Cytoxan Offers Modest Sclerosis Lung Benefits : The first RCT assessing the drug for interstitial lung disease showed a slowing in functional decline.

The decline in lung function among untreated patients with severe disease might have been greater than was seen in this trial, and thus would lead to a more pronounced difference between groups, these observers argue.

The best therapy for lung disease in scleroderma remains unknown.

According to Dr. Clements, preliminary results of a trial in the United Kingdom by Athol Wells, M.D., of Royal Brompton Hospital, London, and colleagues show dramatic improvements in lung function among patients receiving intravenous cyclophosphamide for 6 months—a 3% boost, compared with a 3% decline among those receiving placebo. After 6 months, the patients in the U.K. trial are switched to Imuran (azathioprine), a lesser immunosuppressive.

“In that context we have a confirmation that Cytoxan works and that immunosuppression helps,” said Dr. Clements. Nevertheless, he added, less noxious drugs would clearly be preferable.

Cyclophosphamide is “a sledge hammer,” he said. “Whether it is the right drug, we're still not sure.”