Systemic sclerosis & Raynaud's
Myeloablation Followed by Hematopoietic Stem Cell Transplantation and Long‐Term Normalization of Systemic Sclerosis Molecular Signatures
Authors' conclusion
Paralleling the observed clinical benefit, HSCT leads to durable long-term normalization of the molecular signature in SSc, with completion of immune resetting to 54 months after HSCT.
Study context
- Population
- Systemic sclerosis (SSc) participants of the SCOT trial with peripheral blood cell (PBC) samples at baseline, 38 and 54 months after randomization; healthy controls30 with 38-month and 26 with 54-month samples
- Comparison
- Myeloablation followed by hematopoietic stem cell transplantation (HSCT) vs 12 months of IV CYC
- Primary endpoint
- Global PBC transcript (gene expression module) changes
- Follow-up
- 38 and 54 months after randomization
Methodological detailslongitudinal gene expression study, paired comparison to baseline
- Longitudinal gene expression study
- Paired comparison to baseline
Primary result
As reported in the abstract
PBC gene expression modules vs baseline (paired) · 38 and 54 months
- HSCT: significant down-regulation of interferon modules and up-regulation of cytotoxic/NK module at 38 and 54 months, indicating long-term normalization of baseline SSc signature
- CYC: no differentially expressed modules
- 38-month samples: HSCT = 19, CYC = 11; 54-month samples: HSCT = 16, CYC = 11
Further findings
PBC gene expression modules vs healthy controls · 38 and 54 months
- HSCT at 38 months: up-regulation of B cell and plasmablast modules, down-regulation of myeloid and inflammation modules
- HSCT at 54 months: no differentially expressed modules vs healthy controls, suggesting completion of immune reconstitution
- CYC: continued SSc transcript signature vs controls at both time points
Original publication
Myeloablation Followed by Hematopoietic Stem Cell Transplantation and Long‐Term Normalization of Systemic Sclerosis Molecular Signatures. Arthritis Rheumatol. Published 29 April 2024. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →