WEDNESDAY, Jan. 3, 2018 (HealthDay News) — Stem cell transplants could offer new hope for people with a severe form of scleroderma — a debilitating and deadly condition that affects the immune system, a new study suggests.
“Scleroderma hardens the skin and connective tissues and, in its severe form, leads to fatal organ failure, most often the lungs,” said the study’s lead author, Dr. Keith Sullivan. He is a professor of medicine and cellular therapy at Duke University Medical Center.
“In these severe cases, conventional drug therapies are not very effective long-term, so new approaches are a priority,” Sullivan said in a hospital news release.
Drugs to suppress the immune system are the standard of care in the United States for scleroderma with internal organ involvement, according to the researchers. Their study tested the effectiveness of stem cell transplant along with high-dose chemotherapy and whole-body radiation to treat the disease. The study was funded by the U.S. National Institutes of Health.
For the study, 75 people with scleroderma were randomly assigned to receive one of two treatments. Roughly half of the group received a stem cell transplant designed to destroy their defective immune system and replace it with their own treated blood stem cells. The other half received 12 months of conventional immune-suppressing treatment.
After 10 years, survival was better among those who underwent chemotherapy, whole-body radiation and a stem cell transplant, the study found. People in this group also had less need for immune-suppressing drugs after their transplant.
“These results show that individuals with poor-prognosis scleroderma can improve and live longer and that these advances appear durable,” Sullivan said.
The researchers noted, though, that stem cell transplant was riskier. It was associated with more serious side effects, such as low blood counts, infections and death, the study found.
“Patients and their doctors should carefully weigh the pros and cons of intensive treatment with stem cell transplant, but this may hopefully set a new standard in this otherwise devastating autoimmune disease,” Sullivan said.
“These advances show the value of medical research and clinical trials in finding better therapies to advance health,” he added.
The study was scheduled for publication in the Jan. 4 issue of the New England Journal of Medicine.
The American College of Rheumatology has more on scleroderma.