Systemic sclerosis & Raynaud's
Clinical and Molecular Findings After Autologous Stem Cell Transplantation or Cyclophosphamide for Scleroderma: Handling Missing Longitudinal Data
Authors' conclusion
Longitudinal trends estimated from 2 statistical models affirm the efficacy of HSCT over CYC in severe SSc.
Study context
- Population
- Individuals with systemic sclerosis (SSc) randomised to CYC or hematopoietic stem cell transplantation (HSCT)
- Participants
- 33 vs 34
- Comparison
- HSCT vs CYC
- HSCTn = 33
- CYCn = 34reference
- Primary endpoint
- Longitudinal trends of clinical, pulmonary function and quality of life measures, accounting for early failures
Methodological detailsmixed-effects regression models (missing at random), shared parameter models (missing not at random)
- Mixed-effects regression models (missing at random)
- Shared parameter models (missing not at random)
Primary result
As reported in the abstract
FVC longitudinal trend after accounting for participant loss
HSCT
0.77
percentage points/year
CYC
−3.70
/year
P = 0.004
Further findings
Pulmonary and quality of life measures in inflammatory (n = 20) and fibroproliferative (n = 20) molecular subsets
- HSCT superior long-term trends vs CYC
mRSS in fibroproliferative subset
- HSCT superior
Normal-like subset (n = 22)
- Superiority of HSCT less apparent
Original publication
Clinical and Molecular Findings After Autologous Stem Cell Transplantation or Cyclophosphamide for Scleroderma: Handling Missing Longitudinal Data. Arthritis Care Res (Hoboken). Published 16 November 2022. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →