Switching to nonacog beta pegol in hemophilia B: Outcomes from a Canadian real‐world, multicenter, retrospective study

Abstract

Background The Canadian Bleeding Disorders Registry (CBDR) captures data from 24 hemophilia treatment centers and patients directly. Nonacog beta pegol (N9‐GP) was approved in Canada in 2018. Objectives To assess treatment outcomes following switching to N9‐GP in a real‐world setting. Methods CBDR data for Canadian male patients (aged 7–72 years) with hemophilia B receiving prophylactic N9‐GP for ≥6 months as of March 31, 2021, were included. To allow comparison with the previously used products, only patients for whom data were available in the CBDR for at least 6 months before the switch to N9‐GP were included in this retrospective analysis. Results Forty‐two patients were included in the analysis (total observation period: 148.0 patient‐years). The distribution of disease severity was 62% severe, 36% moderate, 2% mild, with 62% of patients previously receiving recombinant factor IX‐Fc‐fusion protein (rFIXFc) and 38% previously receiving standard half‐life (SHL) recombinant factor IX (rFIX). During a median follow‐up period of 2.3 years on N9‐GP prophylaxis, 232 bleeds were reported in 30 patients, 29% of patients reported zero bleeds. The median overall annualized bleeding rate on N9‐GP was 0.73 for patients switching from rFIXFc (previously 1.44) and 2.10 for patients switching from SHL rFIX (previously 6.06). Median total annualized factor consumption (IU/kg) was lower with N9‐GP than with previous SHL rFIX (2152 vs 3018) and previous rFIXFc (1766 vs 2278). Conclusions Results from this first real‐world study of N9‐GP in patients with hemophilia B suggest optimal bleeding control with low factor consumption after switching to N9‐GP, irrespective of the previous product.

Pattern of use and clinical outcomes with rIX‐FP in pediatric/adolescent patients with haemophilia B in Italy: Results from IDEAL real‐world study

“…Though infusion interval was increased, prophylaxis with rIX‐FP was highly effective in bleeding prevention, as demonstrated by a low mean ABR and a 0.0 median ABR maintained across all the prophylaxis regimens. ABR reported in our study encompass also traumatic events (at least 42% of all events) and are aligned with phase III trials and real‐world evidence published involving other FIX 21–26 …”

“…ABR reported in our study encompass also traumatic events (at least 42% of all events) and are aligned with phase III trials and real-world evidence published involving other FIX. [21][22][23][24][25][26] The goal of zero bleeding events is currently considered the target of prophylaxis, 1 to avoid/delay permanent joint damage, 2,27-30 indeed, the rate of subjects with zero bleedings with rIX-FP at 2YFU was 84.6% (N = 11), compared to 69.2% (N = 9) of the previous treatment.…”