Hemophilia treatment innovation: 50 years of progress and more to come

Authors: Mannucci, PM

Affiliations: Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.

Publication: Journal of thrombosis and haemostasis : JTH ; 2023 ; 21. 403–412

Abstract: With the goal of emphasizing the striking advances that materialized in hemophilia care particularly in the last 10 years, the progress of knowledge that started from the 1970s will first be sketched as background. Subsequently, the unmet needs pertaining to therapeutic adherence and thus to prophylaxis effectiveness led to the availability of factor VIII and IX products with an extended plasma half-life as well as to emicizumab, the first nonfactor medicine for subcutaneous administration in patients with hemophilia A with or without inhibitor. The issue of a still lacking cure for the disease is approached by means of gene therapy, the first products of which were approved in 2022 for adults with both hemophilia types. Finally, views will be offered on further progress that is expected in the next few years and how patients and their care providers may make personalized choices among the wide array of therapeutic options.