Autologous blood stem cell transplants significantly improved survival in patients with high-risk scleroderma, despite significant treatment-related mortality, a randomized trial showed.
Compared with conventional pulsed cyclophosphamide therapy, the hazard ratio for death in those undergoing the stem cell transplant was 0.22 (95% CI 0.08 to 0.58, P=0.002) after 8 years of follow-up in the 156-patient trial, reported Jaap van Laar, MD, PhD, of Newcastle University in the England.
Although eight patients in the study's stem cell arm died as a result of treatment versus none receiving only cyclophosphamide, deaths from disease progression were dramatically reduced with the transplant procedure. Only five patients undergoing transplant died from progression during follow-up, compared with 15 in the cyclophosphamide-only arm, van Laar said at a press briefing, held before his formal presentation at the European League Against Rheumatism's annual meeting.
Scleroderma, also known as systemic sclerosis, is a rare autoimmune condition involving connective tissue affecting about one in 10,000 people; it is devastating in those unlucky enough to have it. Fibrosis in the lungs and other organs leads to diminished function, frequently ending with multiple organ failure and death.
The current standard of care is intravenous pulsed cyclophosphamide, which kills the immune cells that cause scleroderma but also kills other cells as well, resulting in significant toxicity.