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A single centre retrospective study of low dose prophylaxis with extended half‐life factor IX for severe haemophilia B
Author(s) -
Rampotas Alexandros,
Desborough Michael J. R.,
RazaBurton Sayma,
Taylor Stephanie,
Wilkinson Alice,
Hall Georgina W,
Shapiro Susan,
Curry Nicola
Publication year - 2020
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/hae.13936
Subject(s) - haemophilia , medicine , haemophilia b , interquartile range , haemophilia a , factor ix , quality of life (healthcare) , cohort , retrospective cohort study , surgery , pediatrics , nursing
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.