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Cyclophosphamide To Treat Patients with Scleroderma Lung Disease FREE

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The summary below is from the full report titled “Cyclophosphamide Is Associated with Pulmonary Function and Survival Benefit in Patients with Scleroderma and Alveolitis.” It is in the 20 June 2000 issue of Annals of Internal Medicine (volume 132, pages 947-954). The authors are B. White, W.C. Moore, F.M. Wigley, H.Q. Xiao, and R.A. Wise.

Ann Intern Med. 2000;132(12):947. doi:10.7326/0003-4819-132-12-200006200-00044
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What is the problem and what is known about it so far?

Scleroderma is a disease of unknown cause that produces fibrosis (hardening or scarring) of the connective tissue in the skin, blood vessels, and internal organs. This results in a tight, shiny appearance of the skin and potentially life-threatening abnormalities of the lungs, heart, kidneys, and digestive tract. Scleroderma lung disease is difficult to treat. Lung inflammation (alveolitis) occurs before lung scarring (fibrosis). Cyclophosphamide is a drug that decreases inflammation; therefore, it may be useful in treating patients with scleroderma lung disease.

Why did the researchers do this particular study?

To find out whether patients with scleroderma lung disease who receive cyclophosphamide have better lung function and live longer than patients who do not get this drug.

Who was studied?

The researchers studied 103 patients with scleroderma lung disease at the Johns Hopkins and University of Maryland Scleroderma Center.

How was the study done?

Some of the patients were treated with cyclophosphamide, depending on their doctor's decision. All of the patients underwent procedures to obtain pieces of lung tissue (biopsies) or lung cells (washings) that could be examined for inflammation. The researchers looked for signs of alveolitis on the lung biopsies or washings. They then compared lung function, which they evaluated by using special breathing tests, and survival among patients who got cyclophosphamide and patients who did not get this drug.

What did the researchers find?

Of the 103 patients, 69 had alveolitis and 34 did not. Thirty-nine of the patients with alveolitis got cyclophosphamide and 30 did not. Patients with alveolitis who got cyclophosphamide did better on the breathing tests and survived longer than those who did not get the drug. Side effects of cyclophosphamide treatment included hair loss, infection, and bleeding into the urinary tract.

What were the limitations of the study?

Patients were not randomly assigned to get or to not get cyclophosphamide. It is possible, therefore, that the doctors may have given the drug to the patients who had the best chances of doing well. Also, there were too few patients to be able to identify which types of patients with scleroderma lung disease were most likely to benefit from treatment with cyclophosphamide.

What are the implications of the study?

Patients with scleroderma lung disease involving inflammation of the lungs who get cyclophosphamide appear to have improvements in their breathing and live longer than similar patients who do not get this drug.





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