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Real-life experience in switching to new extended half-life products at European haemophilia centres.

Abstract

The concept of replacement therapy in haemophilia is changing significantly thanks to the switch from standard products to extended half-life products. These novel drugs are showing beneficial effects overcoming current prophylaxis limitations by reducing the infusion frequency, maintaining a higher trough level to ensure a lower risk of bleeding, and making treatment significantly less distressing to patients by improving the quality of life. Real-life data on the efficacy of novel drugs and their impact on routine management of haemophilia A and B patients are still limited. This manuscript reports the results of a European survey conducted by the European Association for Haemophilia and Allied Disorders (EAHAD) at the beginning of 2018 on the clinical management of patients using extended half-life recombinant FVIII and FIX fusion products, since at the time of the survey none of the PEGylated products were available yet. We report data on the efficacy of these novel drugs by 33 European haemophilia centres that have already switched to extended half-life fusion products, showing a significant reduction in the number of infusions and a satisfactory trough levels in the clinical care of haemophilia patients, with a greater impact for haemophilia B.

Authors+Show Affiliations

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy. Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy.Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy.European Association for Hemophilia and Allied Disorders, Brussels, Belgium.Division of Haematology, Haemostasis and Thrombosis Unit, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels, Belgium.Department of Infection, Immunity and Cardiovascular Disease, University of Sheffield, Sheffield, UK. Sheffield Haemophilia and Thrombosis Centre, Sheffield, UK.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

31418967

Citation

Peyvandi, Flora, et al. "Real-life Experience in Switching to New Extended Half-life Products at European Haemophilia Centres." Haemophilia : the Official Journal of the World Federation of Hemophilia, 2019.
Peyvandi F, Garagiola I, Boscarino M, et al. Real-life experience in switching to new extended half-life products at European haemophilia centres. Haemophilia. 2019.
Peyvandi, F., Garagiola, I., Boscarino, M., Ryan, A., Hermans, C., & Makris, M. (2019). Real-life experience in switching to new extended half-life products at European haemophilia centres. Haemophilia : the Official Journal of the World Federation of Hemophilia, doi:10.1111/hae.13834.
Peyvandi F, et al. Real-life Experience in Switching to New Extended Half-life Products at European Haemophilia Centres. Haemophilia. 2019 Aug 16; PubMed PMID: 31418967.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Real-life experience in switching to new extended half-life products at European haemophilia centres. AU - Peyvandi,Flora, AU - Garagiola,Isabella, AU - Boscarino,Marco, AU - Ryan,Aislin, AU - Hermans,Cedric, AU - Makris,Michael, Y1 - 2019/08/16/ PY - 2019/03/08/received PY - 2019/07/02/revised PY - 2019/07/15/accepted PY - 2019/8/17/entrez KW - extended half-life products KW - hemophilia KW - infusion KW - treatment KW - trough level JF - Haemophilia : the official journal of the World Federation of Hemophilia JO - Haemophilia N2 - The concept of replacement therapy in haemophilia is changing significantly thanks to the switch from standard products to extended half-life products. These novel drugs are showing beneficial effects overcoming current prophylaxis limitations by reducing the infusion frequency, maintaining a higher trough level to ensure a lower risk of bleeding, and making treatment significantly less distressing to patients by improving the quality of life. Real-life data on the efficacy of novel drugs and their impact on routine management of haemophilia A and B patients are still limited. This manuscript reports the results of a European survey conducted by the European Association for Haemophilia and Allied Disorders (EAHAD) at the beginning of 2018 on the clinical management of patients using extended half-life recombinant FVIII and FIX fusion products, since at the time of the survey none of the PEGylated products were available yet. We report data on the efficacy of these novel drugs by 33 European haemophilia centres that have already switched to extended half-life fusion products, showing a significant reduction in the number of infusions and a satisfactory trough levels in the clinical care of haemophilia patients, with a greater impact for haemophilia B. SN - 1365-2516 UR - https://www.unboundmedicine.com/medline/citation/31418967/Real-life_experience_in_switching_to_new_extended_half-life_products_at_European_haemophilia_centres L2 - https://doi.org/10.1111/hae.13834 DB - PRIME DP - Unbound Medicine ER -