Therapy Update: Scleroderma Lung Study 2005
Positive results for Scleroderma Lung Study
presented at American Thoracic Society Meeting by Dr. Donald Tashkin of
UCLA Medical Center and panel of experts. To read more about this study
please read the following newspaper articles.
(Please note that one of the newspaper articles states,
"About 150,000 Americans have scleroderma." This number refers
solely to those affected by systemic sclerosis and does not take into
account those Americans diagnosed with the localized form of the disease.)
- "Cancer Drug Helps Scleroderma Patients," by
Kathleen Doheny,
"HealthDay" reporter
- "Drug alleviates scleroderma; Disease of lungs and
connective tissues responds to anti-cancer medication," by Thomas
H. Maugh II, "Los Angeles Times."
"Cancer Drug Helps Scleroderma Patients,"
by Kathleen Doheny,
"HealthDay" reporter
TUESDAY, May 24, 2005 (HealthDay News) -- A drug used for leukemia and
certain other cancers also helps to treat the lung disease associated
with scleroderma, a debilitating connective tissue disease, new research
shows.
The drug, cyclophosphamide (Cytoxan, Neosar) improved the patients' breathing
problems and boosted their energy levels, said study author Dr. Donald
Tashkin, a professor of medicine at the David Geffen School of Medicine,
University of California, Los Angeles (UCLA).
Tashkin presented the research Monday at the American Thoracic Society's
international conference, in San Diego.
Scleroderma is a disease in which hardening of the skin is one of the
most visible manifestations, according to the Scleroderma Foundation.
About 300,000 people have the disease in the United States, according
to foundation estimates. The exact cause is not known, but the disease
can also affect the blood vessels and internal organs, Tashkin said.
"About 80 percent of these patients develop lung involvement,"
Tashkin said. Over time the lungs scar, making breathing difficult. This
scarring is believed to be caused by inflammation, in which an abnormal
number of white blood cells collect in the lungs, he explained.
The more severe the scarring, the higher the mortality rate, the UCLA
expert added, and more than half of deaths from the disease are due to
lung-related problems.
In the study, Tashkin and his colleagues assigned 162 patients with scleroderma,
average age about 48, to either a group that got the drug for a year or
another group that got a placebo. To be eligible for the study, the patients
needed to have significant shortness of breath, meaning they became short
of breath after walking just two or three flights of stairs, Tashkin said.
The team evaluated white blood cell counts and platelet counts, and checked
lung function.
Lung function improved significantly in those on the drug compared with
those taking placebo. "We also found a significant and meaningful
effect on improving shortness of breath," he said.
When the patients answered a health assessment questionnaire, those on
the drug also said they had more energy or "peppiness," he said.
How does the therapy work?
"Our initial hypothesis was that it suppresses inflammation,"
Tashkin said. "It may be doing more than that, we don't know. There
is controversy about whether or not the scarring is necessarily related
to the inflammation. I believe it is, but maybe not directly."
The drug probably has some additional, undiscovered effects, besides being
anti-inflammatory, Tashkin said.
The patients receiving the drug did have more serious adverse effects
than the placebo group: five people receiving the medicine got pneumonia,
compared to one in the placebo group. In each group, two patients died,
but not from drug toxicity, Tashkin said.
A spokeswoman for the Scleroderma Foundation praised the study. "That's
phenomenal," Carolyn Weller, vice president of education and research
for the foundation, said when told of the results about improved lung
function.
"That gives our patient population [treatment] options with early
lung disease," she added. Until now, the main option was prednisone,
a corticosteroid. "And staying on high dosages [of prednisone] for
long periods of time can have an effect on kidney function," she
said.
According to Weller, some doctors are already using cyclophosphamide
"off label" -- prescribing the drug for a purpose not specifically
approved by the U.S. Food and Drug Administration -- to treat the scarring.
"This study will give doctors the data they need to be able to prescribe
it on a wider basis," she said.
More information
To learn more about scleroderma, visit the Scleroderma Foundation www.scleroderma.org/medical/overview.shtm.
"Drug alleviates scleroderma;
Disease of lungs and connective tissues responds to anti-cancer medication,"
by Thomas H. Maugh II, "Los Angeles Times."
May 24, 2005 – An anti-cancer drug that suppresses the immune system
is the first drug that has been proved to alleviate the most devastating
effects of scleroderma, a disease of the connecting tissues and lungs.
Patients taking cyclophosphamide had a significant improvement in lung
function and a reduction in breathlessness. They also reported feeling
healthier and more energetic, Dr. Donald Tashkin of the University of
California, Los Angeles told a San Diego meeting of the American Thoracic
Society on Monday.
Until now scleroderma therapies have been based on "a sense of what
ought to work without very good evidence," said Dr. Athol Wells of
the Royal Brompton Hospital in London, who was not involved in the research.
The new study is the first controlled clinical trial to show a positive
scleroderma treatment, Tashkin said.
Scleroderma affects as many as 300,000 Americans, according to the Scleroderma
Foundation. Its cause is unknown, although there is some evidence of a
genetic link. Women are three to four times as likely as men to suffer
from it.
The disorder is marked by an immune system attack on connective tissues
that leads to scarring of the skin and internal organs, particularly the
lungs.
Symptoms can range from mild to very severe. Hardening of the skin is
one of the most visible manifestations. In some cases, the skin becomes
so rigid that patients have difficulty moving their limbs and opening
and closing their eyelids and mouths.
About 80 percent of patients develop scarring of the lungs, which makes
it difficult to breath and can be fatal. As many as half of patients with
lung scarring die within 10 years.
A broad variety of drugs have been tested against the disorder with little
benefit. The treatment does have side effects, which were anticipated
because it is a powerful anti-cancer drug, although it was used in lower
doses than are common in cancer treatment. The drug lowers white cell
count and platelet count, and the number of cases of pneumonia among recipients
was slightly higher than in the placebo group.
But scleroderma "has a devastating impact on quality of life in
its victims," Tashkin said, "and the risk-benefit ratio is weighted
strongly in favor of benefit."
The next step, Tashkin said, will be to combine cyclophosphamide with
other drugs to further improve lung function.
|