Dataset Information

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.