Long-term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A.
adult and elderly patients
annual bleeding rate
effectiveness
haemophilia A
plasma-derived factor VIII concentrate
prophylaxis
Journal
Haemophilia : the official journal of the World Federation of Hemophilia
ISSN: 1365-2516
Titre abrégé: Haemophilia
Pays: England
ID NLM: 9442916
Informations de publication
Date de publication:
May 2020
May 2020
Historique:
received:
15
01
2020
revised:
02
03
2020
accepted:
16
03
2020
pubmed:
16
4
2020
medline:
15
12
2020
entrez:
16
4
2020
Statut:
ppublish
Résumé
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. 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. 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. 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. 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.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
467-477Subventions
Organisme : Biotest AG
Organisme : Alnylam
Organisme : Bayer
Organisme : Biogen
Organisme : Chugai
Organisme : CSL
Organisme : Novo Nordisk
Organisme : Octapharma
Organisme : Pfizer
Organisme : Roche
Organisme : Takeda
Organisme : Sobi
Organisme : UniQure
Organisme : Biomarin
Organisme : Freeline
Organisme : Gilead
Organisme : Intersero
Organisme : Jansen
Organisme : Federal Ministry of Education
Organisme : and Research
Organisme : EU
Organisme : IMI
Organisme : FP7
Organisme : Horizon 2020
Organisme : Baxalta
Informations de copyright
© 2020 The Authors. Haemophilia published by John Wiley & Sons Ltd.
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