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

Lookup NU author(s): Professor Jaap van Laar

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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-termsurvival benefit of HSCT. The ASTIS and SCOT trials will determine whether the benefits of HSCT outweigh the risks of serious adverse events including treatment-relatedmortality of around 6-10% and potential long-term complications. Better patient selection and safer transplant regimens may improve the outcome of HSCT for SSc.

Publication metadata

Author(s): Naraghi K, van Laar JM

Publication type: Article

Publication status: Published

Journal: Current Rheumatology Reports

Year: 2013

Volume: 15

Issue: 5

Print publication date: 01/05/2013

ISSN (print): 1523-3774

Publisher: Springer

URL: http://dx.doi.org/10.1007/s11926-013-0326-2

DOI: 10.1007/s11926-013-0326-2

PubMed id: 23516015

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