RT Journal Article
T1 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
A1 Kenet, Gili
A1 Chen, Yeu-Chin
A1 Lowe, Gillian
A1 Percy, Charles
A1 Tran, Huyen
A1 von Drygalski, Annette
A1 Trossaert, Marc
A1 Reding, Mark
A1 Oldenburg, Johannes
A1 Mingot-Castellano, Maria Eva
A1 Park, Young-Shil
A1 Peyvandi, Flora
A1 Ozelo, Margareth C.
A1 Mahlangu, Johnny
A1 Quinn, Jennifer
A1 Huang, Mei
A1 Reddy, Divya B.
A1 Kim, Benjamin
K1 haemophilia A
K1 noninterventional study
K1 FVIII prophylaxis
K1 On-demand
K1 Adults
K1 Euroqol
K1 Africa
K1 Safety
K1 Care
AB 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.
PB Mdpi
YR 2021
FD 2021-12-01
LK https://hdl.handle.net/10668/24435
UL https://hdl.handle.net/10668/24435
LA en
DS RISalud
RD Apr 6, 2025