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- Title
Real‐world data demonstrate improved bleed control and extended dosing intervals for patients with haemophilia B after switching to recombinant factor IX Fc fusion protein (rFIXFc) for up to 5 years.
- Authors
Shapiro, Amy; Chaudhury, Ateefa; Wang, Michael; Escobar, Miguel; Tsao, Elisa; Barnowski, Christopher; Feng, Jing; Jain, Nisha; Quon, Doris V.
- Abstract
Introduction: In clinical trials, recombinant factor IX fusion protein (rFIXFc) has demonstrated safety, efficacy and prolonged activity with extended dosing intervals for treatment of haemophilia B. Aim: To assess the real‐world clinical utility of rFIXFc in a variable patient population and routine clinical practice. Methods: A multicentre, retrospective chart review was conducted of patients with haemophilia B who had received rFIXFc prophylaxis or on‐demand treatment for ≥6 months across six sites in the United States. Results: Sixty‐four eligible patients were identified who had a median (range) duration on rFIXFc of 2.7 (0.5‐5.0) years. Of 32 patients on rFIXFc prophylaxis who switched from prophylaxis with another factor treatment (ie pre‐rFIXFc) and had a known pre‐rFIXFc dosing interval, the initial dosing interval was lengthened for 26 (81%) patients and maintained for the remaining 6 (19%) patients. Most (n = 48 [91%]) patients who received rFIXFc prophylaxis from the beginning to the end of the chart review period (n = 53) maintained or lengthened the dosing interval from first through last dose of rFIXFc. For patients receiving rFIXFc prophylaxis, there was an approximate 50% reduction in weekly factor consumption compared with pre‐rFIXFc prophylaxis. Overall annualized bleed rates, annualized spontaneous bleed rates and annualized joint bleed rates decreased after switching to rFIXFc prophylaxis (n = 24 with bleed data). Compliance to recommended treatment improved or remained stable in most patients with available data (30/31). Conclusion: Recombinant factor IX fusion protein prophylaxis improved bleed control, reduced overall consumption, reduced frequency of infusion and improved compliance for patients with haemophilia B in a real‐world setting.
- Subjects
UNITED States; CHIMERIC proteins; HEMOPHILIA; PATIENT compliance; BOREL sets; HEMORRHAGE
- Publication
Haemophilia, 2020, Vol 26, Issue 6, p975
- ISSN
1351-8216
- Publication type
Article
- DOI
10.1111/hae.14152