Adopting a Standard of Care for Systemic Sclerosis: The Mayo Clinic Experience.

OBJECTIVE

Systemic sclerosis (SSc) is an autoimmune disease marked by immune dysregulation, leading to progressive fibrosis and multiorgan dysfunction. Autologous hematopoietic cell transplantation (autoHCT) has demonstrated superiority over conventional immunosuppression in randomized trials yet remains underused in the United States. We report outcomes following implementation of autoHCT as a standard-of-care strategy at our institution.

METHODS

We performed a retrospective analysis of consecutive patients with severe SSc undergoing autoHCT between April 1, 2020, and December 31, 2023. Eligibility required progressive disease despite standard immunosuppressive therapy. Primary endpoints were overall survival (OS) and event free survival (EFS). Secondary endpoints included cumulative incidence of relapse and nonrelapse mortality (NRM), organ-specific outcomes, and transplant-related complications.

RESULTS

Twenty-five patients underwent autoHCT. Median age was 46 years; 80% were female. At a median follow-up of 23 (interquartile range 16.4-31.9) months, two-year OS was 91.67% (95% confidence interval [CI] 81.25-100.0) and EFS was 74.51% (95% CI 58.78-94.45). Two-year cumulative incidence of relapse was 23.93% (95% CI 4.27 -43.59), and NRM was 8.33% (95% CI 2.97-19.64). Significant improvement in Modified Rodnan Skin Score was observed at day +90 and +180 (P < 0.05), whereas pulmonary function stabilized. Infectious complications occurred in 56% of patients, primarily viral reactivations.

CONCLUSION

Implementation of autoHCT for severe SSc in a multidisciplinary care model was associated with favorable survival and meaningful improvement in skin disease, with acceptable toxicity. Earlier referral and optimization of conditioning strategies may further reduce relapse and treatment-related morbidity.