Comparative analysis of the pivotal studies of extended half-life recombinant FVIII products for treatment of haemophilia A

Authors: Mannucci PM; Cortesi, PA; Di Minno, MND; Sanò, M; Mantovani, LG; Di Minno, G.

Affiliations: Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy ; Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy ; Department of Translational Medical Sciences, Federico II University, Naples, Italy ; ASL CN2 Alba-Bra, Farmacia Ospedaliera e Servizio Farmaceutico Territoriale, Alba, Italy ; IRCCS Multimedica Research Hospital, Sesto San Giovanni, Italy ; Department of Clinical Medicine and Surgery, Federico II University, Naples, Italy.

Publication: Haemophilia 2021 May

Abstract: The need to reduce the burden of injections and improve adherence and clinical outcomes in haemophilia A led to the development of recombinant FVIII products endowed with an extended plasma half-life (EHL-rFVIII) in comparison with standard half-life products (SHL-rFVIII). Lack of head-to-head studies makes difficult to grasp the relative value of each treatment option. We conducted a combined evaluation of the individual pivotal trials in order to assess between-product differences regarding the reported efficacy results and FVIII consumption. We evaluated 4 EHL-rFVIII products available to treat patients with haemophilia A without inhibitors and also a SHL-rFVIII as a comparator. In the frame of these clinical studies, all the EHL-rFVIII products showed a decrease in the injection burden coupled with good clinical efficacy, even though there were between-product differences in terms of reduction in injection frequencies. Further, between-product differences in terms of weekly/yearly consumption of rFVIII expressed in IU/Kg were identified, suggesting a different economic impact for the different EHL-rFVIII products in the context of comparable clinical efficacy. The present findings based upon the review of pivotal studies done in the frame of a highly selected clinical scenario should be integrated with real-life data.