In people with scleroderma, the body's immune system attacks connective tissues throughout the body. This leads to formation of scar tissue in the skin and organs. Many patients die from scleroderma-related lung disease.
There's no cure for scleroderma, but there's some evidence the immune-suppressing cancer drug Cytoxan might slow the deadly scleroderma-related lung disease.
"Our findings indicate a significant, albeit quite modest, treatment effect of [Cytoxan]," conclude UCLA researcher Donald P. Tashkin, MD, and colleagues.
However, while Cytoxan is used to treat certain cancers, it can also cause several different kinds of cancer and is linked to serious side effects. Therefore, some other researchers say the drug should not be used for routine care in scleroderma sufferers.
Does Cytoxan really work? A clinical trial at 13 U.S. medical centers tested the drug against an inactive placebo in 158 patients for one year.
The bottom line: Cytoxan has a small benefit. Compared with those who got the placebo, patients receiving the drug did 2.5% better on a test of lung function. They also had somewhat fewer instances of shortness of breath and less thickening of the skin, as well as somewhat better health-related quality of life.
Those findings appear in the June 22 issue of The New England Journal of Medicine.
Accompanying the report is an editorial by Fernando J. Martinez, MD, and W. Joseph McCune, MD, of the University of Michigan, questioning routine use of Cytoxan for scleroderma patients.
"The modest therapeutic response [to Cytoxan in the Tashkin study] and the potential for significant toxic effects do not, in our opinion, support the conclusion that one year of daily [Cytoxan] should be considered routine therapy for all [patients with scleroderma lung disease]," they write.