Kreuth V initiative: European consensus proposals for treatment of hemophilia using standard products, extended half-life coagulation factor concentrates and non-replacement therapies

Authors: Peyvandi, F., Berger, K., Seitz, R., Hilger, A., Hecquet, ML., Wierer, M., Buchheit, KH., O’Mahony, B., Bok, A., Makris, M., Mansmann, U., Schramm, W., and Mannucci, PM.

Publication: Haematologica; 105,8:2038-2043. September 2020

Affiliations: Fdn IRCCS Ca Granda Osped Maggiore Policlin, Angelo Bianchi Bon Hemophilia & Thrombosis Ctr, Milan, Italy; Fdn Luigi Villa, Milan, Italy; Univ Milan, Dept Pathophysiol & Transplantat, Milan, Italy; Ludwig Maximilians Univ Munchen, Univ Hosp, Dept Med 3, Munich, Germany; Ludwig Maximilians Univ Munchen, Inst Med Informat Proc Biometry & Epidemiol IBE, Munich, Germany; Paul Ehrlich Inst, Langen, Germany; European Directorate Qual Med & Healthcare, Strasbourg, France; Trinity Coll Dublin, Dublin, Ireland ; European Haemophilia Consortium, Brussels, Belgium; Sheffield Haemophilia & Thrombosis Ctr, Sheffield, S Yorkshire, England; Ludwig Maximilians Univ Munchen, Dept Transfus Med & Haemostasis, 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 factor VIII (FVIII) and FIX products are used, a personalized treatment regimen, 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 efficacy 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 recommended 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 recommendations.