Novel approaches to hemophilia therapy: successes and challenges
Novel approaches to hemophilia therapy: successes and challenges
复制标题
DOI：
10.1182/blood-2017-08-742312
复制
发表时间：
2017-11-23
期刊：
影响因子：
20.3
通讯作者：
Samelson-Jones, Benjamin J.
中科院分区：
文献类型：
--
作者：
Arruda, Valder R.;Doshi, Bhavya S.;Samelson-Jones, Benjamin J.
血友病A和血友病B的新疗法可能会继续改变临床实践。从延长半衰期到非因子产品和基因治疗，这些创新方法有可能通过降低输注频率来提高依从性，促进预防，为抑制剂患者提供替代方案以及简化给药途径来提高护理标准。每个类别都有内在的挑战，可能会限制这些有前途的疗法的更广泛应用。迄今为止，没有一个国家专门处理向发展中国家提供治疗的挑战。
New therapies for hemophilia A and hemophilia B will likely continue to change clinical practice. Ranging from extended half-life to nonfactor products and gene therapy, these innovative approaches have the potential to enhance the standard of care by decreasing infusion frequency to increase compliance, promoting prophylaxis, offering alternatives to inhibitor patients, and easing route of administration. Each category has intrinsic challenges that may limit the broader application of these promising therapies. To date, none specifically address the challenge of dispersing treatment to the developing world.