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Daily dosing prophylaxis for haemophilia: a randomized crossover pilot study evaluating feasibility and efficacy.

Lindvall, Karin LU ; Astermark, Jan LU ; Björkman, S; Ljung, R; Carlsson, K S; Persson, S and Berntorp, Erik LU (2012) In Haemophilia 18(6). p.855-859
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... (More)
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. (Less)
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Haemophilia
volume
18
issue
6
pages
855 - 859
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • wos:000310553600018
  • pmid:22681244
  • scopus:84868206189
ISSN
1351-8216
DOI
10.1111/j.1365-2516.2012.02879.x
language
English
LU publication?
yes
id
b39e1662-9cbb-4336-a006-c7ad55dc320d (old id 2859644)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/22681244?dopt=Abstract
date added to LUP
2012-07-04 13:29:38
date last changed
2017-11-12 04:05:48
@article{b39e1662-9cbb-4336-a006-c7ad55dc320d,
  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.},
  author       = {Lindvall, Karin and Astermark, Jan and Björkman, S and Ljung, R and Carlsson, K S and Persson, S and Berntorp, Erik},
  issn         = {1351-8216},
  language     = {eng},
  number       = {6},
  pages        = {855--859},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Haemophilia},
  title        = {Daily dosing prophylaxis for haemophilia: a randomized crossover pilot study evaluating feasibility and efficacy.},
  url          = {http://dx.doi.org/10.1111/j.1365-2516.2012.02879.x},
  volume       = {18},
  year         = {2012},
}