HSCT Seen as Superior to Current Therapies for Severe, Progressive Sclerosis

Autologous hematopoietic stem cell transplantation (HSCT), evaluated in 18 people with rapidly progressing systemic sclerosis, found the experimental therapy superior to standard treatments for the disease for this patient population. The retrospective report detailing these findings, “Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis,” ran in the Aug. 22 edition of the journal Bone Marrow Transplantation.

In systemic sclerosis, the immune system mistakenly attacks bodily tissues, causing scarring (fibrosis) and an abnormal vasculature, which can affect the lungs and compromise breathing.

Autologous HSCT involves collecting bone marrow cells from the body of the person with the disease. The faulty immune cells are then destroyed through chemotherapy, and harvested cells returned to the circulatory system of the patient, to form new and, hopefully, healthy immune system cells.

The study, led by Nicoletta Del Papa of the University of Milan, focused on 18 patients with rapidly progressing systematic sclerosis who underwent HSCT between 2003 to 2011. Investigators collected data from these patients for up to 5 years, and compared the clinical outcomes with those of 36 similar patients who had received conventional treatments. These treatments include immune system-suppressing medications such as methotrexate, azothioprine and prednisone. Researchers measured skin quality and lung capacity using standard tests.

Overall, lung capacity and the severity of skin problems were better in the group treated with HSCT when compared to those treated with standard therapies. The effects were statistically meaningful, adding further evidence to support the stem cell treatment. Survival over a period of 5 years was also greater for those individuals who had received HSCT, compared to those taking conventional treatments.

“This study confirms that the [autologous] HSCT is effective in prolonging survival, as well as in inducing a rapid reduction of skin involvement and disease activity, and preserving lung function in patients with [rapidly progressing systemic sclerosis],” the researchers concluded.

Because the patients enrolled in the trial had quickly progressing disease and other specific characteristics, the authors noted that certain patient populations might be best suited for the treatment. “Our study also suggests that patients with a high level of disease activity, severe skin involvement and without severe heart and lung involvement before transplantation procedure could be the best candidates to undergo this aggressive treatment.”

Overall, the study shows that autologous HSCT holds promise and might even be a preferred treatment for people with rapidly progressing systemic sclerosis, although more research is needed to confirm this possibility.