Blood Res.  2020 Dec;55(4):246-252. 10.5045/br.2020.2020225.

Safety and effectiveness of Rixubis in patients with hemophilia B: a real-world, prospective, postmarketing surveillance study in South Korea

Affiliations
  • 1Department of Pediatrics, Daegu Catholic University Medical Center, Daegu, Korea
  • 2Korea Hemophilia Foundation Clinic, Gwangju, Korea
  • 3Korea Hemophilia Foundation Clinic, Korea
  • 4HughChul Kim Internal Medicine Clinic, Korea
  • 5Sunchun Chung Hospital, Seoul, Korea
  • 6Hematology, Growth and Emerging Markets, Takeda Pharmaceutical International, Singapore
  • 7Global Patient Safety Evaluation, Takeda Pharmaceutical Company Ltd, Cambridge, MA, USA

Abstract

Background
Rixubis (recombinant factor IX, nonacog gamma) is indicated for the control and prevention of bleeding episodes, perioperative management, and routine prophylaxis in hemophilia B patients. This real-world, postmarketing surveillance study aimed to evaluate the safety and effectiveness of Rixubis in adult and pediatric hemophilia B patients in South Korea.
Methods
This prospective, observational, multicenter study (clinicaltrials.gov identifier: NCT029 22231) was conducted in hemophilia B patients between April 2015 and April 2019, who were observed for up to 6 months after the initiation of Rixubis treatment. Safety was evaluated based on the number and severity of adverse events (AEs) and serious AEs (SAEs). Hemostatic effectiveness was assessed by physicians and patients by using a four-point scale and rated as excellent, good, fair, or no response based on treatment type.
Results
In all, 58 patients were enrolled from four centers by seven physicians during the study period. The safety and effectiveness analysis sets included 57 and 54 patients, respectively. Overall, 11 AEs were reported in eight patients (14.0%), of which three were SAEs and occurred in three patients (5.3%). All 11 AEs were reported as unexpected and mild in severity, with no anaphylactic reaction, and 10 AEs (90.9%) resolved. The majority of AEs (10) were unrelated to Rixubis. Of the 142 hemostatic effectiveness assessments, 123 (86.6%) were reported as good or excellent.
Conclusion
Rixubis demonstrated an acceptable safety and effectiveness profile in the treatment of bleeding, perioperative management, and prophylaxis in hemophilia B patients in a real-world setting in South Korea.

Keyword

Hemophilia B; Rixubis; Safety; Effectiveness; South Korea; Bleeding

Figure

  • Fig. 1 Overview of the study population. Abbreviation: CRF, case report form.


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