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Ferrata Storti Foundation
Haematologica 2020 Volume 105(8):2038-2043
Kreuth V initiative: European consensus proposals for treatment of hemophilia using standard products, extended half-life coagulation factor concentrates and non-replacement therapies
Flora Peyvandi,1,2 Karin Berger,3,4 Rainer Seitz,5 Anneliese Hilger,5 Marie-Laure Hecquet,6 Michael Wierer,6 Karl-Heinz Buchheit,6 Brian O’Mahony,7,8 Amanda Bok,8 Mike Makris,9 Ulrich Mansmann,4 Wolfgang Schramm10 and Pier
1 Mannuccio Mannucci
1Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy; 2Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy; 3University Hospital, Ludwig-Maximilian University, Department of Medicine III, Munich, Germany; 4Institute for Medical Information Processing, Biometry and Epidemiology (IBE), Ludwig-Maximilian-University, Munich, Germany; 5Paul-Ehrlich-Institut, Langen, Germany; 6European Directorate for the Quality of Medicines and Healthcare, Strasbourg, France; 7Trinity College, Dublin, Ireland; 8European Haemophilia Consortium, Brussels, Belgium; 9Sheffield Haemophilia and Thrombosis Centre, Sheffield, UK and 10Department of Transfusion Medicine and Haemostasis, Ludwig-Maximilians-University, Munich, Germany
ABSTRACT
This report contains the updated consensus recommendations for optimal hemophilia care produced in 2019 by three Working Groups (WG) on behalf of the European Directorate for Quality of Medicines and Healthcare in the frame of the Kreuth V Initiative. WG1 recommended access to prophylaxis for all patients, the achievement of plasma factor trough levels of at least 3-5% when extended half-life fac- tor VIII (FVIII) and FIX products are used, a personalized treatment regi- men, and a choice of chromogenic assays for treatment monitoring. It was also emphasized that innovative therapies should be supervised by hemophilia comprehensive care centers. WG2 recommended mandatory collection of postmarketing data to assure the long-term safety and effi- cacy of new hemophilia therapies, the establishment of national patient registries including the core data recommended by the European Medicines Agency and the International Society on Thrombosis and Haemostasis, with adequate support under public control, and greater collaboration to facilitate a comprehensive data evaluation throughout Europe. WG3 discussed methodological aspects of hemophilia care in the context of access decisions, particularly for innovative therapies, and rec- ommended that clinical studies should be designed to provide the quality of evidence needed by regulatory authorities, HTA bodies and healthcare providers. The dialogue between all stakeholders in hemophilia care and patient organizations should be fostered to implement these recommen- dations.
Introduction
The Wildbad Kreuth Initiative started in 1999 with a seminar including experts from 15 European Community member states. This was followed over the next few years by a series of four meetings. Treatment of hemophilia has always been the focus of the initiative, given the increasing number of diagnosed patients and the importance of providing them with optimal therapies. The objectives of the Initiative were to evaluate the state of hemophilia therapy, identify areas in need of further studies, and provide updated recommendations for optimal use of blood
Correspondence:
FLORA PEYVANDI,
flora.peyvandi@unimi.it
Received:November 18, 2019. Accepted: May 20, 2020. Pre-published: May 28, 2020.
doi:10.3324/haematol.2019.242735
Check the online version for the most updated information on this article, online supplements, and information on authorship & disclosures: www.haematologica.org/content/105/8/2038
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