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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.


DOI:10.2147/JBM.S338077
PMID:34785969
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8590726/
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
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/04f9/8590726/7cf952ca557e/JBM-12-951-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/04f9/8590726/7cf952ca557e/JBM-12-951-g0001.jpg

相似文献

[1]
Life after Autologous Hematopoietic Stem Cell Transplantation for Systemic Sclerosis.

J Blood Med. 2021-11-9

[2]
Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party.

Haematologica. 2021-2-1

[3]
Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience.

Adv Rheumatol. 2021-2-6

[4]
Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis.

Ann Rheum Dis. 2008-1

[5]
[Autologous hematopoietic stem cell transplantation for systemic sclerosis : Position statement of the stem cell therapy working party of the German Society of Rheumatology].

Z Rheumatol. 2020-6

[6]
Autologous haematopoietic stem cell transplantation for Japanese patients with systemic sclerosis: Long-term follow-up on a phase II trial and treatment-related fatal cardiomyopathy.

Mod Rheumatol. 2018-9

[7]
Health-related quality of life in systemic sclerosis before and after autologous haematopoietic stem cell transplant-a systematic review.

Rheumatology (Oxford). 2020-4-1

[8]
Association of Autologous Hematopoietic Stem Cell Transplantation in Systemic Sclerosis With Marked Improvement in Health-Related Quality of Life.

Arthritis Rheumatol. 2021-2

[9]
Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years' experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases.

Haematologica. 2009-9-22

[10]
CD34-selected versus unmanipulated autologous haematopoietic stem cell transplantation in the treatment of severe systemic sclerosis: a post hoc analysis of a phase I/II clinical trial conducted in Japan.

Arthritis Res Ther. 2019-1-22

引用本文的文献

[1]
Case report: A successful second autologous hematopoietic stem cell transplantation in refractory systemic sclerosis, with positive effect on skin involvement, pulmonary function and microcirculation.

Front Immunol. 2022

[2]
Autoantibodies as Biomarker and Therapeutic Target in Systemic Sclerosis.

Biomedicines. 2022-9-1

[3]
Mesenchymal Stromal Cells for Enhancing Hematopoietic Engraftment and Treatment of Graft-Versus-Host Disease, Hemorrhages and Acute Respiratory Distress Syndrome.

Front Immunol. 2022-3-18

[4]
Autologous Haematopoietic Stem Cell Transplantation and Systemic Sclerosis: Focus on Interstitial Lung Disease.

Cells. 2022-3-1

本文引用的文献

[1]
Erectile function and connective tissue diseases. Prevalence of erectile dysfunction in German men with systemic sclerosis compared to other connective tissue diseases and healthy subjects.

Clin Exp Rheumatol. 2021

[2]
Long term outcomes of the French ASTIS systemic sclerosis cohort using the global rank composite score.

Bone Marrow Transplant. 2021-9

[3]
A randomised, open-label trial to assess the optimal treatment strategy in early diffuse cutaneous systemic sclerosis: the UPSIDE study protocol.

BMJ Open. 2021-3-18

[4]
Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience.

Adv Rheumatol. 2021-2-6

[5]
Machine learning integration of scleroderma histology and gene expression identifies fibroblast polarisation as a hallmark of clinical severity and improvement.

Ann Rheum Dis. 2021-2

[6]
Evolution of Systemic Sclerosis-Associated Interstitial Lung Disease One Year After Hematopoietic Stem Cell Transplantation or Cyclophosphamide.

Arthritis Care Res (Hoboken). 2022-3

[7]
Machine learning predicts stem cell transplant response in severe scleroderma.

Ann Rheum Dis. 2020-9-15

[8]
Association of Autologous Hematopoietic Stem Cell Transplantation in Systemic Sclerosis With Marked Improvement in Health-Related Quality of Life.

Arthritis Rheumatol. 2021-2

[9]
An update on the pharmacotherapeutic options and treatment strategies for systemic sclerosis.

Expert Opin Pharmacother. 2020-7-17

[10]
Cardiac safe hematopoietic stem cell transplantation for systemic sclerosis with poor cardiac function: a pilot safety study that decreases neutropenic interval to 5 days.

Bone Marrow Transplant. 2021-1

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