TY  - JOUR
A1  - Miesbach, Wolfgang
A1  - Kittler, Sabine
A1  - Bauhofer, Artur
A1  - Königs, Christoph
A1  - Becker, Thomas
A1  - Nemes, László
A1  - Staus, Alexander
A1  - Schüttrumpf, Jörg
T1  - Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A
T2  - Haemophilia
N2  - Introduction: Prophylaxis with factor VIII (FVIII) concentrates in children with haemophilia A (HA) is current standard of care. The benefit of prophylactic treatment for adult HA patients is not commonly accepted.
Aim: To investigate the benefit of prophylaxis over on‐demand treatment in adult and elderly patients with severe or non‐severe HA in a real‐life setting.
Methods: Data from 163 patients comprising 1202 patient‐years were evaluated for 7.5 (±5.3) years. The effects on the annual bleeding rate (ABR, including spontaneous and traumatic bleeds) of treatment with a plasma‐derived FVIII concentrate, the patient's age and disease severity were investigated. The effect of changing the treatment from on demand to continuous prophylaxis on the patients’ ABRs was further analysed.
Results: Prophylaxis had the greatest effect on the ABRs of patients of any age with severe or non‐severe HA. The difference in ABR of all patients treated on demand (median 31.4; interquartile range (IQR) 27.6; N = 83) compared with those treated prophylactically (median 1.3; IQR 3.6; N = 122) was statistically significant (P < .05), even for patients with non‐severe HA (median 8.4; IQR 15.5; N = 11) vs median 1.5; IQR 4.2 (N = 17), P < .05). Patients, aged up to 88 years, switching from on demand to continuous prophylaxis showed the lowest median ABR (1.1; N = 51) after their regimen change.
Conclusion: Any (even low‐frequency) prophylaxis results in lower ABR than on‐demand treatment. Patients switching to prophylaxis benefitted the most, irrespective of age or HA severity. Prophylactic treatment—even tertiary—is the regimen of choice for patients of any age, including elderly patients, with severe or non‐severe HA.
KW  - adult and elderly patients
KW  - annual bleeding rate
KW  - effectiveness
KW  - haemophilia A
KW  - plasma-derived factor VIII concentrate, prophylaxis
Y1  - 2020
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/56461
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-564614
SN  - 1351-8216
VL  - 26
SP  - 467
EP  - 477
PB  - Wiley-Blackwell
CY  - Oxford [u.a.]
ER  -