Daily dosing prophylaxis for haemophilia: a randomized crossover pilot study evaluating feasibility and efficacy.
(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:
https://lup.lub.lu.se/record/2859644
- author
- Lindvall, Karin LU ; Astermark, Jan LU ; Björkman, S ; Ljung, R ; Carlsson, K S ; Persson, S and Berntorp, Erik LU
- organization
- publishing date
- 2012
- type
- Contribution to journal
- publication status
- published
- subject
- in
- Haemophilia
- volume
- 18
- issue
- 6
- pages
- 855 - 859
- publisher
- Wiley-Blackwell
- external identifiers
-
- wos:000310553600018
- pmid:22681244
- scopus:84868206189
- pmid:22681244
- 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
- 2016-04-04 07:05:54
- date last changed
- 2022-05-28 01:23:44
@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 = {{Wiley-Blackwell}}, 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}}, doi = {{10.1111/j.1365-2516.2012.02879.x}}, volume = {{18}}, year = {{2012}}, }