High-dose chemotherapy and transplantation of autologous CD34+ selected stem cells for progressive systemic sclerosis adjusted to cardiac and lung manifestation: an open-label, non-inferiority phase 2 trial

Key Points

• The aim is to evaluate the efficacy and safety of high-dose chemotherapy combined with autologous CD34+ stem cell transplantation in patients with progressive systemic sclerosis.
• Conducted as an open-label, non-inferiority phase 2 trial
• Involved patients with advanced organ involvement
• Evaluated three-year overall survival rates
• Demonstrated comparable three-year overall survival in the treated group
• Confirmed feasibility of the adapted regimen
• Highlighted significant risks associated with aHSCT in this patient population