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Stem cell transplantation and mesenchymal cells to treat autoimmune diseases - 31/05/16

Doi : 10.1016/j.lpm.2016.05.002 
Alan Tyndall 1, , Jacob M. van Laar 2
1 University of Basel, department of rheumatology, 4, Petersgraben, 4031 Basel, Baseltstadt, Switzerland 
2 University medical center Utrecht, department of rheumatology and clinical immunology, Utrecht, The Netherlands 

Alan Tyndall, University of Basel, department of rheumatology, 4, Petersgraben, 4031 Basel, Baseltstadt, Switzerland.

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Summary

Since the start of the international stem cell transplantation project in 1997, over 2000 patients have received a haematopoietic stem cell transplant (HSCT), mostly autologous, as treatment for a severe autoimmune disease, the majority being multiple sclerosis (MS), systemic sclerosis (SSc) and Crohn's disease. There was an overall 85% 5-year survival and 43% progression-free survival. Around 30% of patients in all disease subgroups had a complete response, often durable despite full immune reconstitution. In many cases, e.g. systemic sclerosis, morphological improvement such as reduction of skin collagen and normalization of microvasculature was documented, beyond any predicted known effects of intense immunosuppression alone. It is hoped that the results of the three running large prospective randomized controlled trials will allow modification of the protocols to reduce the high transplant-related mortality which relates to regimen intensity, age of patient, and comorbidity. Mesenchymal stromal cells (MSC), often incorrectly called stem cells, have been the intense focus of in vitro studies and animal models of rheumatic and other diseases over more than a decade. Despite multiple plausible mechanisms of action and a plethora of positive in vivo animal studies, few randomised controlled clinical trials have demonstrated meaningful clinical benefit in any condition so far. This could be due to confusion in cell product terminology, complexity of clinical study design and execution or agreement on meaningful outcome measures. Within the rheumatic diseases, SLE and rheumatoid arthritis (RA) have received most attention. Uncontrolled multiple trial data from over 300 SLE patients have been published from one centre suggesting a positive outcome; one single centre comparative study in 172 RA was positive. In addition, small numbers of patients with Crohn's disease, multiple sclerosis, primary Sjögren's disease, polymyositis/dermatomyositis and type II diabetes mellitus have received MSC therapeutically. The possible reasons for this apparent mismatch between expectation and clinical reality will be discussed.

In this issue

Innovations in autoimmune diseases
L. Guillevin, France
Stem cell transplantation and mesenchymal cells to treat autoimmune diseases
A. Tyndall, Switzerland, and J.M. van Laar, Netherlands
New therapeutic approaches for ANCA-associated vasculitides
B. Chaigne, L. Guillevin, France
New therapeutic approaches in rheumatoid arthritis
R.F. van Vollenhoven, Sweden
Future treatments for Sjögren's syndrome
J.-O. Pers, A. Saraux, France
Therapeutic innovations in endocrine diseases: part 1: new medical treatments for chronic excess of endogenous cortisol (Cushing's syndrome)
X. Bertagna, France
Therapeutic innovations in endocrine diseases: part 2: modified-release glucocorticoid compounds: what good do they provide to the adrenal insufficient patient?
Y. Reznik, France
Therapeutic innovations in endocrine diseases: part 3: temozolomide and future therapeutics for aggressive pituitary tumors and carcinomas
H. Lasolle, G. Raverot, France
Therapeutic innovations in endocrine diseases: part 4: pasireotide: long-acting release somatostatin analogue
J.-L. Wémeau, France
Therapeutic innovations in endocrine diseases: part 5: rituximab and graves’ orbitopathy
M. Ladsous, France

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