Hemophilia Research Today is a free monthly online journal that collates and summarizes the latest research about Hemophilia, including details on genetics, causes, symptoms, blood transfusion.

Prolonged bleeding-free period following prophylactic infusion of recombinant factor VIII reconstituted with pegylated liposomes.

Spira J, Plyushch OP, Andreeva TA, Andreev Y

Näsbyvägen 38, 135 53 Tyreso, Sweden. jack.spira@telia.com

Prophylactic treatment for hemophilia A involves infusion of factor VIII (FVIII) concentrates every 2 to 3 days. Liposomes can be efficacious vehicles for medicines, and surface modification by PEGylation can prolong liposome circulation time. When reconstituted with PEGylated liposomes (PEGLip's), recombinant FVIII binds noncovalently but with high affinity to the external liposome surface. This preparation showed prolongation of FVIII half-life and increased protection from bleeding in preclinical models. Here we report a blinded, controlled, crossover, multicenter clinical study that evaluated the time free from bleeding episodes in patients with hemophilia A during prophylaxis with standard rFVIII (no liposomes) or PEGLip rFVIII (PEGLip reconstituted) at 25 and 35 IU/kg doses. Of 24 enrolled patients, 23 were eligible for efficacy analysis. Mean number of days without bleeds was 7.2 +/- 1.7 with standard rFVIII compared with 13.3 +/- 4.8 with PEGLip rFVIII at the 35 IU/kg dose and 5.9 +/- 1.7 with standard rFVIII versus 10.9 +/- 2.9 with PEGLip rFVIII at the 25 IU/kg dose (P < .05 between treatment groups for each dose). PEGLip rFVIII was well tolerated. These data suggest that reconstitution of rFVIII with PEGLip's may reduce the frequency of treatment during prophylaxis.

Published 20 November 2006 in Blood, 108(12): 3668-73.
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