High-intensity immunosuppression versus modern standard care in poor-prognosis diffuse cutaneous systemic sclerosis:...

Area of Science:

• Rheumatology
• Immunology
• Hematology

Background:

• Diffuse cutaneous systemic sclerosis (dcSSc) is a severe autoimmune disease.
• Autologous hematopoietic stem-cell transplantation (HI-IS/HSCT) is recommended for selected poor-prognosis dcSSc cases.
• Real-world data on HI-IS/HSCT outcomes are limited.

Purpose of the Study:

• To compare 5-year outcomes of HI-IS/HSCT recipients with propensity-matched controls receiving standard care.
• To evaluate the efficacy of HI-IS/HSCT in improving survival and disease parameters in poor-prognosis dcSSc.
• To identify predictors of event-free survival (EFS) after HI-IS/HSCT.

Main Methods:

• Retrospective multicenter study of ACR/EULAR-2013 dcSSc patients treated after 2013.
• Propensity score matching (20 variables) for 1:1 comparison of HI-IS/HSCT and conventional immunosuppression groups.
• Assessment of 5-year overall survival (OS), EFS, progression-free survival (PFS), toxicities, and Global Rank Composite Score.

Main Results:

• Five-year OS was similar (90%) in both groups.
• HI-IS/HSCT significantly improved 5-year EFS (76% vs. 46%) and PFS (82% vs. 40%).
• HI-IS/HSCT led to greater skin improvement and pulmonary stabilization (FVC) but higher grade ≥4 toxicities (36% vs. 8%).

Conclusions:

• HI-IS/HSCT offers superior EFS and PFS, skin improvement, and pulmonary stabilization in poor-prognosis dcSSc.
• Overall survival is high and similar between HI-IS/HSCT and conventional care.
• Early HSCT is an effective disease-stabilizing therapy for selected dcSSc patients with careful cardiac screening.