Slowing Scleroderma

(Ivanhoe Newswire) -- For patients with scleroderma, the prognosis is rarely encouraging. There may now be hope for this group. A study sponsored by the National Institutes of Health shows it may be possible to slow the deterioration caused by the disease.

About 300,000 Americans, primarily women, suffer from the autoimmune disease scleroderma. The condition is characterized by hardening of the skin and organs, which leads to disfigurement and disability. Patients often lose their battle to the disease within 10 years when the disease attacks their lungs.

In the current study, researchers announced "small but statistically significant improvement in lung function and less shortness of breath" in patients who were treated with the cancer drug cyclophosphamide. Additionally, the treated patients reported feeling healthier and more energetic.

Also, while the primary goal was improvement in lung function, researchers noted an improvement in shortness of breath, skin thickening and functional ability as well.

More than 150 patients at 13 centers were randomly assigned to receive either the drug or a placebo for a year. They were monitored every three months during the study and for an additional year after the treatment stopped.

Retrospective studies have indicated the treatment could be beneficial. It has therefore become a common treatment, but this is the first randomized study to formally look at the benefits.

Some side effects noted were low white blood counts and complication from infection. Researchers say these were managed by adjusting the dosage. They also indicate the benefits outweighed the risks.

Finally, researchers say they are encouraged by the results but warn caution is still warranted since the long-term effects have not been evaluated.

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SOURCE: The New England Journal of Medicine, 2006;354:2655-2666