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血友病护理的过去、现在与未来。

Haemophilia care then, now and in the future.

作者信息

Oldenburg J, Dolan G, Lemm G

机构信息

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

出版信息

Haemophilia. 2009 Jan;15 Suppl 1:2-7. doi: 10.1111/j.1365-2516.2008.01946.x.

Abstract

Epidemiological data show the benefits of dramatically improved haemophilia care in all life-stages. There are improved administration techniques and dosing regimens, a shift from on-demand treatment to prophylaxis, successful treatment protocols for immune tolerance induction in patients with inhibitors and enhanced approaches to overall patient management. Improvements also include the introduction of virus inactivation methods for plasma derived clotting factor concentrates and the development of recombinant factor VIII therapy, which practically eliminated the risk of infectious disease transmission. Recombinant factor concentrates are recommended as treatment of choice by several guidelines today. All these developments have resulted in increased health-related quality of life and life expectancy in haemophilia patients, who are transitioning from childhood to adulthood with healthy joints and an overall healthy status today. Because of increased life expectancy, these patients are expected to experience age-related clinical problems that were not previously observed in this population. With respect to this, the spectrum of haemophilia care will be extended to diseases of older ages with the need of including further disciplines in comprehensive haemophilia care programmes. Despite these advances, the short half-life of factor VIII, requiring re-administration every 2 or 3 days and the development of inhibitors remains a challenge. Bayer's research and development currently focuses on the optimization of recombinant coagulation factors to address these challenges. Haemophilia care has experienced significant improvements within the past decades. Novel technologies and continued clinical research have facilitated the development of treatment regimen that resulted in dramatic increases in the life expectancy and quality of life of haemophilia patients. To set the scene for the following papers dealing with haemophilia care from paediatrics to geriatrics, developments behind these improvements and some aspects of future research will be presented in this paper.

摘要

流行病学数据显示,在血友病患者的所有生命阶段,血友病护理都得到了显著改善,带来了诸多益处。给药技术和给药方案得到改进,治疗模式从按需治疗转向预防治疗,针对有抑制物的患者诱导免疫耐受的治疗方案取得成功,整体患者管理方法也得到加强。改进还包括对血浆源性凝血因子浓缩物采用病毒灭活方法,以及开发重组凝血因子VIII疗法,这实际上消除了传染病传播风险。如今,多项指南推荐将重组凝血因子浓缩物作为首选治疗方法。所有这些进展都提高了血友病患者与健康相关的生活质量和预期寿命,他们如今正以健康的关节和整体健康状态从儿童期过渡到成年期。由于预期寿命延长,这些患者预计会出现此前该人群未曾出现过的与年龄相关的临床问题。就此而言,血友病护理范围将扩展至老年疾病,在综合血友病护理计划中需要纳入更多学科。尽管取得了这些进展,但凝血因子VIII半衰期短,需要每2至3天重新给药,以及抑制物的出现仍然是一大挑战。拜耳公司目前的研发工作重点是优化重组凝血因子以应对这些挑战。在过去几十年里,血友病护理取得了显著进步。新技术和持续的临床研究推动了治疗方案的发展,使血友病患者的预期寿命和生活质量大幅提高。为了为后续从儿科到老年科的血友病护理论文做好铺垫,本文将介绍这些改善背后的进展以及未来研究的一些方面。

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