The study found that a significantly higher proportion of patients taking the drug, cyclophosphamide, showed improvement in lung function compared with those taking a placebo. Patients taking the drug also had significant reductions in breathlessness and reported feeling more energetic and in better health.
"Until now, there has been no proven therapy for scleroderma, and 60-70% of patients die within 10 years," said Donald Tashkin, M.D., Professor of Medicine at the David Geffen School of Medicine at the University of California, Los Angeles, one of the authors of the new study on the drug.
Although previous studies have suggested that cyclophosphamide may benefit people suffering from scleroderma, this study, known as the Scleroderma Lung Study, is the first large, randomized, controlled trial to study the drug's effectiveness against scleroderma lung disease. The study, sponsored by the National Institutes of Health, was conducted at 13 centers throughout the United States.
Scleroderma is a progressive disease that leads to the hardening and tightening of the skin and connective tissues-the fibers that provide the framework and support for the body. The disease can affect the blood vessels and internal organs, and about 80% of patients also have a problem with their lungs that leads to scarring (fibrosis). This scarring is caused by inflammation, in which an abnormal number of white blood cells accumulate in the lungs. The inflammation and scarring make breathing more difficult. More than half of scleroderma deaths are due to lung-related problems, Dr. Tashkin said.
About 150,000 Americans have scleroderma. Three to four time
Contact: Jim Augustine
American Thoracic Society