Advanced therapies for hemophilia: Reality or fantasy?

Department of Physiology, Biological Sciences School, Complutense University of Madrid, Spain and Royal Foundation "Victoria Eugenia" of Haemophilia, Spain. .
Expert Review of Hematology (Impact Factor: 2.07). 06/2012; 5(3):245-7. DOI: 10.1586/ehm.12.25
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Available from: Antonio Liras, Mar 07, 2014
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    ABSTRACT: Introduction: In hemophilia, advanced therapies are warranted from a conceptual and methodological standpoint. Current advanced therapy strategies are centered on the use of adeno-associated viral vectors, although problems related to immunogenicity and hepatotoxicity still remain. Areas covered: Future clinical trials will have to scrupulously observe international bioethical standards in terms of patient selection, particularly children. Patient recruitment rates are likely to remain low due to the stringent exclusion criteria usually imposed on the trial population regarding their hepatic and immunological markers and the presence of viral coinfection; and to the existence of an optimal palliative treatment. Expert opinion: Accordingly, the results obtained are likely to be of low statistical significance, which could hinder their application to clinical practice. Another important issue is the degree to which society embraces these new emerging therapies. The unfamiliarity of society with these new methods, together with the many unresolved questions about them that remain, may threaten their acceptance not only by society at large but also by health-care professionals, which would limit their translational application to clinical practice.
    Expert opinion on biological therapy 03/2015; 15(5):1-10. DOI:10.1517/14712598.2015.1029451 · 3.74 Impact Factor