Myeloablation followed by autologous stem cell transplantation normalises systemic sclerosis molecular signatures

Shervin Assassi, Xuan Wang, Guocai Chen, Ellen Goldmuntz, Lynette Keyes-Elstein, Jun Ying, Paul K. Wallace, Jacob Turner, W. Jim Zheng, Virginia Pascual, John Varga, Monique E. Hinchcliff, Chiara Bellocchi, Peter McSweeney, Daniel E. Furst, Richard A. Nash, Leslie J. Crofford, Beverly Welch, Ashley Pinckney, Maureen D. MayesKeith M. Sullivan

Research output: Contribution to journalArticlepeer-review


In the SCOT trial, the effectiveness of haematopoietic stem cell transplantation (HSCT) was compared to cyclophosphamide (CYC) treatment in systemic sclerosis (SSc) patients. The objective was to observe global molecular changes in whole blood transcripts and serum protein levels following treatments. Pre and post-treatment (at 8 and 26 months) analyses revealed that at baseline, interferon (IFN) and neutrophil modules were heightened, while the cytotoxic/NK module was lowered in SSc patients compared to unaffected controls. Post-treatment, a significant decrease in IFN and neutrophil modules and an increase in the cytotoxic/NK module were observed in the HSCT group, with no notable change in the CYC group. This molecular 'correction' associated with HSCT correlated with clinical improvements in pulmonary and skin scores, implying that HSCT could significantly amend disease-related molecular signatures in systemic sclerosis, unlike conventional CYC treatment.
Original languageEnglish
Pages (from-to)1371-1378
Number of pages8
JournalAnnals of the Rheumatic Diseases
Issue number10
StatePublished - Oct 1 2019

ASJC Scopus Subject Areas

  • Rheumatology
  • Immunology and Allergy
  • Immunology
  • General Biochemistry,Genetics and Molecular Biology


  • cyclophosphamide
  • hematopoietic stem cell transplantation
  • interferon signature
  • neutrophil
  • systemic sclerosis

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