Scleroderma with internal organ involvement is a debilitating and lethal autoimmune disorder with few effective treatments. But a study led by Duke Health researchers has found new cause for optimism using an aggressive stem cell transplant regimen.
The researchers, publishing in the Jan. 4 issue of the New England Journal of Medicine, found significantly improved survival among patients with a severe form of scleroderma who underwent chemotherapy, whole body radiation and a stem cell transplant. Patients also had less need for immune suppressant drugs after transplant.
“Scleroderma hardens the skin and connective tissues and in its severe form leads to fatal organ failure, most often the lungs” said lead author Keith Sullivan, MD, James B. Wyngaarden Professor of Medicine and Cellular Therapy. “In these severe cases, conventional drug therapies are not very effective long-term, so new approaches are a priority.”
Earlier publications suggested that stem cell transplant might be a viable treatment, using less intensive treatment without irradiation. These studies showed that reduced-intensity stem cell transplant improved survival, but the disease often returned and patient safety remained a concern. As a result, conventional immunosuppressive drug treatment remained the standard of care in the U.S.
In the current study, Sullivan and colleagues developed a transplant conditioning regimen that included high-dose chemotherapy plus whole-body radiation to fully wipe out the patient’s defective immune-forming system, with the aim of improving survival and diminishing the effects of the disease. They limited radiation by shielding patients’ kidneys and lungs while repopulating the blood and immune system.