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Update on Stem Cell Transplantation for Systemic Sclerosis: Recent Trial Results

  • SCLERODERMA (J VARGA, SECTION EDITOR)
  • Published:
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Abstract

Systemic sclerosis (SSc) is a heterogeneous condition characterized by the deposition of excess collagen in skin and internal organs due to vasculopathy, immune activation, low grade inflammation, and fibrosis. Progressive diffuse cutaneous SSc with organ involvement has a poor prognosis. The employment of autologous hematopoietic stem cell transplantation (HSCT) as a means to escalate immunosuppressive therapy has resulted in rapid and sustained improvement of skin thickening and functional ability, stabilization of major organ function with some improvement of vital capacity in pilot studies, registry analyses, and the phase II ASSIST trial. Results from the phase III ASTIS trial corroborate these findings and show long-term survival benefit of HSCT. The ASTIS and SCOT trials will determine whether the benefits of HSCT outweigh the risks of serious adverse events including treatment-related mortality of around 6–10 % and potential long-term complications. Better patient selection and safer transplant regimens may improve the outcome of HSCT for SSc.

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Conflict of Interest

Kamran Naraghi declares no conflict of interest.

Jacob M. van Laar has received speakers’ fees/honoraria from Roche, Genentech, Pfizer, Abbott Laboratories, Menarini, Bristol-Myers Squibb, and Miltenyi-Biotec; has received grant support from Amgen, Roche, Genentech, and Trubion; has served as a consultant for Roche UK; has received payment for development of educational presentations from Tonic and Benecke; has had travel/accommodations expenses covered/reimbursed by Roche, Genentech, Pfizer, Abbott Laboratories, Menarini, Bristol-Myers Squibb, and Miltenyi-Biotec; and received discounts on CliniMACS columns for the ASTIS trial.

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Authors and Affiliations

  1. Department of Rheumatology, The James Cook University Hospital, Middlesbrough, UK

    Kamran Naraghi & Jacob M. van Laar

  2. Musculoskeletal Research Group, Institute of Cellular Medicine, Newcastle University, The Medical School, 4th Floor Cookson Building, Newcastle upon Tyne, NE2 4HH, UK

    Jacob M. van Laar

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  1. Kamran Naraghi
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Correspondence to Jacob M. van Laar.

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This article is part of the Topical Collection on Scleroderma

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Naraghi, K., van Laar, J.M. Update on Stem Cell Transplantation for Systemic Sclerosis: Recent Trial Results. Curr Rheumatol Rep 15, 326 (2013). https://doi.org/10.1007/s11926-013-0326-2

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  • DOI: https://doi.org/10.1007/s11926-013-0326-2

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