Abstract Number: PB1020
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Current standard of care treatment for severe hemophilia A and B (SHA and SHB) is 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.
Aims: 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%, in the era of standard half-life products.
Methods: Data was retrospectively collected from hemophilia-specific patient diaries used for home treatment, medical records and entries into the Austrian Hemophilia Registry from year 2012 to 2017, a time-period where extended-half life products were not routinely used.
Results: 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 on-demand therapy. A detailed description of the study cohort and study results is shown in Table 1 and Table 2. 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 and 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. On-demand therapy and hepatitis B and C were associated with higher ABR, but not age, weight and HIV positivity.
Conclusions: Bleeding rates and 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.
| Severe Hemophilia A (n=47) | Severe Hemophilia B (n=6) | |
| Median (IQR) age, years | 38 (30-47) | 38 (28.3-58.3) |
| Median (IQR) height, cm | 178.5 (173.8-181.0) | 176.5 (167.8-180.3) |
| Median (IQR) weight, kg | 79 (69-90) | 72.5 (68.8-84.8) |
| Median (IQR) body mass index (BMI) | 24.7 (2.6-28.1) | 23.8 (22.1-27.2) |
| Median (IQR) observation time, days | 1096 (744-1461) | 1374 (365-1552) |
| Median (IQR) number of infusions (i.e. exposure days) during observation per patient | 325 (114-471) | 294 (107-360) |
[Table 1: Description of the study cohort and demographics]
| Severe Hemophilia A (n=47) | Severe Hemophilia B (n=6) | |
| Treatment regimen Prophylaxis, n (%) |
26 (55.3) |
5 (83.3) |
| On-Demand, n(%) | 13 (27.7 | 0 (0) |
| “Switcher”-group, n (%) | 8 (17.0) | 1 (16.7) |
| Prescribed factor dosage in prophylaxis Median (IQR) IU/kg/week |
n = 31*
66.7 (53.3-87.0) |
n = 6
40.1 (30.9-57.3) |
| Mean (±SD) IU/kg/week | 71.7 (±23.4) | 42.5 (±12.9) |
| Factor product type, n (%) Recombinant |
35 (74.5) |
2 (33.3) |
| Plasma-derived | 12 (25.5 | 4 (66.7) |
| Median (IQR) factor consumption (IU/year) | 190051 (89250-254892) | 143655 (109662-223353) |
| *For prescribed factor dosage in the prophylaxis group, n is lower as this value could only be calculated for subjects who were on prophylaxis at some point in the observation period. This includes all subjects with SHA on prophylaxis and 5 subjects from the “Switcher”-group who were prescribed a defined prophylactic regimen. For 3 subjects of the switcher-group” with SHA, prescribed weekly prophylactic infusions were unknown. | ||
[Table 2: Treatment patterns and factor utilization in patients with severe hemophilia A and B]
To cite this abstract in AMA style:
Perschy L, Rejtö J, Pabinger I, Ay C, Kaider A. Treatment Patterns and Bleeding Outcomes in Persons with Severe Hemophilia A and B in a Real-World Setting [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/treatment-patterns-and-bleeding-outcomes-in-persons-with-severe-hemophilia-a-and-b-in-a-real-world-setting/. Accessed September 29, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/treatment-patterns-and-bleeding-outcomes-in-persons-with-severe-hemophilia-a-and-b-in-a-real-world-setting/