ABSTRACT
Patients with severe rapidly progressive systemic sclerosis (SSc) have a poor prognosis. Standard immunosuppressive drugs have modest effects at best on stabilizing disease in some patients, but do not improve overall survival. Hematopoietic stem cell transplant (HSCT) is the first treatment to have disease modifying effects in these patients, with regression of skin and lung fibrosis and increased event-free and overall survival. However, early studies reported high transplant-related mortality rates. Gradual improvements in patient selection, center experience, and our understanding of the risks associated with different conditioning regimens has led to better standard of care guidelines and safer HSCT conditioning, with progressively more patients being treated.
