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Stem Cell Therapy in Autoimmune Rheumatic Diseases: a Comprehensive Review

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Abstract

The clinical management of autoimmune rheumatic diseases (ARD) has undergone significant changes in the last few decades, leading to remarkable improvements in clinical outcomes of many patients with mild to moderate ARD. On the other hand, severe refractory ARD patients often have high morbidity and mortality. Extensive basic research and clinical evidence has opened the door to new encouraging perspectives, such as the establishment of a role of stem cell transplantation (SCT) in the strategic management of ARD. Given the great heterogeneity of ARD, it is difficult to assign an optimal SCT regimen to all ARD patients. SCT remains a challenging mode of therapy in ARD patients from the standpoints of both efficacy and safety. As the clinical data of SCT in ARD increases and as we improve our understanding of stem cell biology and the downstream effects on the immune system, the future is promising for the development of optimal personalized SCT regimens in ARD.

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Abbreviations

ACR:

American College of Rheumatology

Allo-HSCT:

Allogeneic HSCT

ANCA:

Antineutrophil cytoplasmic antibody

Anti-CCP antibodies:

Autoantibody to citrullinated peptide

ARD:

Autoimmune rheumatic diseases

ASSIST:

American Scleroderma Stem Cell Versus Immune Suppression Trial

ASTIRA:

Autologous Stem Cell Transplantation International Rheumatoid Arthritis

ASTIS:

Autologous Stem Cell Transplantation International Scleroderma trial

Auto-HSCT:

Autologous HSCT

BD:

Behcet’s disease

CYC:

Cyclophosphamide

DCs:

Dendritic cells

DM:

Dermatomyositis

DMARDs:

Disease-modifying antirheumatic drugs

EBMT/EULAR:

European Bone Marrow Transplant/European League Against Rheumatism

FBS:

Fetal bovine serum

Flu:

Fludarabine

GVA:

Graft-vs-autoimmunity

GVHD:

Graft-vs-host disease

HLA:

Human leukocyte antigen

HSCs:

Hematopoietic stem cells

HSCT:

Hematopoietic stem cell transplantation

ILD:

Interstitial lung disease

JIA:

Juvenile idiopathic arthritis

MHC:

Major histocompatibility complex

MP:

Methylprednisolone

MSCs:

Mesenchymal stem cells

PL/PRP:

Platelet lysate/platelet-rich plasma

PM:

Polymyositis

PSV:

Primary systemic vasculitis

QOL:

Quality of life

RA:

Rheumatoid arthritis

rbATG:

G rabbit antithymocyte globulin

SCOT:

Scleroderma: Cyclophosphamide or Transplantation trial

SCT:

Stem cell transplantation

SLE:

Systemic lupus erythematosus

SLEDAI:

SLE Disease Activity Index

SS:

Sjögren’s syndrome

SSc:

Systemic sclerosis

TBI:

Total body irradiation

Tregs:

T regulatory cells

TRM:

Transplant-related mortality

VOD:

Veno-occlusive disease of the liver

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Acknowledgments

This study is supported by the Natural Sciences Foundation of China (no. 81241094) and Science and Technology program of basic research projects of Qingdao City (no. 13-1-4-138-jch).

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Correspondence to Patrick S. C. Leung.

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Liu, B., Shu, S., Kenny, T.P. et al. Stem Cell Therapy in Autoimmune Rheumatic Diseases: a Comprehensive Review. Clinic Rev Allerg Immunol 47, 244–257 (2014). https://doi.org/10.1007/s12016-014-8445-8

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