免疫介导的神经系统自身免疫性疾病的造血干细胞移植经济学。

Q2 Medicine
Sophie L Hughes, Matthew J Prettyjohns, John A Snowden, Basil Sharrack
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引用次数: 0

摘要

自体造血干细胞移植(AHSCT)是一种治疗自身免疫性疾病的方法,可抑制炎症,重置免疫系统,从而阻止耐药患者的疾病活动和残疾进展。本章回顾了AHSCT治疗多发性硬化症(MS)的现有指南和卫生经济评价,并从英国国家医疗服务体系(NHS)和个人社会服务的角度进行了成本效用分析,比较了AHSCT与针对高度活动性复发缓解型多发性硬化症(RRMS)患者的疾病改变疗法(DMTs),该分析基于已发表的唯一一项针对该人群的随机对照试验 "MIST"。在为期 5 年的时间跨度内,AHSCT 比 MIST 中的 DMTs 更具优势(更有效、成本更低)。在每 QALY 20,000 英镑的临界值下,AHSCT 具有成本效益的可能性为 100%。造成这一结果的原因是,与 AHSCT 的前期费用相比,DMTs 的持续费用较高,而 AHSCT 的疗效较好。与纳他珠单抗相比,结果没有变化;AHSCT仍占优势。这些结果支持目前关于高度活动性RRMS的AHSCT的指南建议。由于缺乏卫生经济学分析,AHSCT治疗进展性和侵袭性多发性硬化症及其他免疫介导的神经系统疾病的成本效益仍不确定,这反映出临床证据基础有限。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Economics of hematopoietic stem cell transplant in immune-mediated neurologic autoimmune diseases.

Autologous hematopoietic stem cell transplantation (AHSCT) is a therapeutic procedure for autoimmune diseases which suppresses inflammation and resets the immune system, thereby halting disease activity and disability progression in treatment-resistant patients. This chapter reviews existing guidelines and health economic evaluations of AHSCT for multiple sclerosis (MS) and presents a cost-utility analysis from the UK NHS and personal social services perspective comparing AHSCT with disease-modifying therapies (DMTs) in patients with highly active relapsing-remitting MS (RRMS) based on the only published randomized controlled trial, "MIST," in this population. Over a 5-year time horizon, AHSCT was dominant (more effective and less costly) over the DMTs in MIST. At a threshold of £20,000 per QALY, there was a 100% probability that AHSCT was cost-effective. This result is explained by the high ongoing costs of DMTs compared with the up-front cost of AHSCT, combined with the high effectiveness of AHSCT. When compared with natalizumab, the result did not change; AHSCT remained dominant. These results support current guideline recommendations regarding AHSCT for highly active RRMS. The cost-effectiveness of AHSCT in progressive and aggressive MS and other immune-mediated neurologic diseases remains uncertain due to a lack of health economic analyses, reflecting the limited clinical evidence base.

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来源期刊
Handbook of clinical neurology
Handbook of clinical neurology Medicine-Neurology (clinical)
CiteScore
4.10
自引率
0.00%
发文量
302
期刊介绍: The Handbook of Clinical Neurology (HCN) was originally conceived and edited by Pierre Vinken and George Bruyn as a prestigious, multivolume reference work that would cover all the disorders encountered by clinicians and researchers engaged in neurology and allied fields. The first series of the Handbook (Volumes 1-44) was published between 1968 and 1982 and was followed by a second series (Volumes 45-78), guided by the same editors, which concluded in 2002. By that time, the Handbook had come to represent one of the largest scientific works ever published. In 2002, Professors Michael J. Aminoff, François Boller, and Dick F. Swaab took on the responsibility of supervising the third (current) series, the first volumes of which published in 2003. They have designed this series to encompass both clinical neurology and also the basic and clinical neurosciences that are its underpinning. Given the enormity and complexity of the accumulating literature, it is almost impossible to keep abreast of developments in the field, thus providing the raison d''être for the series. The series will thus appeal to clinicians and investigators alike, providing to each an added dimension. Now, more than 140 volumes after it began, the Handbook of Clinical Neurology series has an unparalleled reputation for providing the latest information on fundamental research on the operation of the nervous system in health and disease, comprehensive clinical information on neurological and related disorders, and up-to-date treatment protocols.
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