Long-Term Outcomes of Reduced-Intensity Conditioning Hematopoietic Stem Cell Transplantation for Patients With...

Area of Science:

• Immunology
• Hematology
• Rheumatology

Background:

• High-intensity conditioning autologous hematopoietic stem cell transplantation (AHSCT) is the standard for advanced systemic sclerosis (SSc).
• The efficacy of reduced-intensity conditioning (RIC) prior to AHSCT in SSc patients with cardiac involvement is not well-established.

Purpose of the Study:

• To evaluate the long-term outcomes of RIC AHSCT in patients with SSc and cardiac involvement.
• To identify pre-transplant predictors of overall survival (OS) and progression-free survival (PFS) after RIC AHSCT.

Main Methods:

• A cohort of 42 SSc patients with cardiac involvement underwent RIC AHSCT.
• Progression was defined by DMARD reinitiation or end-organ failure worsening.
• OS and PFS were analyzed using Kaplan-Meier, with Cox regression for risk factor identification.

Main Results:

• Five-year OS was 75% and five-year PFS was 59% in the study cohort.
• Pre-transplant supplemental oxygen use and elevated B-type natriuretic peptide (BNP) were associated with increased mortality.
• Anti-Scl-70 antibody positivity correlated with disease progression.

Conclusions:

• RIC AHSCT is a safe and potentially effective treatment for severe SSc manifestations, including cardiac involvement.
• Pre-transplant factors like oxygen use, BNP levels, and anti-Scl-70 status predict outcomes, aiding risk stratification.