Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a real-world setting
- PDF / 506,068 Bytes
- 9 Pages / 595.276 x 790.866 pts Page_size
- 10 Downloads / 158 Views
ORIGINAL ARTICLE
Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a real-world setting Cihan Ay 1
&
Leonard Perschy 1 & Judit Rejtö 1 & Alexandra Kaider 2 & Ingrid Pabinger 1
Received: 6 March 2020 / Accepted: 1 September 2020 # The Author(s) 2020
Abstract The current standard of care treatment for severe hemophilia A and B (SHA and SHB) is the prophylactic intravenous replacement of coagulation factor VIII or IX (FVIII/FIX) to prevent spontaneous bleeding. Persons with hemophilia without prophylactic treatment receive therapy in case of bleeding, i.e., on demand. To assess treatment patterns, utilization of products, and bleeding outcomes in a real-world cohort of persons with SHA and SHB, defined as FVIII or FIX activity < 1%, data was retrospectively collected from hemophilia-specific patient diaries used for home treatment, medical records, and entries into the Austrian Hemophilia Registry from the year 2012 to 2017. Fifty-three male persons with SHA (n = 47) and SHB (n = 6) were included; 26 with SHA and 5 with SHB were on prophylaxis, 8 and 1 switched therapy regimen, and 13 and 0 received ondemand therapy. Persons on prophylaxis used a mean factor FVIII or FIX dose of 71.7 and 40.1 IU/kg/week. Median (IQR) annualized bleeding rates (ABR) in SHA were 28.0 (23.4–31.3) in the on-demand, 4.9 (1.6–13.5) in the prophylaxis group, and 3.0 (2.0–6.8) in the prophylactic group of SHB. Three persons with SHA had zero bleeds during the observation period. Ondemand therapy and hepatitis B and C were associated with higher ABR but not age, weight, and HIV positivity. Bleeding rates and the proportion of on-demand therapy in persons with hemophilia were high in our real-world cohort. Further improvement is needed, which might be facilitated with the advent of factor products with extended half-life or non-factor therapies. Keywords Hemophilia A . Hemophilia B . Factor VIII . Factor IX . Hemorrhage . Half-life
Introduction Hemophilia, a rare bleeding disorder with X-chromosomal recessive inheritance pattern, is characterized by the deficiency of coagulation factor VIII (FVIII) in the case of hemophilia A or IX (FIX) in the case of hemophilia B. Severe hemophilia (defined as factor activity < 1%) is associated with a high risk of spontaneous bleeding, mostly affecting joints.
Electronic supplementary material The online version of this article (https://doi.org/10.1007/s00277-020-04250-9) contains supplementary material, which is available to authorized users. * Cihan Ay [email protected] 1
Clinical Division of Haematology and Haemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria
2
Center for Medical Statistics, Informatics and Intelligent Systems, Medical University of Vienna, Vienna, Austria
To prevent bleeding in severe hemophilia A and B (SHA and SHB), treatment with regular factor concentrate infusions is required [1–3]. In clinical practice, prophylaxis is frequently individualized based on bleeding phenotype, lifestyle and level
Data Loading...
