilia B undergoing elective surgery (N = 38 surgeries; 21 major, 17 minor). Predefined preoperative hemostatic factor IX levels (80-100% of normal for major and 30-60% for minor surgeries) were maintained for each patient. Intraoperative efficacy was rated as "excellent" or "good" for all surgeries. Postoperative hemostatic efficacy on day of discharge was rated as "excellent," "good," and "fair," respectively, for 29 (76.3%), 7 (18.4%), and 2 (5.3%) surgical procedures. All adverse events were considered unrelated to study drug; most frequently reported was mild procedural pain (9 patients). No thrombotic events, severe allergic reactions, or inhibitor formation were observed. Nonacog gamma was well tolerated and effective for intraoperative and postoperative hemostatic management of PTPs with hemophilia B.NCT01507896, EudraCT: 2011-000413-39.

• Authors: Jerzy Windyga, Margarita Timofeeva, Oleksandra Stasyshyn, Vasily Mamonov, José Luis Lamas Castellanos, Toshko Lissitchkov, Krzysztof Chojnowski, Miranda Chapman, Borislava G Pavlova, Srilatha Tangada
• Journal: Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis (Clin Appl Thromb Hemost) 2020 Jan-Dec Vol. 26 Pg. 1076029620946839 ISSN: 1938-2723 [Electronic] United States
• PMID: 32816519 (Publication Type: Clinical Trial, Phase III, Journal Article, Multicenter Study)

Chemical References

• Hemostatics
• Recombinant Proteins
• Factor IX

Topics

• Adolescent
• Adult
• Aged
• Blood Loss, Surgical (prevention & control)
• Child
• Factor IX (adverse effects, therapeutic use)
• Female
• Hemophilia B (drug therapy)
• Hemostatics (therapeutic use)
• Humans
• Male
• Middle Aged
• Perioperative Care (methods)
• Perioperative Period
• Postoperative Hemorrhage (prevention & control)
• Prospective Studies
• Recombinant Proteins (adverse effects, therapeutic use)
• Surgical Procedures, Operative (methods)
• Young Adult