WEDNESDAY, Jan. 25 (HealthDay News) -- A new preliminary report
suggests that the active ingredient in Viagra, sildenafil, could
reduce the size of large growths that can disfigure the bodies of
children.
The findings could point to yet another use for the medicine,
which was first developed as a heart medication until researchers
noticed that it helped impotent men have erections. This time,
researchers stumbled upon an alternate use while using a
Viagra-like drug to treat a rare condition that causes high blood
pressure in the arteries that lead to the lungs.
There are caveats: The treatment is very expensive, the research
is only in its early stages, and the medication may not be a cure.
Still, the research raises the prospect that "we could treat some
of these little kids who have little or no hope," said report
co-author Dr. Alfred Lane, a professor of dermatology and
pediatrics at Stanford University School of Medicine.
The growths in question are known as severe lymphatic
malformations. They appear in children, including babies, and
create disfiguring growths of fluid and vessels.
The growths can be as big as a volleyball or a basketball, Lane
said. They seem to appear when the lymphatic system, a component of
the body's immune system, becomes clogged, although the exact cause
isn't clear, he said.
In some cases, the growths can be dangerous, such as when they
pose a risk of blocking an airway pressuring a nearby organ.
Surgery to remove the growth is one option, although it may not
be possible, he said. For some children, "there's not a whole lot
you can do about it."
That's where sildenafil may help.
Researchers used a form of the medication called Revatio to
treat a baby girl who suffered from pulmonary hypertension, the
condition that causes high blood pressure in certain arteries. The
investigators found that the medication had another effect: it
reduced the size of a lymphatic growth.
The child, who was severely ill, died. But researchers were
curious about the effects of the drug, and they tried it on two
other children. Their growths shrunk and became softer after 12
weeks.
The parents of the children decided to continue giving the drug
to their kids; it's not clear how they're doing now, but Lane will
see one of the patients soon.
The drug may not eliminate a growth, "but if it can reduce it to
the size that they can remove it, that would be good," Lane
said.
Revatio costs $800 to $1,000 a month, Lane said, although the
Pfizer drug company is donating the drug for research purposes.
While the dose is low, potential side effects include dizziness,
eye problems, nosebleeds and nausea, Lane said.
Researchers don't know how the drug works to reduce the size of
the growths, Lane noted, although one possibility is that it makes
it easier for the lymph system to drain fluid.
A new study of the treatment is underway.
Dr. Richard Smith, a pediatric otolaryngologist who's familiar
with the report, said it offers an "exciting and serendipitous
finding." But it must still be validated to prove that it truly
holds promise, said Smith, vice chair of the University of Iowa's
department of otolaryngology -- head and neck surgery.
The report appears in the Jan. 26 issue of the
New England Journal of Medicine.
More information
Children's Hospital Boston has details about
lymphatic malformations.