A single centre retrospective study of low dose prophylaxis with extended half-life factor IX for severe haemophilia B.
Haemophilia
; 26(2): 278-281, 2020 Mar.
Article
en En
| MEDLINE
| ID: mdl-32083769
ABSTRACT
INTRODUCTION:
Extended half-life factor IX concentrates (EHL-FIX) can be administered weekly to prevent bleeding for persons with severe haemophilia B. We report the experience of a large UK haemophilia comprehensive care centre using low dose EHL-FIX for persons with severe haemophilia B.AIM:
The low doses used in real world are approximately half of the doses used in clinical trials. We aim to assess the efficacy and safety of low dose EHL-FIX.METHODS:
Data from a cohort of 13 patients who were switched from standard half-life factor IX (SHL-FIX) to Alprolix® (mean dose 31.5 IU/kg) and seven patients who switched from standard half-life factor IX to Idelvion® (mean dose 20.2 IU/kg) were included.RESULTS:
The median annualized bleeding rate was similar for SHL-FIX (median 3, interquartile range [IQR] 1-5) and EHL-FIX (median 3, IQR 1-5.25). Quality of life scores, measured using the European Quality of Life 5 Dimensions assessment were similar for SHL-FIX (median 0.76, IQR 0.63-0.84) and EHL-FIX (median 0.79, IQR 0.58-0.88).CONCLUSION:
This study shows that EHL-FIX given at low doses can be effective for prevention of bleeding for persons with severe haemophilia B.Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Calidad de Vida
/
Factor IX
/
Hemofilia B
Tipo de estudio:
Observational_studies
Aspecto:
Patient_preference
Límite:
Adolescent
/
Adult
/
Female
/
Humans
/
Male
/
Middle aged
Idioma:
En
Revista:
Haemophilia
Asunto de la revista:
HEMATOLOGIA
Año:
2020
Tipo del documento:
Article
País de afiliación:
Reino Unido