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系统性硬化症自体造血干细胞移植后的生活

Life after Autologous Hematopoietic Stem Cell Transplantation for Systemic Sclerosis.

作者信息

Moraes Daniela A, Oliveira Maria Carolina

机构信息

Division of Clinical Immunology, Department of Internal Medicine, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, Brazil.

Center for Cell-Based Therapy, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, Brazil.

出版信息

J Blood Med. 2021 Nov 9;12:951-964. doi: 10.2147/JBM.S338077. eCollection 2021.

Abstract

Stem cell transplantation has been investigated as treatment for severe and progressive systemic sclerosis (SSc) for the past 25 years. To date, more than 1000 SSc patients have been transplanted worldwide. Overall and event-free survival have increased over the years, reflecting stricter patient selection criteria and better clinical management strategies. This review addresses long-term outcomes of transplanted SSc patients, considering phase I/II and randomized clinical trials, as well as observational studies and those assessing specific aspects of the disease. Clinical outcomes are discussed comparatively between studies, highlighting advances, drawbacks and controversies in the field. Areas for future development are also discussed.

摘要

在过去25年里,干细胞移植已被作为重症和进行性系统性硬化症(SSc)的治疗方法进行研究。迄今为止,全球已有1000多名SSc患者接受了移植。多年来,总体生存率和无事件生存率有所提高,这反映了更严格的患者选择标准和更好的临床管理策略。本综述探讨了接受移植的SSc患者的长期预后,涉及I/II期和随机临床试验,以及观察性研究和评估该疾病特定方面的研究。将对各项研究的临床结果进行比较讨论,突出该领域的进展、缺点和争议。还将讨论未来的发展方向。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/04f9/8590726/7cf952ca557e/JBM-12-951-g0001.jpg

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