Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A

  • 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.

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Verfasserangaben:Wolfgang MiesbachORCiDGND, Sabine KittlerORCiD, Artur BauhoferGND, Christoph KönigsORCiDGND, Thomas Becker, László Nemes, Alexander Staus, Jörg SchüttrumpfGND
URN:urn:nbn:de:hebis:30:3-564614
DOI:https://doi.org/10.1111/hae.13988
ISSN:1351-8216
Titel des übergeordneten Werkes (Englisch):Haemophilia
Verlag:Wiley-Blackwell
Verlagsort:Oxford [u.a.]
Dokumentart:Wissenschaftlicher Artikel
Sprache:Englisch
Datum der Veröffentlichung (online):15.04.2020
Datum der Erstveröffentlichung:15.04.2020
Veröffentlichende Institution:Universitätsbibliothek Johann Christian Senckenberg
Datum der Freischaltung:03.11.2020
Freies Schlagwort / Tag:adult and elderly patients; annual bleeding rate; effectiveness; haemophilia A; plasma-derived factor VIII concentrate, prophylaxis
Jahrgang:26
Seitenzahl:15
Erste Seite:467
Letzte Seite:477
HeBIS-PPN:477820859
Institute:Medizin / Medizin
DDC-Klassifikation:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Sammlungen:Universitätspublikationen
Lizenz (Englisch):License LogoCreative Commons - Namensnennung-Nicht kommerziell 4.0