Arruda Valder R, Doshi Bhavya S, Samelson-Jones Benjamin J
The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
F1000Res. 2018 Apr 24;7. doi: 10.12688/f1000research.12491.1. eCollection 2018.
Several new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement products and non-factor options with 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.
近年来出现了几种治疗血友病的新疗法。这些策略涵盖了半衰期延长的凝血因子替代产品、具有改善药代动力学特征的非凝血因子选项，以及旨在实现表型治愈的基因疗法。虽然这些产品有可能极大地改变血友病的治疗方式，但在更广泛的适用性、长期安全性以及为每位患者选择何种治疗方案方面，仍存在一些挑战和问题。在此，我们回顾这些新兴疗法，重点关注每一类疗法中的争议和未解决的问题。
