Alexandros Rampotas1, Michael J R Desborough2,3,4, Sayma Raza-Burton5,6, Stephanie Taylor5,6, Alice Wilkinson7, Georgina W Hall7, Susan Shapiro5,6, Nicola Curry5,6. 1. NIHR Academic Clinic Fellow in Haematology, Haematology Department, Oxford University Hospitals NHS Foundation Trust, Oxford, UK. 2. Oxford Biomedical Research Centre, Oxford Haemophilia and Thrombosis Centre, Oxford, UK. 3. Nuffield Division of Clinical Laboratory Sciences, Oxford Clinical Research in Transfusion Medicine, University of Oxford, Oxford, UK. 4. Haemophilia and Thrombosis Centre, St Thomas' Hospital, London, UK. 5. Oxford Haemophilia and Thrombosis Centre, Churchill Hospital, Oxford, UK. 6. NIHR BRC Blood Theme, Oxford Centre for Haematology, Oxford University, Oxford, UK. 7. Paediatric Haemophilia centre, Children's Hospital, John Radcliffe hospital, Headington, UK.
Abstract
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.
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.
Authors: Mairead O'Donovan; Eimear Quinn; Kate Johnston; Evelyn Singleton; Julie Benson; Brian O'Mahony; Declan Noone; Cleona Duggan; Ruth Gilmore; Kevin Ryan; James S O'Donnell; Niamh M O'Connell; Johnny Mahlangu Journal: Res Pract Thromb Haemost Date: 2021-10-11
Authors: Daniel P Hart; Davide Matino; Jan Astermark; Gerard Dolan; Roseline d'Oiron; Cédric Hermans; Victor Jiménez-Yuste; Adriana Linares; Tadashi Matsushita; Simon McRae; Margareth C Ozelo; Sean Platton; Darrel Stafford; Robert F Sidonio; Andreas Tiede Journal: Ther Adv Hematol Date: 2022-04-02