Long-term follow-up tracks benefits and risks of stem cell transplants for severe systemic sclerosis
An international study reports 60% progression-free survival at 10 years after autologous bone marrow transplantation, adding evidence of sustained positive clinical outcomes and long-term safety in patients treated for early severe systemic sclerosis.
An international study of 80 patients with early severe systemic sclerosis found that autologous bone marrow transplantation confers long-term benefits and poses manageable risks. At 10 years after the procedure, 60% of patients experienced no further progression of their disease (progression-free survival) and 71% survived without death (overall survival).
The median improvement in skin involvement was dramatic, with the median modified Rodnan skin score dropping from 24 at baseline to just 5 at 10 years. Lung function remained relatively stable, with forced vital capacity and diffusing capacity of the lungs for carbon monoxide both staying at around 70% of predicted values. Importantly, 43% of patients were alive and had no need for additional immunosuppressive treatment at the 10-year mark.
However, the study also identified a 6% early mortality rate attributable to the transplantation itself and a 13% non-relapse mortality rate over the long term. The study's authors emphasize that careful patient selection, pretransplant assessment, and rigorous long-term monitoring are crucial for realizing the potential benefits of autologous hematopoietic stem cell transplantation in this challenging condition.
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