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Current state and future directions of autologous hematopoietic stem cell transplantation in systemic lupus erythematosus
  1. Gabor G Illei1,
  2. Ricard Cervera2,3,
  3. Richard K Burt4,
  4. Andrea Doria2,5,
  5. Falk Hiepe2,6,
  6. David Jayne2,7,
  7. Steven Pavletic8,9,
  8. Thierry Martin2,10,
  9. Alberto Marmont2,11,
  10. Riccardo Saccardi2,12,
  11. Alexandre E Voskuyl2,13,
  12. Dominique Farge2,14
  1. 1National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, Maryland, USA
  2. 2Autoimmune Diseases Working Party from the European Bone Marrow Transplant Association
  3. 3Department of Autoimmune Diseases, Hospital Clínic, Barcelona, Catalonia, Spain
  4. 4Division of Immunotherapy, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA
  5. 5Division of Rheumatology, Department of Clinical and Experimental Medicine, University of Padova, Padova, Italy
  6. 6Department of Rheumatology and Clinical Immunology, Charité – Universitätsmedizin Berlin, and German Rheumatism Research Center Berlin – a Leibniz Institute, Berlin, Germany
  7. 7Nephrology and Vasculitis, Addenbrooke's Hospital, Cambridge, UK
  8. 8Experimental Transplantation and Immunology Branch National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA
  9. 9Center for International Blood and Marrow Research, Milwaukee, Wisconsin, USA
  10. 10Department of Clinical Immunology, Strasbourg University Hospital, Université de Strasbourg, Strasbourg, France
  11. 11Department of Hematology, Ospedale di San Martino, Genoa, Italy
  12. 12Haematology Department, Careggi University Hospital, Florence, Italy
  13. 13Department of Rheumatology, VU University Medical Center, Amsterdam, The Netherlands
  14. 14Service de Médecine Interne, Hopital St Louis, Vellefaux, Paris, France
  1. Correspondence to Gabor G Illei, 10 Center Drive, National Institute of Dental and Craniofacial Research, National Institutes of Health, Rm 1N110, Bethesda, Maryland 20892, USA; illeig{at}


Autologous haematopoietic stem cell transplantation (AHSCT) has been proposed as a treatment modality which may arrest the autoimmune disease process and lead to sustained treatment-free remissions. Since the first consensus statement in 1997, approximately 200 autologous bone marrow or haematopoietic stem cell transplantations (HSCTs) have been reported worldwide for systemic lupus erythematosus (SLE). The current state of AHSCT in SLE was reviewed at a recent meeting of the autoimmune working party of the European Group for Blood and Marrow Transplantation. There was general agreement among experts in this field that in patients with severe SLE refractory to conventional immunosuppressive treatments, AHSCT can achieve sustained clinical remissions (ranging from 50% to 70% disease-free survival at 5 years) associated with qualitative immunological changes not seen with other forms of treatment. However, this clinical benefit is associated with an increase in short-term mortality in most studies. Improving patient selection, long-term follow-up of patients after AHSCT, optimisation of induction and maintenance treatment together with detailed analysis of the immune system are identified as key areas for future research. Optimally, AHSCT should be compared with conventional treatment in randomised controlled trials. Development of stronger transplant registries, defining a core set of clinical data and standardising biological sample collections would make future collaborations and comparison of studies more feasible.

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  • Funding This work was in part supported by the intramural research programme of the National Institute of Dental and Craniofacial Research, National Institutes of Health.

  • Patient consent Not obtained.

  • Provenance and peer review Not commissioned; externally peer reviewed.