Long-term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A.
Haemophilia
; 26(3): 467-477, 2020 May.
Article
em En
| MEDLINE
| ID: mdl-32293085
ABSTRACT
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.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Hemofilia A
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article