This project explores the feasibility of using a public health su

This project explores the feasibility of using a public health surveillance system to conduct national surveillance for inhibitors. Staff at 17 U.S. haemophilia treatment centres (HTC) enrolled patients with haemophilia A and B into this prospective study. HTC staff provided detailed check details historic data on product use and inhibitors at baseline, and postenrolment patients provided monthly detailed infusion logs. A central laboratory

performed inhibitor tests on blood specimens that were collected at baseline, annually, prior to any planned product switch or when clinically indicated. The central laboratory also performed genotyping of all enrolled patients. From January 2006 through June 2012, 1163 patients were enrolled and followed up for 3329 person-years. A total of 3048 inhibitor tests were performed and 23 new factor VIII inhibitors were identified, 61% of which were not clinically apparent. Infusion logs were submitted for 113 205 exposure days. Genotyping revealed 431 distinct mutations causing haemophilia, 151 of which had not previously been reported elsewhere in the world. This study provided critical information about the practical issues that must be addressed to successfully implement national inhibitor surveillance. Centralized testing with routine monitoring and confirmation of locally identified inhibitors will provide

valid and representative data with which to evaluate inhibitor incidence and prevalence, FK228 chemical structure monitor trends in occurrence rates and identify potential inhibitor outbreaks associated with products. “
“Summary.  Patients with haemophilia

can now look forward to greater life expectancy than ever before – a development that can be attributed to improved healthcare strategies Acyl CoA dehydrogenase and more effective treatments. In the last few decades, the treatment of haemophilia patients with inhibitors has also witnessed dramatic improvements through the development of bypassing agents, including recombinant activated factor VII (rFVIIa, NovoSeven®; Novo Nordisk, Bagsværd, Denmark). Growing evidence suggests that early initiation of treatment with rFVIIa results in greater haemostatic efficacy with fewer doses, leading to improved overall outcome. The new NovoSeven® room temperature stable formulation has been designed to optimize on-demand treatment by facilitating early initiation of therapy for haemorrhagic episodes, which brings the potential benefits of faster bleed resolution, reduced frequency of re-bleeding and reduced product consumption. This is particularly important for inhibitor patients, in whom orthopaedic complications are more severe than in non-inhibitor patients. Early treatment might help improve musculoskeletal status and therefore reduce disability, which improves patient quality of life and aids integration into society. These clinical advantages are also accompanied by both short-term and long-term economic benefits.

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