Abstract

Regular replacement therapy (prophylaxis) for haemophilia has been shown to prevent development of disabling arthropathy and to provide a better quality of life compared to treatment on demand; however, at a substantially higher cost. Calculations based on pharmacokinetic principles have shown that shortening dose intervals may reduce cost. The aim of this prospective, randomized, crossover pilot study was to address whether daily dosing is feasible, if it reduces concentrate consumption and is as effective in preventing bleeding as the standard prophylactic dosing regimen. In a 12+12 month crossover study, 13 patients were randomized to start either their own previously prescribed standard dose, or daily dosing adjusted to maintain at least the same trough levels as obtained with the standard dose. Ten patients completed the study. A 30% reduction in cost of factor concentrates was achieved with daily prophylaxis. However, the number of bleeding events increased in some patients in the daily dosing arm and patients reported decreased quality of life during daily prophylaxis. Daily treatment had a greater impact on daily life, and the patients found it more stressful.Prophylaxis with daily dosing may be feasible and efficacious in some patients. A substantial reduction of factor consumption and costs can be realized, but larger studies are needed before the introduction of daily prophylaxis into clinical routine can be recommended.

Links

Publisher Full Text

Authors

Lindvall K, Astermark J, Björkman S, Ljung R, Carlsson KS, Persson S, Berntorp E

Institution

Malmö Centre for Thrombosis and Haemostasis, Lund University, Malmö, Sweden. karin.lindvall@med.lu.se

Source

Haemophilia : the official journal of the World Federation of Hemophilia 18:6 2012 Nov pg 855-9

MeSH

Adolescent
Adult
Child, Preschool
Cross-Over Studies
Drug Administration Schedule
Factor IX
Factor VIII
Hemophilia A
Hemophilia B
Hemorrhage
Humans
Joint Diseases
Middle Aged
Pilot Projects
Prospective Studies
Quality of Life
Young Adult

Pub Type(s)

Clinical Trial
Journal Article
Randomized Controlled Trial

Language

eng

PubMed ID

22681244