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The following is a summary of “Myeloablation followed by hematopoietic stem cell transplantation leads to long-term normalization of systemic sclerosis molecular signatures,” published in the March 2024 issue of Rheumatology by Wareing et al.

In Scleroderma: Cyclophosphamide or Transplantation trial (SCOT), when myeloablation was followed by hematopoietic stem cell transplantation (HSCT), Systemic Sclerosis (SSc) peripheral blood gene cell expression was normalized at a 26-month visit.

Researchers conducted a retrospective study analyzing the molecular alterations over 54 months post-randomization in individuals who underwent either HSCT or received intravenous cyclophosphamide (CYC) for 12 months.

They performed Global PBC transcript at pretreatment baseline at 38 and 54 months post-randomization and in healthy controls using Illumina HT-12 arrays.

The results had 30 (HSCT = 19 and CYC = 11) participants for 38 months and 26 (HSCT = 16 and CYC= 11) participants for a 54-month sample available. The HSCT arm showed significant regulation of interferon and up-regulation of cytotoxic/NK modules. The CYC arm detected no differentially expressed modules. Compared with Healthy controls, the HSCT arm showed an upregulation of B cell and plasmablast modules and a downregulation of myeloid and inflammation modules. No differentially expressed modules were observed in the HSCT arm after 54 months. The CYC arm showed an SSc transcript signature compared to controls at both time points.

Investigators concluded that HSCT leads to durable long-term normalization of the molecular signature in SSc with completion of immune resetting to 54 months post-HSCT.

Source: acrjournals.onlinelibrary.wiley.com/doi/abs/10.1002/art.42847