Haematopoietic stem cell transplantation in autoimmune diseases: From basic science to clinical practice.

Abstract:

:Based on animal studies and serendipitous clinical cases, haematopoietic stem cell transplantation (HSCT) has been used since 1995 as a specific treatment for patients with severe treatment-resistant autoimmune disease (ADs). Despite other clinical developments for autoimmune diseases, including biological therapies, there has been an ongoing requirement for HSCT in some diseases and several thousand procedures have been registered in databases for a wide variety of diseases, predominantly for treatment with autologous HSCT. Currently, the main indications are multiple sclerosis, systemic sclerosis and Crohn's disease, which are supported by large series and randomised controlled trials (RCTs), whereas retrospective registry analyses support benefit in a range of rarer indications. Research into mechanisms of action has provided insight into how tolerance may be achieved with an intensive one-off treatment. In addition to the profound anti-inflammatory and immunosuppressive effects provided by the cytotoxic regimen, long-term responses in some diseases may be explained by 'resetting' the immune system through thymic reprocessing and generation of increased T-regulatory cell activity. This review aims to summarise the gradual evolution of HSCT in severe autoimmune diseases over the last 20 years, focussing on the recent publication of clinical and scientific studies, as well as evidence-based guidelines and recommendations.

journal_name

Curr Res Transl Med

authors

Kelsey PJ,Oliveira MC,Badoglio M,Sharrack B,Farge D,Snowden JA

doi

10.1016/j.retram.2016.03.003

subject

Has Abstract

pub_date

2016-04-01 00:00:00

pages

71-82

issue

2

issn

2452-3186

pii

S2452-3186(16)30001-0

journal_volume

64

pub_type

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