Hematopoietic stem cell transplantation for systemic sclerosis: history and current status

Curr Opin Rheumatol. 2011 Nov;23(6):519-29. doi: 10.1097/BOR.0b013e32834aa45f.

Abstract

Purpose of review: Systemic sclerosis (SSc) remains one of the last severe autoimmune disease with a poor prognosis and modest response to immunosuppressive therapy. Mortality in severe diffuse disease with internal organ involvement is elevated. Autologous hematopoietic transplantation (HSCT) has emerged in the last decade as a promising disease-modifying treatment.

Recent findings: In phase I/II trials, HSCT has demonstrated to induce impressive reversal of skin fibrosis, neoangiogenesis, improved functionality and quality of life, and stabilization of internal organ function. Treatment-related mortality was reduced over time by better pretransplant evaluation and by treating patients earlier in disease.

Summary: Two out of three randomized trials of autologous HSCT for SSc have been concluded: the nonmyeloablative American Systemic Sclerosis Immune Suppression versus Transplant, and Autologous Stem cell Transplantation International Scleroderma. The myeloablative Scleroderma Cyclophosphamide versus Transplant instead is still recruiting patients. The soon expected results from these trials should clarify the role of autologous HSCT in the challenging management of severe SSc.

Publication types

  • Historical Article
  • Review

MeSH terms

  • Clinical Trials, Phase I as Topic
  • Clinical Trials, Phase II as Topic
  • Hematopoietic Stem Cell Transplantation* / history
  • Hematopoietic Stem Cell Transplantation* / methods
  • History, 20th Century
  • History, 21st Century
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Randomized Controlled Trials as Topic
  • Scleroderma, Systemic / therapy*
  • Transplantation Conditioning
  • Transplantation, Autologous

Substances

  • Immunosuppressive Agents