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血友病抑制物患者的成就、挑战与未满足需求:2014年11月20日于法国巴黎召开的研讨会报告

Achievements, challenges and unmet needs for haemophilia patients with inhibitors: Report from a symposium in Paris, France on 20 November 2014.

作者信息

Dargaud Y, Pavlova A, Lacroix-Desmazes S, Fischer K, Soucie M, Claeyssens S, Scott D W, d'Oiron R, Lavigne-Lissalde G, Kenet G, Escuriola Ettingshausen C, Borel-Derlon A, Lambert T, Pasta G, Négrier C

机构信息

Unite d'Hemostase Clinique, Hopital Cardiologique Louis Pradel, Universite Lyon 1, Lyon, France.

Institute of Experimental Haematology and Transfusion Medicine, University Clinic, Bonn, Germany.

出版信息

Haemophilia. 2016 Jan;22 Suppl 1(Suppl 1):1-24. doi: 10.1111/hae.12860.

Abstract

Over the past 20 years, there have been many advances in haemophilia treatment that have allowed patients to take greater control of their disease. However, the development of factor VIII (FVIII) inhibitors is the greatest complication of the disease and a challenge in the treatment of haemophilia making management of bleeding episodes difficult and surgical procedures very challenging. A meeting to discuss the unmet needs of haemophilia patients with inhibitors was held in Paris on 20 November 2014. Topics discussed were genetic and non-genetic risk factors for the development of inhibitors, immunological aspects of inhibitor development, FVIII products and inhibitor development, generation and functional properties of engineered antigen-specific T regulatory cells, suppression of immune responses to FVIII, prophylaxis in haemophilia patients with inhibitors, epitope mapping of FVIII inhibitors, current controversies in immune tolerance induction therapy, surgery in haemophilia patients with inhibitors and future perspectives for the treatment of haemophilia patients with inhibitors. A summary of the key points discussed is presented in this paper.

摘要

在过去20年里,血友病治疗取得了许多进展,使患者能够更好地控制自己的疾病。然而,凝血因子VIII(FVIII)抑制物的产生是该疾病最严重的并发症,也是血友病治疗中的一项挑战,它使得出血事件的管理变得困难,外科手术极具挑战性。2014年11月20日在巴黎召开了一次会议,讨论有抑制物的血友病患者尚未满足的需求。讨论的主题包括抑制物产生的遗传和非遗传风险因素、抑制物产生的免疫学方面、FVIII产品与抑制物产生、工程化抗原特异性调节性T细胞的生成及功能特性、对FVIII免疫反应的抑制、有抑制物的血友病患者的预防、FVIII抑制物的表位作图、免疫耐受诱导治疗中的当前争议、有抑制物的血友病患者的手术以及有抑制物的血友病患者治疗的未来前景。本文呈现了所讨论要点的总结。

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