Novel drugs to treat hemophilia

Expert Opinion on Emerging Drugs
Leonard A ValentinoMichele Grygotis

Abstract

Hemophilia management requires replacement of the deficient clotting factor. Inhibitor development complicates treatment, and the implementation of and adherence to prophylaxis, considered the standard of care for severe hemophilia, is hampered by the high cost and the need for frequent intravenous infusions. New, less expensive therapies are needed to reduce morbidities, enhance convenience and improve outcomes. We conducted a search of the Medline database from 1990 to 2010 for English language articles pertaining to hemophilia drug therapy. More than 4000 citations were obtained and examined for topic relevance. Additionally, proceedings from the American Society of Hematology, International Society on Thrombosis and Hemostasis, World Federation of Hemophilia, National Hemophilia Foundation and European Association for Haemophilia and Allied Disorders and the ClinicalTrials.gov website were reviewed for relevant publications and information. This article summarizes current research efforts focused on developing less immunogenic and more potent and/or longer-acting clotting factor concentrates that uniquely promote hemostasis, are more affordable and do not require intravenous administration. The availability of orally admini...Continue Reading

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Citations

Sep 21, 2012·Expert Review of Hematology·Mindy L Simpson, Leonard A Valentino
Jul 26, 2011·Expert Opinion on Biological Therapy·Leonard A Valentino
Jan 14, 2012·Biologicals : Journal of the International Association of Biological Standardization·Zuben E SaunaBasil Golding
Jun 5, 2013·Haemophilia : the Official Journal of the World Federation of Hemophilia·A E PastoftB Lauritzen
Apr 26, 2014·Haemophilia : the Official Journal of the World Federation of Hemophilia·F PeyvandiA Srivastava

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