Rationale for a randomized controlled trial comparing two prophylaxis regimens in adults with severe hemophilia A: the Hemophilia Adult Prophylaxis Trial.
Rationale for a randomized controlled trial comparing two prophylaxis regimens in adults with severe hemophilia A: the Hemophilia Adult Prophylaxis Trial.
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DOI：
10.1586/ehm.11.52
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发表时间：
2011-10
影响因子：
2.8
通讯作者：
Ragni MV
中科院分区：
文献类型：
--
作者：
Ragni MV
A major goal of comprehensive hemophilia care is to prevent occurrence of bleeds by prophylaxis or regular preventive factor, one or more times weekly. Although prophylaxis is effective in reducing bleeding and joint damage in children, whether it is necessary to continue into adulthood is not known. The purpose of this article is to describe a Phase III randomized controlled trial to evaluate prophylaxis comparing two dose regimens in adults with severe hemophilia A. I hypothesize that adults with mature cartilage and joints are less susceptible to joint bleeds and joint damage, and that once-weekly recombinant factor VIII prophylaxis, with up to two rescue doses per week, is as effective as thrice-weekly prophylaxis in reducing bleeding frequency, but less costly and more acceptable, with higher quality of life. The ultimate goal of this project is to determine whether once-weekly prophylaxis is any worse than thrice-weekly prophylaxis in reducing joint bleeding frequency, while potentially utilizing less factor, at lower cost, leading to a better quality of life. This is an innovative concept, as it challenges the current paradigm of thrice-weekly prophylaxis in adults, which is based on dosing in children. Furthermore, this trial will assess interdose thrombin generation, a novel tissue factor-based assay of hemostasis, to determine if individualized thrombin generation can predict more individualized prophylaxis dosing, which would be practice changing.
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影响因子：
11.1
作者：
NILSSON, IM;BERNTORP, E;PETTERSSON, H
通讯作者：
PETTERSSON, H
DOI：
10.2106/00004623-197759030-00001
发表时间：
1977-01-01
影响因子：
5.3
作者：
ARNOLD, WD;HILGARTNER, MW
通讯作者：
HILGARTNER, MW
影响因子：
10.4
作者：
Feldman, BM;Pai, M;Blanchette, VS
通讯作者：
Blanchette, VS
影响因子：
3.9
作者：
Hay, C. R. M.
通讯作者：
Hay, C. R. M.
DOI：
10.1111/j.1365-2516.2009.02058.x
发表时间：
2009-09
期刊：
Haemophilia : the official journal of the World Federation of Hemophilia
影响因子：
--
作者：
Ragni MV;Ojeifo O;Feng J;Yan J;Hill KA;Sommer SS;Trucco MN;Brambilla DJ;Hemophilia Inhibitor Study
通讯作者：
Hemophilia Inhibitor Study