Postmarketing safety and effectiveness of recombinant factor IX (nonacog alfa) in Japanese patients with haemophilia B.

Abstract

INTRODUCTION:

In 2010, nonacog alfa became the first recombinant factor IX (rFIX) available in Japan for patients with haemophilia B.

AIM:

To determine real-world safety (adverse events, incidence of inhibitors) and effectiveness of nonacog alfa in Japan.

METHODS:

This multicentre, prospective, observational, postmarketing surveillance study enrolled previously treated and untreated patients (PTPs and PUPs, respectively) who were observed for 1 and 2 years, respectively, after initiating nonacog alfa therapy. Safety and effectiveness were assessed for each treatment type. Annualized bleeding rate (ABR) and incremental recovery of rFIX were also evaluated.

RESULTS:

Overall, 312 of 314 patients enrolled from 173 sites were eligible for the safety analysis set (PTPs, 281; PUPs, 28; other, 3). Mean age was 25.4 (PTPs) and 14.8 (PUPs) years. Haemophilic severity ranged from mild to severe, and 133 (42.6%) patients had haemophilic arthropathy. Of 285 patients (PTPs, 257; PUPs, 28) in the effectiveness set, 112 received on-demand treatment for 1161 bleeding episodes (effectiveness rate, 93.7%) and 185 received routine prophylaxis (effectiveness rate, 95.5%). No spontaneous bleeding was observed in 52.4% of patients during prophylactic treatment. Median ABR was lower during routine prophylaxis (2.0) vs the rest of the observation period (8.3). A weak negative correlation was found between body weight and the reciprocal of rFIX recovery. Eleven adverse drug reactions occurred in 7 PTPs (2.2% [7/312]); recurrence of inhibitor was observed in 1 patient, but no new inhibitor developed in PTPs or PUPs.

CONCLUSION:

Nonacog alfa therapy is safe and effective in the real-world scenario in Japan.

• Authors: Katsuyuki Fukutake, Masashi Taki, Tadashi Matsushita, Michio Sakai, Ami Takata, Hiromi Yamaguchi, Toshiyuki Karumori
• Journal: Haemophilia : the official journal of the World Federation of Hemophilia (Haemophilia) Vol. 25 Issue 4 Pg. e247-e256 (Jul 2019) ISSN: 1365-2516 [Electronic] England
• PMID: 31168882 (Publication Type: Journal Article, Observational Study)
• Copyright: © 2019 The Authors. Haemophilia Published by John Wiley & Sons Ltd.

Chemical References

• Recombinant Proteins
• Factor IX

Topics

• Adolescent
• Adult
• Aged
• Factor IX (adverse effects, therapeutic use)
• Female
• Hemophilia B (complications, drug therapy)
• Hemorrhage (complications)
• Humans
• Japan
• Male
• Middle Aged
• Product Surveillance, Postmarketing
• Recombinant Proteins (adverse effects, therapeutic use)
• Safety
• Young Adult