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Article

Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study

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The National Hemophilia Center, Amalia Biron Research Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel Aviv University, Tel Hashomer, Tel Aviv-Yafo 52621, Israel
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Haemophilia Care and Research Center, Tri-Service General Hospital, National Defense Medical Center, Taipei 11490, Taiwan
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West Midlands Comprehensive Care Haemophilia Centre, Queen Elizabeth Hospital, Birmingham B15 2TH, UK
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Haemophilia Treatment Centre, Haemostasis & Thrombosis Unit, The Alfred Hospital, Melbourne, VIC 3004, Australia
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Department of Molecular Medicine, Scripps Research Institute, La Jolla, CA 92037, USA
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Centre Régional de Traitement des Hémophiles, CHU de Nantes, 44093 Nantes, France
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Center for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis, MN 55454, USA
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Center for Rare Diseases and Institute of Experimental Haematology and Transfusion Medicine, University Hospital Bonn, 53127 Bonn, Germany
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Regional University Hospital, Carlos Haya, 29010 Málaga, Spain
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Kyung Hee University Hospital at Gangdong, Seoul 134-727, Korea
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Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, 20122 Milan, Italy
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Department of Pathophysiology and Transplantation, Università degli Studi di Milano, 20122 Milan, Italy
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Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas 13083-878, SP, Brazil
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Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg 2193, South Africa
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BioMarin Pharmaceutical UK Ltd., London WC1A 2SL, UK
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BioMarin Pharmaceutical Inc., Novato, CA 94949, USA
*
Author to whom correspondence should be addressed.
Academic Editor: Paul Monahan
J. Clin. Med. 2021, 10(24), 5959; https://doi.org/10.3390/jcm10245959
Received: 12 November 2021 / Revised: 13 December 2021 / Accepted: 15 December 2021 / Published: 18 December 2021
(This article belongs to the Special Issue Haemophilia: Current Treatment and Challenges)
Regular prophylaxis with exogenous factor VIII (FVIII) is recommended for individuals with severe haemophilia A (HA), but standardised data are scarce. Here, we report real-world data from a global cohort. Participants were men ≥18 years old with severe HA (FVIII ≤ 1 IU/dL) receiving regular prophylaxis with FVIII. Participants provided 6 months of retrospective data and were prospectively followed for up to 12 months. Annualised bleeding rate (ABR) and FVIII utilisation and infusion rates were calculated. Differences between geographic regions were explored. Of 294 enrolled participants, 225 (76.5%) completed ≥6 months of prospective follow-up. Pre-baseline and on-study, the median (range) ABR values for treated bleeds were 2.00 (0–86.0) and 1.85 (0–37.8), respectively; the median (range) annualised FVIII utilisation rates were 3629.0 (1008.5–13541.7) and 3708.0 (1311.0–14633.4) IU/kg/year, respectively; and the median (range) annualised FVIII infusion rates were 120.0 (52.0–364.0) and 122.4 (38.0–363.8) infusions/year, respectively. The median (range) Haemo-QoL-A Total Score was 76.3 (9.4–100.0) (n = 289), ranging from 85.1 in Australia to 67.7 in South America. Physical Functioning was the most impacted Haemo-QoL-A domain in 4/6 geographic regions. Despite differences among sites, participants reported bleeding requiring treatment and impaired physical functioning. These real-world data illustrate shortcomings associated with FVIII prophylaxis for this global cohort of individuals with severe HA. View Full-Text
Keywords: haemophilia A; noninterventional study; FVIII prophylaxis haemophilia A; noninterventional study; FVIII prophylaxis
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MDPI and ACS Style

Kenet, G.; Chen, Y.-C.; Lowe, G.; Percy, C.; Tran, H.; von Drygalski, A.; Trossaërt, M.; Reding, M.; Oldenburg, J.; Mingot-Castellano, M.E.; Park, Y.-S.; Peyvandi, F.; Ozelo, M.C.; Mahlangu, J.; Quinn, J.; Huang, M.; Reddy, D.B.; Kim, B. Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study. J. Clin. Med. 2021, 10, 5959. https://doi.org/10.3390/jcm10245959

AMA Style

Kenet G, Chen Y-C, Lowe G, Percy C, Tran H, von Drygalski A, Trossaërt M, Reding M, Oldenburg J, Mingot-Castellano ME, Park Y-S, Peyvandi F, Ozelo MC, Mahlangu J, Quinn J, Huang M, Reddy DB, Kim B. Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study. Journal of Clinical Medicine. 2021; 10(24):5959. https://doi.org/10.3390/jcm10245959

Chicago/Turabian Style

Kenet, Gili, Yeu-Chin Chen, Gillian Lowe, Charles Percy, Huyen Tran, Annette von Drygalski, Marc Trossaërt, Mark Reding, Johannes Oldenburg, Maria E. Mingot-Castellano, Young-Shil Park, Flora Peyvandi, Margareth C. Ozelo, Johnny Mahlangu, Jennifer Quinn, Mei Huang, Divya B. Reddy, and Benjamin Kim. 2021. "Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study" Journal of Clinical Medicine 10, no. 24: 5959. https://doi.org/10.3390/jcm10245959

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