1.
Daily dosing prophylaxis for haemophilia: a randomized crossover pilot study evaluating feasibility and efficacy
Lindvall K, Astermark J, Bjorkman S, Ljung R, Carlsson KS, Persson S, Berntorp E
Haemophilia. 2012;18((6):):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 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. 2012 Blackwell Publishing Ltd.
2.
A prospective, randomized trial of daily prophylaxis in hemophilia
Berntorp E, Lindvall K, Astermark J, Kampf L, Steen Carlsson K, Ljung R, Bj÷rkman S
ISTH Congress. 2009;: Abstract No. PP-WE-570.
3.
A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study
Astermark J, Donfield SM, DiMichele DM, Gringeri A, Gilbert SA, Waters J, Berntorp E, FENOC Study Group
Blood. 2007;109((2):):546-51.
Abstract
The development of inhibitory antibodies to factor VIII is a serious complication of hemophilia. FEIBA (factor VIII inhibitor-bypassing activity), an activated prothrombin complex concentrate (aPCC), and NovoSeven, recombinant factor VIIa (rFVIIa), are used as hemostatic bypassing agents in treating patients with inhibitors. The FENOC study was designed to test equivalence of the products in the treatment of ankle, knee, and elbow joint bleeding. A prospective, open-label, randomized, crossover, equivalency design was used. The parameters of interest were the percentage of patients who reported efficacy in response to FEIBA and the percentage that reported efficacy in response to NovoSeven. A difference in these percentages of no more than 15% was determined to be a clinically acceptable magnitude for equivalence of the 2 products. The primary outcome was evaluation 6 hours after treatment. Data for 96 bleeding episodes contributed by 48 participants were analyzed. The criterion for declaring the 2 products equivalent at 6 hours was not met; however, the confidence interval of the difference in percentages of efficacy reported for each product only slightly exceeded the 15% boundary (-11. 4%-15. 7%), P=. 059. FEIBA and NovoSeven appear to exhibit a similar effect on joint bleeds, although the efficacy between products is rated differently by a substantial proportion of patients. This trial was registered at www. clinicaltrials. gov as #NCT00166309.
4.
Health-economic analysis of alternative bypassing agents in haemophilia complicated by an inhibitor: the FEIBA NovoSeven comparative study (FENOC)
Carlsson KS, Astermark J, Donfield SM, Berntorp E
Blood. 2006;108((11):): Abstract No. 457.
5.
The FEIBA® NovoSeven® comparative study (FENOC) a randomized evaluation of by-passing agents in hemophilia complicated by inhibitors
Berntorp E, Donfield S, Waters J, Mattson E, DiMichele D, Gringeri A, Astermark J
Blood. 2005;106((11):): Abstract No. 324.