Liras Antonio
Complutense University of Madrid, Research Institute 12 October Hospital of Madrid, Department of Physiology , Madrid , Spain +34649907879 ;
Expert Opin Biol Ther. 2015 May;15(5):713-22. doi: 10.1517/14712598.2015.1029451. Epub 2015 Mar 31.
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.
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.
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.
从概念和方法学的角度来看，血友病需要先进的治疗方法。目前的先进治疗策略主要集中在使用腺相关病毒载体上，尽管与免疫原性和肝毒性相关的问题仍然存在。
未来的临床试验在患者选择方面，尤其是儿童患者，必须严格遵守国际生物伦理标准。由于通常对试验人群的肝脏和免疫标记以及病毒合并感染情况设置严格的排除标准，且存在最佳的姑息治疗方法，患者招募率可能会持续较低。
因此，所获得的结果可能在统计学上意义不大，这可能会阻碍其应用于临床实践。另一个重要问题是社会对这些新兴疗法的接受程度。社会对这些新方法的不熟悉，以及关于它们仍存在的许多未解决问题，可能不仅会威胁到整个社会对它们的接受，也会威胁到医疗保健专业人员对它们的接受，这将限制它们向临床实践的转化应用。
