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J. Clin. Med. 2017, 6(4), 39; doi:10.3390/jcm6040039

Outcome of Clinical Trials with New Extended Half-Life FVIII/IX Concentrates

Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Via Pace 9, 20122 Milan, Italy
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Academic Editors: Massimo Morfini and Giovanni Di Minno
Received: 29 January 2017 / Revised: 9 March 2017 / Accepted: 11 March 2017 / Published: 28 March 2017
(This article belongs to the Special Issue Outstanding Advances in Hemophilia Therapies)
View Full-Text   |   Download PDF [239 KB, uploaded 28 March 2017]

Abstract

The development of a new generation of coagulation factors with improved pharmacokinetic profile will change the paradigm of treatment of persons with hemophilia (PWH). The standard treatment in PWH is represented by regular long-term prophylaxis that, given intravenously twice or thrice weekly, is associated with a not-negligible burden on patients’ quality of life. The availability of drugs with improved pharmacokinetic profile may improve prophylaxis feasibility and protection against bleeding episodes. This article summarizes the main results obtained from clinical trials with modified factor VIII (FVIII) and factor IX (FIX) molecules. Published literature on new molecules for replacement treatment in hemophilia A and B was retrieved using PubMed search, and all ongoing clinical trials have been researched via www.clinicaltrials.gov. Such new molecules are usually engineered to have a longer plasma half-life than that which has been obtained by chemical modification (i.e., conjugation with polyethylene glycol, PEG) or by creating recombinant fusion proteins. Results from phase I/III studies in previously treated adults and children are now available for the vast majority of new products, including the results of their use in a surgical setting. On the contrary, trials involving previously untreated patients are still ongoing for all and results not yet available. View Full-Text
Keywords: hemophilia A; hemophilia B; extended half-life concentrates; long-acting products; pegylation; glycopegylation; Fc fusion; albumin fusion hemophilia A; hemophilia B; extended half-life concentrates; long-acting products; pegylation; glycopegylation; Fc fusion; albumin fusion
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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MDPI and ACS Style

Mancuso, M.E.; Santagostino, E. Outcome of Clinical Trials with New Extended Half-Life FVIII/IX Concentrates. J. Clin. Med. 2017, 6, 39.

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