Therapy Update: Scleroderma Lung Study
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.
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