05 U h−1dL−1). Two infusion-related events occurred: one with Hyate:C, one with placebo. Four of five subjects without anti-porcine FVIII inhibitors at baseline remained porcine FVIII inhibitor negative 29 days after infusion. A single dose of OBI-1 appears to have higher bioavailability than Hyate:C in subjects with haemophilia A without measurable anti-porcine Panobinostat FVIII inhibitors, and is well tolerated. These results should be confirmed in a larger phase 2/3 study. “
“Clinical registries or databases have an increasing role in the management of inherited bleeding disorders. Initially, research-based registries provided valuable data and now national databases are increasingly being developed with multiple

stakeholders, including persons with haemophilia (PWH) and payers, to enable improvements and efficiencies in care. Registries are extending to international collaborations to collect adverse event data and comparisons of national approaches to the management of haemophilia to improve the availability of product to PWH. Clinical registries have a clear role in the monitoring and benchmarking of quality of care and health outcomes

in many areas of medicine. Registries can be disease or complication selleck inhibitor specific with a strong clinical or research focus, or a broad collection of data relating to a disease or condition – either based on geographic region or area of care, nationally or internationally. Clear recommendations for the establishment of national registries in haemophilia care are made by the World Federation MCE of Haemophilia (WFH) [1] and leading professional groups in haemophilia [2], whereas registries in specific areas provide valuable information in rarer disorders, e.g. the rare bleeding disorders register [3]. National registries enable the documentation of clinical need with demographic information and the opportunity for planning of the resources and treatment product required. Strong clinical governance is required for national registries with input from key stakeholders – including

clinicians, payers and persons with haemophilia (PWH). Advancing information technology allows engagement with PWH to input treatment and usage data, and allows either more real time data for outcomes of specific treatment or measures of quality of life and impact on daily life. However, increasing data collection from individuals requires stringent adherence to security and privacy requirements to ensure that there is no impact on the PWH engaging in the registries. Descriptions of three types of registry illustrate their value: the UK Haemophilia Doctors’ Organisation (UKHCDO) National Haemophilia Database (NHD), a European approach to adverse event monitoring and the WFH compilation of information from national patient organizations, – all impact improving patient care. The National Patient Registry in the UK was established in 1968 in Oxford following the establishment of Haemophilia Centres by the Department of Health in the UK.