Spotlight on Research 2007

October 2007 (historical)

Oral Cyclophosphamide Improves Quality of Life in Patients with Scleroderma Lung Disease

A recent study of patients with scleroderma-related lung disease found that one year of treatment with the immunosuppressive drug cyclophosphamide (CYC) significantly improved health-related quality of life (HRQOL), as reported by the patients. Earlier reports from this same investigation noted an improvement in lung function as well. The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and the National Heart, Lung, and Blood Institute (NHLBI) supported this research.

The research article, written by Dinesh Khanna, M.D., of the University of Cincinnati, Ohio, reported that of the 158 patients enrolled, 47 percent of patients treated with CYC rated their health as somewhat or much better after one year compared to 18 percent of the patients in the placebo group. Complementing the patient-reported improvements, physical tests for breathing ability showed modest improvements in the CYC-treated group.

The paper featured a recent advancement in HRQOL, the "minimum clinically important difference (MCID)," which relates the smallest improvements that patients perceive as beneficial and may lead to a change in disease management. The MCID adds a "fine tuning" factor to help determine whether statistically meaningful differences between baseline scores (those at the beginning of a study) and later scores are clinically significant.

The measurement tools discussed in this article represent an important move forward in public health and research. The MCID makes it even clearer when/if a treatment is significantly effective. This study's outcomes pave the way for future trials to better evaluate new treatments and medicines in the full context of patient preferences, decisions, and risk assessment. These dimensions are particularly important for patients with severe and chronic conditions.

The researchers indicate that they will be following these patients in a second year of study. In addition to NIH funding, the research is supported by the Arthritis Foundation and the Scleroderma Foundation.

Scleroderma, also called systemic sclerosis, is a disabling autoimmune disease characterized by hardening of tissues in many parts of the body, including skin, internal organs and blood vessels. In as many as 70 percent of patients with this disorder, there is scleroderma-related lung impairment, which is difficult to treat and may lead to death.

The mission of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), a part of the Department of Health and Human Services' National Institutes of Health, is to support research into the causes, treatment and prevention of arthritis and musculoskeletal and skin diseases; the training of basic and clinical scientists to carry out this research; and the dissemination of information on research progress in these diseases. For more information about NIAMS, call the information clearinghouse at (301) 495-4484 or (877) 22-NIAMS (free call) or visit the NIAMS Web site at

The National Heart, Lung, and Blood Institute (NHLBI) provides leadership for a national program in diseases of the heart, blood vessels, lung and blood; blood resources; and sleep disorders. Since October 1997, the NHLBI has also had administrative responsibility for the Women's Health Initiative.


Khanna D, et al. Impact of oral cyclophosphamide on health-related quality of life in patients with active scleroderma lung disease: results from the Scleroderma Lung Study. Arthritis and Rheumatism, 2007;56(5):1676-1684.