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血友病治疗的进展。

Advancement in the treatment of haemophilia.

机构信息

Department of Pharmaceutics, I.S.F.College of Pharmacy, Moga, Punjab, India.

Gujarat Forensic Sciences University, Gandhinagar, Gujarat, India.

出版信息

Int J Biol Macromol. 2018 Oct 15;118(Pt A):289-295. doi: 10.1016/j.ijbiomac.2018.06.084. Epub 2018 Jun 19.

DOI:10.1016/j.ijbiomac.2018.06.084
PMID:29928908
Abstract

Poor understanding of the pathophysiological mechanisms involved in Haemophilia is a major obstacle in accessing effective haemophilia disease management. Haemophilia is a life-frightening bleeding problem in which there are repeated bleeding episodes. Various approaches have been used, involves clotting factor replacement therapy for effective bleeding control in Haemophilia. Current advancements in the management of patients with haemophilia include altered pharmacokinetics clotting factor concentrates for better prophylaxis and management of haemophilia. This review sums up the prophylactic treatment, novel production techniques, other treatment techniques and the present position of gene therapy in the treatment of haemophilia.

摘要

对血友病相关病理生理机制的认识不足是血友病有效管理的主要障碍。血友病是一种危及生命的出血问题,会反复发作。为了有效控制血友病出血,已经采用了各种方法,包括凝血因子替代疗法。目前,血友病患者的管理进展包括改变凝血因子浓缩物的药代动力学,以更好地预防和管理血友病。本文综述了血友病治疗中预防性治疗、新型生产技术、其他治疗技术和基因治疗的现状。

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Advancement in the treatment of haemophilia.血友病治疗的进展。
Int J Biol Macromol. 2018 Oct 15;118(Pt A):289-295. doi: 10.1016/j.ijbiomac.2018.06.084. Epub 2018 Jun 19.
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[Revision consensus hemophilia: treatment and responsibility. Nederlandse Vereniging van Hemophilia Patients].[修订版血友病共识:治疗与责任。荷兰血友病患者协会]
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Pharmacokinetics of coagulation factors: clinical relevance for patients with haemophilia.凝血因子的药代动力学:对血友病患者的临床意义
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Int J Mol Sci. 2025 Jun 25;26(13):6100. doi: 10.3390/ijms26136100.
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Quality-of-Life Assessment and Pharmacokinetic Study in Hemophilia A Patients Undergoing Prophylactic Treatment.接受预防性治疗的甲型血友病患者的生活质量评估及药代动力学研究
Pharmacy (Basel). 2025 Feb 2;13(1):16. doi: 10.3390/pharmacy13010016.
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Impact of Replacement Therapy on Pregnancy Outcomes in Hemophilia Carriers: A Historical Cohort Study in Saudi Arabia.
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Life (Basel). 2024 May 11;14(5):623. doi: 10.3390/life14050623.
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Peritesticular scarring: An unusual presentation of hemophilia B.睾丸周围瘢痕形成:乙型血友病的一种罕见表现。
Urol Ann. 2022 Apr-Jun;14(2):183-185. doi: 10.4103/ua.ua_19_21. Epub 2022 Apr 18.
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Rare but not Abdicated: Status of Haemophilia in foothills of Himalaya, Uttarakhand: A cross-sectional study.罕见但并非被忽视:北阿坎德邦喜马拉雅山麓地区血友病的现状:一项横断面研究
J Family Med Prim Care. 2021 Mar;10(3):1437-1442. doi: 10.4103/jfmpc.jfmpc_1613_20. Epub 2021 Apr 8.
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FVIII expression by its native promoter sustains long-term correction avoiding immune response in hemophilic mice.其天然启动子驱动的 FVIII 表达可在血友病小鼠中长期纠正,避免免疫反应。
Blood Adv. 2019 Mar 12;3(5):825-838. doi: 10.1182/bloodadvances.2018027979.