%0 Journal Article
%A Kenet, Gili
%A Chen, Yeu-Chin
%A Lowe, Gillian
%A Percy, Charles
%A Tran, Huyen
%A von Drygalski, Annette
%A Trossaert, Marc
%A Reding, Mark
%A Oldenburg, Johannes
%A Mingot-Castellano, Maria Eva
%A Park, Young-Shil
%A Peyvandi, Flora
%A Ozelo, Margareth C.
%A Mahlangu, Johnny
%A Quinn, Jennifer
%A Huang, Mei
%A Reddy, Divya B.
%A Kim, Benjamin
%T 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
%D 2021
%U https://hdl.handle.net/10668/24435
%X 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 = 18 years old with severe HA (FVIII = 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.
%K haemophilia A
%K noninterventional study
%K FVIII prophylaxis
%K On-demand
%K Adults
%K Euroqol
%K Africa
%K Safety
%K Care
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