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June 20, 2026Annals of the Rheumatic DiseasesOpen Access

Autologous stem cell transplantation (ASCT) for Refractory juvenile-onset systemic sclerosis (JSSc)

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Authors

KTKathryn S. TorokPHPaulina HorveiJLJonathan Li

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Overview

Randomized trial shows durable multisystem improvement in patients with refractory juvenile-onset systemic sclerosis, suggesting early ASCT consideration.

Key Points

  • Assess the safety and clinical outcomes of autologous stem cell transplantation in patients with treatment-refractory juvenile-onset systemic sclerosis.
  • Eight patients with moderate-to-severe JSSc refractory to ≥ 2 disease-modifying antirheumatic drugs underwent CD34+-selected ASCT.
  • Conditioning involved alemtuzumab, rituximab, antithymocyte globulin, total body irradiation, and thiotepa.
  • Clinical outcomes were evaluated at baseline and every 6 months for up to 48 months post-ASCT.
  • All patients engrafted successfully with a median neutrophil and platelet engraftment of 13 and 22 days, respectively.
  • Event-free survival was 100% at a median follow-up of 30 months with no treatment-related deaths or organ toxicities.
  • Skin scores improved by a median of 93%, and 6-minute walk distance improved by 13% with most patients experiencing enhanced gastrointestinal, musculoskeletal function, and quality of life.

Cite This Study

Torok et al. (2026) studied this question.

synapsesocial.com/papers/6a362de1db0793dc1a535d4dhttps://doi.org/10.1016/j.ard.2026.05.022
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