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Forsyth A.L.,Rush University Medical Center | Giangrande P.,Oxford Haemophilia and Thrombosis Center | Hay C.R.M.,Royal Infirmary | Kenet G.,National Hemophilia Center | And 5 more authors.
Haemophilia | Year: 2012

Haemostasis management in people with haemophilia can present a range of challenges to physicians. Specific challenges that may be encountered relate to regimens for immune tolerance induction, use of central venous access devices, optimizing care of paediatric patients with inhibitors and improving outcomes in acquired haemophilia. There are also challenges related to performing surgery, and the establishment of specialist centres is valuable with regard to this. These challenges are considered in the light of available data, and with perspectives gained from the experience of experts treating patients around the world. Sharing this knowledge may help to improve patient management. © 2012 Blackwell Publishing Ltd.


Giangrande P.,Oxford Haemophilia and Thrombosis Center | Seitz R.,Paul Ehrlich Institute | Behr-Gross M.E.,European Directorate for the Quality of Medicines and HealthCare EDQM | Berger K.,Ludwig Maximilians University of Munich | And 4 more authors.
Haemophilia | Year: 2014

This report summarizes recommendations relating to haemophilia therapy arising from discussions among experts from 36 European countries during the Kreuth III meeting in April 2013. To optimize the organization of haemophilia care nationally, it is recommended that a formal body be established in each country to include the relevant clinicians, national haemophilia patient organization, health ministry, paying authority and (if appropriate) regulatory authorities. The minimum factor VIII consumption level in a country should be 3 I.U. per capita. Decisions on whether to adopt a new product should not be based solely on cost. Prophylaxis for children with severe haemophilia is already recognized as the optimum therapy. Ongoing prophylaxis for individual adults should also be provided when required based on clinical decision making by the clinician in consultation with the patient. Children with inhibitors who have failed, or who are not suitable for, immune tolerance therapy should be offered prophylaxis with bypassing agents. Single factor concentrates should be used as therapy wherever possible in patients with rare bleeding disorders. Orphan drug designation for a factor concentrate should not be used to hinder the development, licencing and marketing of other products for the same condition which have demonstrably different protein modification or enhancement. © 2014 John Wiley & Sons Ltd.


Hedner U.,Lund University | Lee C.A.,University of London | Lee C.A.,Oxford Haemophilia and Thrombosis Center
Haemophilia | Year: 2011

This review describes the background for the development of recombinant FVIIa (rFVIIa; NovoSeven) for use in haemophilic patients with inhibitors. The first proof of principle for using pharmacological doses of FVIIa as a haemostatic agent was obtained by producing small amounts of pure plasma-derived FVIIa, which showed encouraging effect in two patients with haemophilia A and inhibitors. To make pure FVIIa available for use in a larger number of patients, rFVIIa was produced that was approved for use in patients with inhibitors against coagulation factors (congenital haemophilia and acquired haemophilia) in 1996 (EU), 1999 (USA) and 2000 (Japan). The efficacy rate in severe bleedings and in major surgery including major orthopaedic surgery has been found to be around 90% in controlled studies, and no serious safety concerns have been demonstrated. The availability of rFVIIa has facilitated the performance of elective major surgery in haemophilia patients with inhibitors. Further steps along the vision of providing a treatment for inhibitor patients similar to non-inhibitor patients have been the efficacy of rFVIIa in home-treatment and recently the encouraging experience in prophylaxis. The concept of using pharmacological doses of rFVIIa as a haemostatic agent is a new one, which has caused difficulties in finding the correct dose. A step forward has been the demonstration that similar efficacy can be achieved after one single dose of 270μgkg-1 instead of three injections of a dose of 90μgkg-1. The higher clearance rate in children suggests that higher doses may be beneficial in children. The availability of rFVIIa has made advances in the understanding of coagulation processes possible. In a cell-based in vitro model, it has been shown that rFVIIa binds to preactivated platelets if present in concentrations of 30nm or higher. By doing so, it activates FX into FXa and enhances the thrombin generation on the activated platelet surface in the absence of FVIII/FIX. Through the increased thrombin generation, a firm, well-structured fibrin haemostatic plug, which is resistant to premature lysis, is formed. By exploiting this mechanism of action, rFVIIa may also be effective in situations other than haemophilia, characterized by an impaired thrombin generation. © 2010 Blackwell Publishing Ltd.


Lee C.A.,University of London | Lee C.A.,Oxford Haemophilia and Thrombosis Center | Berntorp E.E.,Skåne University Hospital | Hoots W.K.,U.S. National Institutes of Health | Hoots W.K.,University of Houston
Textbook of Hemophilia: Second Edition | Year: 2010

The Textbook of Hemophilia has become a definitive resource for all those managing hemophilia patients. It covers all the common and rare bleeding disorders, both in terms of clinical management as well as the genetic, laboratory, financial and psychological aspects. This second edition covers all the latest developments in the field of hemophilia, with new chapters on: the genetic and molecular basis of inherited blood disorders. how to manage adolescent and older patients. emergency medicine and inherited blood disorders. national hemophilia databases. Drawing on the vast experience of the authors, the aim of this textbook remains the same - to improve the care of patients suffering from hemophilia. The book is full of detailed guidance and advice on everyday clinical questions making it invaluable to all trainee and practicing hematologists. © 2010 Blackwell Publishing Ltd.

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