Italian National Blood Centre, National Institute of Health, Rome, Italy.
Department of Hematology and Transfusion Medicine, Carlo Poma Hospital, Mantua, Italy.
Expert Opin Investig Drugs. 2020 Mar;29(3):295-301. doi: 10.1080/13543784.2020.1722999. Epub 2020 Feb 3.
: Hemophilia A and B are congenital bleeding disorders. The current standard management of patients with severe hemophilia is prophylaxis which is given intravenously two or three times weekly; however, this is associated with a significant burden on the quality of life of the patient. The main attempts to improve the management of hemophilia is hence through the development of a new generation of products with properties facilitating prophylaxis and/or a better control of bleeding.: This review describes the preclinical and phase 1/2 studies investigating the innovative products for the management of hemophilia patients with or without coagulation factor inhibitors.: Numerous innovative therapeutics, including factor concentrates and non-clotting factor-based therapies with extended half-life, are under clinical investigation. Among replacement therapies for hemophilia A, the results from phase 1/2 studies indicate that the most interesting products are those bioengineered using XTEN fusion technology. The anti-tissue factor pathway inhibitor antibody concizumab is the most innovative and interesting agent among non-clotting factor products. If the results of ongoing trials confirm the preliminary positive results, these promising agents will provide further improvements in the management and quality of life of patients with hemophilia.
: 血友病 A 和 B 是先天性出血性疾病。目前严重血友病患者的标准治疗方法是预防治疗，即每周静脉注射两到三次；然而，这会给患者的生活质量带来很大负担。因此，改善血友病管理的主要尝试是通过开发具有促进预防和/或更好控制出血特性的新一代产品来实现。: 本综述描述了正在进行的探索具有或不具有凝血因子抑制剂的血友病患者管理的创新产品的临床前和 1/2 期研究。: 正在进行的许多创新疗法，包括因子浓缩物和具有延长半衰期的非凝血因子疗法，都处于临床研究中。在血友病 A 的替代疗法中，1/2 期研究的结果表明，最有趣的产品是那些使用 XTEN 融合技术进行生物工程改造的产品。抗组织因子途径抑制剂抗体 concizumab 是非凝血因子产品中最具创新性和最有趣的药物。如果正在进行的试验的结果证实了初步的积极结果，这些有前途的药物将为血友病患者的管理和生活质量带来进一步的改善。
