| Media Availability: Drug Found to Help Scleroderma Patients
Scleroderma is a progressive condition in which fibrous tissue
grows abnormally, causing the skin to thicken and harden, often
disfiguring and disabling patients. In a life-threatening form of
the disease known as systemic sclerosis, extra tissue also damages
internal organs. Lung tissue is affected in about 80 percent of
these patients, about half of whom die within 10 years as a result.
Overall, scleroderma affects an estimated 300,000 Americans, most
of them women.
For the first time, a randomized clinical trial has proven that
a drug can slow down deterioration of lung function in scleroderma
patients. When tissue in the lungs hardens or scars, breathing becomes
difficult. In the study, participants who received treatment had
small but statistically significant improvement in lung function
(about 3 percent) and less shortness of breath (dyspnea) compared
to those who were not treated. The drug also decreased the extent
of skin thickening. Furthermore, treated participants reported feeling
healthier and more energetic.
The Scleroderma Lung Study was funded by the National Heart, Lung,
and Blood Institute (NHLBI) of the National Institutes of Health
(NIH), with support from the NIH’s National Institute of Arthritis
and Musculoskeletal and Skin Diseases (NIAMS).
In the study, 158 scleroderma patients with early signs of lung
problems were randomly assigned to daily treatment with cyclophosphamide
(Cytoxan), a pill commonly used to treat cancer patients, or to
placebo for one year. Participants were assessed periodically for
an additional year after treatment. Negative side effects, such
as low white blood counts and complications from infection, were
more common among participants treated with the drug; however, these
problems were sufficiently managed by adjustments in dosage. The
researchers conclude that the benefits of treatment appear to outweigh
the risks.
“Cyclophosphamide versus Placebo in Scleroderma Lung Disease,”
by Donald P. Tashkin, M.D., of the University of California at Los
Angeles Medical Center and others, will be published in the June
22 issue of the New England Journal of Medicine.
NIH experts in lung diseases and disorders, as well as experts
in connective tissue disorders, are available to comment on the
study’s findings and the disease. Scleroderma typically occurs
between the ages of 30 and 50 years, most commonly among women.
The average age of study participants was 48 years, and 71 percent
of participants were women.
To schedule an interview with a lung disease expert, call the NHLBI
Communications Office at (301) 496-4236. To interview an expert
on connective tissue disorders, call the NIAMS Communications Office
at (301) 496-8190.
Resources
Handout
on Health: Scleroderma
(http://www.niams.nih.gov/hi/topics/scleroderma/scleroderma.htm)
Scleroderma
Lung Study
(http://sclerodermalungstudy.medsch.ucla.edu/)
The National Heart, Lung, and Blood Institute (NHLBI) and the
National Institute of Arthritis and Musculoskeletal and Skin Diseases
(NIAMS) are components of the National Institutes of Health. NHLBI
plans, conducts, and supports research related to the causes, prevention,
diagnosis, and treatment of heart, blood vessel, lung, and blood
diseases; and sleep disorders. The Institute also administers national
health education campaigns on women and heart disease, healthy weight
for children, and other topics. NHLBI press releases and other materials
are available online at: www.nhlbi.nih.gov.
The mission of NIAMS 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 additional information, call NIAMS’s
Clearinghouse toll free at 1-877-22-NIAMS, or visit the NIAMS Web
site at www.niams.nih.gov.
The National Institutes of Health (NIH) — The Nation's
Medical Research Agency — includes 27 Institutes and Centers
and is a component of the U. S. Department of Health and Human Services.
It is the primary federal agency for conducting and supporting basic,
clinical, and translational medical research, and it investigates
the causes, treatments, and cures for both common and rare diseases.
For more information about NIH and its programs, visit http://www.nih.gov.
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