Carr Marcus E, Tortella Bartholomew J
Robert Wood Johnson Medical School, Rutgers University, New Brunswick, NJ, USA ; Worldwide Research and Development, Pfizer, Inc., Cambridge, MA, USA.
Drexel University College of Medicine, Philadelphia, PA, USA ; Global Innovative Pharma, Pfizer, Inc., Collegeville, PA, USA.
J Blood Med. 2015 Sep 3;6:245-55. doi: 10.2147/JBM.S42669. eCollection 2015.
The evolution of care in hemophilia is a remarkable story. Over the last 60 years, advances in protein purification, protein chemistry, donor screening, viral inactivation, gene sequencing, gene cloning, and recombinant protein production have dramatically enhanced the treatment and lives of patients with hemophilia. Recent efforts have produced enhanced half-life (EHL) clotting factors to better support prophylaxis and decrease the frequency of infusions. Medical needs remain in the areas of alternate modes of administration to decrease the need for venous access, better treatment, and prophylaxis for patients who form antibodies to clotting factors, and ultimately a cure of the underlying genetic defect. In this brief review, the authors summarize data on EHL clotting factors, introduce agents whose mode of action is not clotting factor replacement, and list current gene therapy efforts.
血友病治疗的演变是一个非凡的故事。在过去60年里，蛋白质纯化、蛋白质化学、供体筛查、病毒灭活、基因测序、基因克隆和重组蛋白生产等方面的进展显著改善了血友病患者的治疗和生活。最近的努力已产生了延长半衰期（EHL）的凝血因子，以更好地支持预防并减少输注频率。在替代给药方式以减少静脉通路需求、为对凝血因子形成抗体的患者提供更好的治疗和预防，以及最终治愈潜在基因缺陷等领域，医疗需求依然存在。在这篇简短的综述中，作者总结了关于EHL凝血因子的数据，介绍了作用方式并非凝血因子替代的药物，并列出了当前的基因治疗研究进展。
