Scleroderma is a debilitating autoimmune disease that attacks the connective tissues and especially the lungs. Hardening of the skin is one of the most visible effects of the disease. Over 40 percent of patients will develop symptomatic lung problems – the leading cause of death for scleroderma patients.
FINDINGS:
A new nationwide study headed by UCLA found that a drug used for cancer called cyclophosphamide, helped alleviate the serious effects of scleroderma on breathing, lung function, quality of life, functional disability and skin thickness. This is the first controlled clinical trial to show a positive treatment for scleroderma lung disease.
IMPACT:
Findings will help researchers develop new drug therapies and also better understand development of the disease. The next step is further research into use of cylophosphamide as well as other immunosuppressant drugs.
AUTHORS:
Drs. Donald Tashkin and Michael Roth, professors of pulmonary and critical care medicine; and Drs. Philip Clements and Daniel Furst, professors of rheumatology, David Geffen School of Medicine at UCLA.
FUNDING:
The study was funded by the National Heart, Lung, and Blood Institute of the National Institutes of Health (NIH), with support from the NIH's National Institute of Arthritis and Musculoskeletal and Skin Diseases.
JOURNAL:
The study appears in the June 22 issue of the New England Journal of Medicine. A PDF of the full study is available.
Journal
New England Journal of Medicine