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Daily dosing prophylaxis for haemophilia: A randomized crossover pilot study evaluating feasibility and efficacy
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Pharmacy, Department of Pharmaceutical Biosciences. (translationell pkpd)
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2012 (English)In: Haemophilia, ISSN 1351-8216, E-ISSN 1365-2516, Vol. 18, no 6, 855-859 p.Article in journal (Refereed) Published
Abstract [en]

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.

Place, publisher, year, edition, pages
2012. Vol. 18, no 6, 855-859 p.
Keyword [en]
Factor VIII/IX, Haemophilia, Prophylaxis, QoL
National Category
Medical and Health Sciences
URN: urn:nbn:se:uu:diva-184943DOI: 10.1111/j.1365-2516.2012.02879.xISI: 000310553600018OAI: oai:DiVA.org:uu-184943DiVA: diva2:570408
Available from: 2012-11-19 Created: 2012-11-15 Last updated: 2012-12-10Bibliographically approved

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