Skip to main content

Stem cell transplantation in systemic sclerosis.

Publication ,  Journal Article
van Laar, JM; Sullivan, K
Published in: Curr Opin Rheumatol
November 2013

PURPOSE OF REVIEW: The purpose of this review is to discuss recent published clinical and mechanistic studies on stem cell transplantation for systemic sclerosis and their implications for clinical practice. RECENT FINDINGS: Retrospective analyses of independent cohorts of systemic sclerosis patients treated with autologous stem cell transplantation showed significant improvement of skin thickening, lung function and quality of life, but at the expense of 6-17% treatment-related mortality. Right heart catheterization was employed in one study to identify and exclude patients at risk of serious cardiopulmonary toxicity. The superior efficacy of stem cell transplantation versus intravenous pulses cyclophosphamide was demonstrated in a small randomized, controlled phase 2 trial in 19 systemic sclerosis patients and a large randomized phase 3 trial in 156 patients with severe diffuse cutaneous systemic sclerosis. The latter also showed a survival benefit of transplanted patients despite a 10% transplant-related mortality. Mechanistic studies in transplanted patients have shown major shifts in circulating natural killer cells, T and B lymphocytes immediately after stem cell transplantation, similar to those observed in other autoimmune conditions. Stem cell transplantation of systemic sclerosis patients with lung involvement resulted in demonstrable attenuation of thoracic high-resolution CT (HRCT) abnormalities and serum markers of lung fibrosis. SUMMARY: Stem cell transplantation is an effective treatment option for patients with severe systemic sclerosis, but is associated with toxicity and treatment-related mortality. The available data suggest that patient selection and comprehensive cardiopulmonary screening are critical factors in determining outcome. VIDEO ABSTRACT AVAILABLE: See the Supplementary Digital content 1 (http://links.lww.com/COR/A7).

Duke Scholars

Altmetric Attention Stats
Dimensions Citation Stats

Published In

Curr Opin Rheumatol

DOI

EISSN

1531-6963

Publication Date

November 2013

Volume

25

Issue

6

Start / End Page

719 / 725

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Scleroderma, Systemic
  • Randomized Controlled Trials as Topic
  • Prognosis
  • Patient Selection
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • Arthritis & Rheumatology
  • 3202 Clinical sciences
  • 1103 Clinical Sciences
 

Citation

APA
Chicago
ICMJE
MLA
NLM
van Laar, J. M., & Sullivan, K. (2013). Stem cell transplantation in systemic sclerosis. Curr Opin Rheumatol, 25(6), 719–725. https://doi.org/10.1097/01.bor.0000434669.32150.ac
Laar, Jacob M. van, and Keith Sullivan. “Stem cell transplantation in systemic sclerosis.Curr Opin Rheumatol 25, no. 6 (November 2013): 719–25. https://doi.org/10.1097/01.bor.0000434669.32150.ac.
van Laar JM, Sullivan K. Stem cell transplantation in systemic sclerosis. Curr Opin Rheumatol. 2013 Nov;25(6):719–25.
van Laar, Jacob M., and Keith Sullivan. “Stem cell transplantation in systemic sclerosis.Curr Opin Rheumatol, vol. 25, no. 6, Nov. 2013, pp. 719–25. Pubmed, doi:10.1097/01.bor.0000434669.32150.ac.
van Laar JM, Sullivan K. Stem cell transplantation in systemic sclerosis. Curr Opin Rheumatol. 2013 Nov;25(6):719–725.

Published In

Curr Opin Rheumatol

DOI

EISSN

1531-6963

Publication Date

November 2013

Volume

25

Issue

6

Start / End Page

719 / 725

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Scleroderma, Systemic
  • Randomized Controlled Trials as Topic
  • Prognosis
  • Patient Selection
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • Arthritis & Rheumatology
  • 3202 Clinical sciences
  • 1103 Clinical Sciences