Efficacy and safety of autologous hematopoietic stem cell transplantation for refractory systemic sclerosis after rituximab-bendamustine conditioning.

OBJECTIVES

To evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (auto-HSCT) using a novel rituximab-bendamustine conditioning regimen in patients with refractory systemic sclerosis (SSc).

METHODS

This retrospective cohort study enrolled 216 patients with refractory SSc who received treatment between August 2019 and August 2023. Participants were divided into a transplant group (n=104) receiving the novel auto-HSCT regimen, and a conventional group (n=112) treated with prednisone combined with cyclophosphamide. Efficacy indicators, including the modified Rodnan skin score (mRSS), pulmonary, cardiac and renal function, organ involvement manifestations, autoantibody titers, quality of life (SF-36) scores, and clinical remission status, were assessed at baseline and 24 weeks post-treatment. Adverse events and progression-free survival (PFS) were also analyzed.

RESULTS

At the 24-week follow-up, the transplant group achieved significantly greater mRSS improvement (P<0.001). Significant improvements were also observed in pulmonary function (forced vital capacity [FVC], P=0.009) and cardiac function (left ventricular ejection fraction [LVEF], P=0.005) in the transplant group. The transplant group had a higher overall clinical remission rate (75.00% vs. 60.71%) and longer PFS (all P<0.05). Infusion reactions were more prevalent in the transplant group (15.38% vs. 4.46%), whereas gastrointestinal reactions and hyperglycemia occurred more frequently in the conventional group.

CONCLUSION

Auto-HSCT with a rituximab-bendamustine conditioning regimen yields superior improvements in multi-organ lesions and long-term survival outcomes in patients with refractory SSc compared with conventional therapy, with a tolerable safety profile.