The WFH is in a unique position to be able to play a distinct and supportive role in developing new global research initiatives for these conditions. We look forward with keen anticipation to further research successes in the inherited bleeding disorder community. The author is the recipient of a Canada Research Chair in Molecular Hemostasis. His research

program is supported EPZ-6438 nmr by the Canadian Institutes of Health Research, the Heart and Stroke Foundation of Canada, the Canadian Hemophilia Society and the US National Institutes of Health. The author receives grants from Bayer, Baxter, Biogen-Idec and CSL-Behring. “
“Summary.  Congenital factor XIII (FXIII) deficiency is an extremely rare, yet potentially life-threatening, bleeding disorder, with a 30% rate of spontaneous intracranial haemorrhage. Routine prophylactic management is recommended for all individuals with clinically relevant (FXIII) deficiency and for all symptomatic individuals with congenital factor deficiency. Fibrogammin® P is a purified, pasteurized concentrate of FXIII that appears to carry negligible risk of viral transmission, AZD2014 datasheet unlike other unprocessed products containing FXIII. An ongoing Phase II/III study of Fibrogammin® P in patients with congenital FXIII deficiency is

being conducted to evaluate the prophylactic efficacy and long-term safety of this product. Using retrospective chart review data from subjects enrolled in the Phase II/III study, the current analysis was designed to compare spontaneous bleed-event rates prior to and after the initiation of Fibrogammin® P prophylaxis. Seven subjects

were evaluable for comparison, having received no other prophylactic FXIII-containing product during the 24 months prior to study entry. The mean annual number of spontaneous bleeds was 2.5 events per year prior to Fibrogammin® Mannose-binding protein-associated serine protease P prophylaxis and 0.2 events per year during Fibrogammin® P prophylaxis (P = 0.01). Patients reported no severe bleeds during Fibrogammin® P therapy. This small sample supports a consistent and clinically meaningful reduction in spontaneous bleeding with prophylactic use of Fibrogammin® P. “
“The development of anti-factor (F)VIII antibodies in haemophilia A (HA) subjects undergoing replacement therapy has been well documented. The correlation between antibody development and the FVIII product used for replacement therapy remains a subject of discussion. The aim of this study was to evaluate the presence of anti-FVIII antibodies towards three commercial rFVIII products in 34 HA subjects’ plasmas. Antibodies were quantitated by a Multiplex Fluorescence Immunoassay. All plasmas contained anti-FVIII antibodies at variable concentrations ranging from 50 nm to 570 μm. Eleven of the 20 HA subjects treated with one (r)FVIII product contained inhibitory anti-FVIII antibodies (0.8-3584 BU). The inhibitory antibody titre and the molar concentrations of total antibody were mildly correlated (r2 = 0.6).