Autologous Stem Cell Transplants (sometimes called hematopoietic stem cell transplants and abbreviated HSCT) – This is an experimental treatment that uses the patient’s own stem cells essentially to restart his/her immune system. Autologous stem cell transplant is a complex procedure, and there is definite risk associated with the procedure itself. However, mortality rates are now much lower than in the initial studies as researchers have learned to better screen patients for this procedure. One of the primary risk factors for treatment mortality is heart involvement, so patients who receive HSCT as part of these studies are screened carefully for potential existing cardiac problems before being accepted into the study.
It is far too early to know how long HSCT therapy will last even if successful in the short-term. However, the preliminary data suggest that this treatment approach may result in initial symptom improvement and improved five-year survival rates for patients with diffuse scleroderma. This suggests that for some patients with early-stage, rapidly-progressing diffuse scleroderma, enrolling in an ongoing HSCT research study may be an appropriate option to consider.