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

Authors: Kenet, G; Chen, Y-C; Lowe, G; Percy, C; Tran, H; Drygalski, A von; Trossaert, 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

Affiliations: Tel Aviv Univ, Sheba Med Ctr, Natl Hemophilia Ctr, Amalia Biron Res Inst Thrombosis & Hemostasis, IL-52621 Tel Aviv, Israel; Triserv Gen Hosp, Natl Def Med Ctr, Haemophilia Care & Res Ctr, Taipei 11490, Taiwan; Queen Elizabeth Hosp, West Midlands Comprehens Care Haemophilia Ctr, Birmingham B15 2TH, W Midlands, England; Alfred Hosp, Haemostasis & Thrombosis Unit, Haemophilia Treatment Ctr, Melbourne, Vic 3004, Australia; Scripps Res Inst, Dept Mol Med, La Jolla, CA 92037 USA; CHU Nantes, Ctr Reg Traitement Hemophiles, F-44093 Nantes, France; Univ Minnesota, Ctr Bleeding & Clotting Disorders, Minneapolis, MN 55454 USA; Univ Hosp Bonn, Ctr Rare Dis, D-53127 Bonn, Germany; Univ Hosp Bonn, Inst Expt Haematol & Transfus Med, D-53127 Bonn, Germany; Reg Univ Hosp, Malaga 29010, Spain; Kyung Hee Univ Hosp Gangdong, Seoul 134727, South Korea; Fdn IRCCS Ca Granda Osped Maggiore Policlin, Angelo Bianchi Bonomi Hemophilia & Thrombosis Ctr, I-20122 Milan, Italy; Fdn Luigi Villa, I-20122 Milan, Italy; Univ Milan, Dept Pathophysiol & Transplantat, I-20122 Milan, Italy; Univ Estadual Campinas, Sch Med Sci, Dept Internal Med, Hemoctr UNICAMP, BR-13083878 Campinas, SP, Brazil; Univ Witwatersrand, Charlotte Maxeke Johannesburg Acad Hosp, Hemophilia Comprehens Care Ctr, ZA-2193 Johannesburg, South Africa; BioMarin Pharmaceut UK Ltd, London WC1A 2SL, England; BioMarin Pharmaceut Inc, Novato, CA 94949 USA.

Publication: Journal of Clinical Medicine ; 2021 ; 10 January 2022

Abstract: Regular prophylaxis with exogenous factor VIII (FVIII) is recommended for individuals with severe haemophilia A (HA), but standardized 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. Annualized bleeding rate (ABR) and FVIII utilization 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) annualized FVIII utilization rates were 3629.0 (1008.5-13541.7) and 3708.0 (1311.0-14633.4) IU/kg/year, respectively; and the median (range) annualized 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.