Emerging therapies for hemophilia: controversies and unanswered questions.
Emerging therapies for hemophilia: controversies and unanswered questions.
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DOI：
10.12688/f1000research.12491.1
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发表时间：
2018-01-01
期刊：
影响因子：
--
通讯作者：
Samelson-Jones, Benjamin J
中科院分区：
文献类型：
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作者：
Arruda, Valder R;Doshi, Bhavya S;Samelson-Jones, Benjamin J
近年来出现了几种治疗血友病的新疗法。这些策略包括延长半衰期的因子替代产品和具有改善的药代动力学特征的非因子选择，以及旨在表型治愈的基因治疗。虽然这些产品有可能极大地改变血友病护理，但在更广泛的适用性、长期安全性以及每个患者应选择哪种选择方面仍然存在一些挑战和问题。在这里，我们回顾这些新兴疗法，重点关注每个类别中的争议和未解答的问题。
Several new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement productsand non-factor optionswith improved pharmacokinetic profiles to gene therapy aiming for phenotypic cure. While these products have the potential to change hemophilia care dramatically, several challenges and questions remain regarding broader applicability, long-term safety, and which option to pursue for each patient. Here, we review these emerging therapies with a focus on controversies and unanswered questions in each category.