A single centre retrospective study of low dose prophylaxis with extended half‐life factor IX for severe haemophilia B

Introduction Extended half‐life factor IX concentrates (EHL‐FIX) can be administered weekly to prevent bleeding for persons with severe haemophilia B. We report the experience of a large UK haemophilia comprehensive care centre using low dose EHL‐FIX for persons with severe haemophilia B. Aim The lo...

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Bibliographic Details
Published in:Haemophilia : the official journal of the World Federation of Hemophilia 2020-03, Vol.26 (2), p.278-281
Main Authors: Rampotas, Alexandros, Desborough, Michael J. R., Raza‐Burton, Sayma, Taylor, Stephanie, Wilkinson, Alice, Hall, Georgina W, Shapiro, Susan, Curry, Nicola
Format: Article
Language:English
Subjects:
annual bleeding rate
Bleeding
bleeding disorders
Clinical trials
Coagulation factors
extended half‐life factors
factor IX
Factor IX deficiency
haemophilia
Hemophilia
Prophylaxis
Quality of life
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Summary:Introduction Extended half‐life factor IX concentrates (EHL‐FIX) can be administered weekly to prevent bleeding for persons with severe haemophilia B. We report the experience of a large UK haemophilia comprehensive care centre using low dose EHL‐FIX for persons with severe haemophilia B. Aim The low doses used in real world are approximately half of the doses used in clinical trials. We aim to assess the efficacy and safety of low dose EHL‐FIX. Methods Data from a cohort of 13 patients who were switched from standard half‐life factor IX (SHL‐FIX) to Alprolix® (mean dose 31.5 IU/kg) and seven patients who switched from standard half‐life factor IX to Idelvion® (mean dose 20.2 IU/kg) were included. Results The median annualized bleeding rate was similar for SHL‐FIX (median 3, interquartile range [IQR] 1‐5) and EHL‐FIX (median 3, IQR 1‐5.25). Quality of life scores, measured using the European Quality of Life 5 Dimensions assessment were similar for SHL‐FIX (median 0.76, IQR: 0.63‐0.84) and EHL‐FIX (median 0.79, IQR: 0.58‐0.88). Conclusion This study shows that EHL‐FIX given at low doses can be effective for prevention of bleeding for persons with severe haemophilia B.
ISSN:1351-8216
1365-2516
DOI:10.1111/hae.13936