Media availability: Drug found to help scleroderma patients


Scleroderma is a progressive condition in which fibrous tissue grows abnormally, causing the skin to thicken and harden, often disfiguring and disabling patients. In a life-threatening form of the disease known as systemic sclerosis, extra tissue also damages internal organs. Lung tissue is affected in about 80 percent of these patients, about half of whom die within 10 years as a result. Overall, scleroderma affects an estimated 300,000 Americans, most of them women.

For the first time, a randomized clinical trial has proven that a drug can slow down deterioration of lung function in scleroderma patients. When tissue in the lungs hardens or scars, breathing becomes difficult. In the study, participants who received treatment had small but statistically significant improvement in lung function (about 3 percent) and less shortness of breath (dyspnea) compared to those who were not treated. The drug also decreased the extent of skin thickening. Furthermore, treated participants reported feeling healthier and more energetic.

The Scleroderma Lung Study was funded by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH), with support from the NIH's National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS).

In the study, 158 scleroderma patients with early signs of lung problems were randomly assigned to daily treatment with cyclophosphamide (Cytoxan), a pill commonly used to treat cancer patients, or to placebo for one year. Participants were assessed periodically for an additional year after treatment. Negative side effects, such as low white blood counts and complications from infection, were more common among participants treated with the drug; however, these problems were sufficiently managed by adjustments in dosage. The researchers conclude that the benefits of treatment appear to outweigh the risks.

"Cyclophosphamide versus Placebo in Scleroderma Lung Disease," by Donald P. Tashkin, M.D., of the University of California at Los Angeles Medical Center and others, will be published in the June 22 issue of the New England Journal of Medicine.


NIH experts in lung diseases and disorders, as well as experts in connective tissue disorders, are available to comment on the study, findings and the disease. Scleroderma typically occurs between the ages of 30 and 50 years, most commonly among women. The average age of study participants was 48 years, and 71 percent of participants were women.



To schedule an interview with a lung disease expert, call the NHLBI Communications Office at 301-496-4236. To interview an expert on connective tissue disorders, call the NIAMS Communications Office at 301-496-8190.


Handout on Health: Scleroderma

Scleroderma Lung Study

Last reviewed: By John M. Grohol, Psy.D. on 30 Apr 2016
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