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J Autoimmun. 2018 Aug;92:35-46. doi: 10.1016/j.jaut.2018.06.002. Epub 2018 Jun 20.

Hematopoietic stem cell therapy for autoimmune diseases - Clinical experience and mechanisms.

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Department of Rheumatology and Clinical Immunology, Charité - University Medicine Berlin, Germany. Electronic address:
Unité de Médecine Interne, Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Hôpital St-Louis, AP-HP, 1 avenue Claude Vellefaux, 75010 Paris, Université Denis Diderot, France; Centre de Référence des Maladies auto-immunes systémiques Rares d'Ile-de-France (site constitutif), Filière FAI2R, France.
EBMT Paris Study Office / CEREST-TC, Department of Haematology, Saint Antoine Hospital, INSERM UMR 938, Université Pierre et Marie Curie, Paris, France.
The Royal London Hospital, Barts Health NHS Trust, London, E1 1BB UK; Centre for Immunobiology, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, E1 2AT, UK.
Division of Brain Sciences, Department of Medicine, Imperial College London, London, UK.
Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, S10 2JF, UK.


With accumulating evidence and improved outcomes along with recognition that modern biological therapies are not universally effective, require chronic administration and have high acquisition costs, hematopoietic stem cell transplantation (HSCT) has become an emerging direction for cell therapy in autoimmune diseases (ADs). The goal of this therapy is to induce medication-free remissions by resetting the immune system into a naïve and self-tolerant state through eradication of the autoreactive immunologic memory and profound re-configuration of the immune system induced by the transplant procedure. Safety of HSCT has generally improved by implementing internal quality management and external accreditation. Inter-disciplinary guidelines for patient selection, transplant technique and supportive care along with greater center experience should optimize safe and appropriate delivery of HSCT in specific ADs. In this review, we discuss the current role and future perspectives of HSCT in AD, focusing on recent published clinical and scientific studies and recommendations in the field.


Autoimmune diseases; Autoimmunity; Cell therapy; HSCT; Stem cell transplantation

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