Cholesterol Drugs Address
Childhood Disorder
Early Studies
Provide Hopeful News
For some unlucky people,
high cholesterol is passed from one generation to the next,
but children who inherit the disorder may benefit from
cholesterol-lowering drugs known as statins, according
to a report in the Journal of the American Medical
Association.
Children who have
this inherited disorder, known as familial hypercholesterolemia,
are at great risk of having a heart attack as early as their
mid-20s.
"They develop coronary
artery disease at a relatively young age," explains Dr. Albert
Wiegman, a pediatric cardiologist at the Academic Medical Center
in Amsterdam, the Netherlands.
But the class of cholesterol-lowering medications
called statins holds promise for these children.
Statins lower
cholesterol by slowing down the production of cholesterol and
by increasing the liver's ability to remove the LDL-cholesterol
already in the blood.
Study
Looks At One Statin Medication
Under normal conditions,
people inherit two genes responsible for making LDL receptors
- the "fishing rods," as Dr. Wiegman describes them - that remove
cholesterol from the blood.
But in people with
familial hypercholesterolemia, an abnormal gene is passed along.
As a result, half
of their LDL receptors are defective or missing, according
to the organization called MEDPED (Make Early
Diagnosis to Prevent Early Death), a Salt Lake City-based
nonprofit organization that works to help families with inherited
high cholesterol disorders.
The condition results
in abnormally elevated levels of LDL cholesterol beginning at
birth.
An estimated 10 million
people worldwide suffer from the condition, resulting in premature
heart disease and death if not diagnosed and treated properly,
says MEDPED.
"You have to prevent
this disorder as early as possible, in my opinion," Dr. Wiegman
says.
The two-year
study found that children who took the medication pravastatin
had significant improvement in the thickness of the walls of
their carotid arteries, which supply blood to the brain. They
also had sharply reduced levels of low-density lipoprotein (LDL)
cholesterol, the "bad" kind that clogs vessels.
In addition, the medication
worked without any apparent adverse effects on the children's
growth, sexual maturation, hormone levels, or liver or muscle
tissue - at least over the period of the study.
"It seems to be safe
and it works," says Dr. Wiegman, lead author of the study.
"It makes the vessel walls thinner, and perhaps they [will]
have a normal life expectancy."
In an editorial in
the same journal, cardiologist and statin expert Dr. Antonio
M. Gotto Jr., dean of the Weill Medical College of Cornell University
in New York City, concludes the benefit of treating high-risk
youngsters with statins exceeds the potential risk.
"In the case of familial
hypercholesterolemia," Dr. Gotto writes, "the promise of
reducing future cardiovascular morbidity and mortality, as well
as future demands on acute care and more expensive medical approaches,
would make aggressive treatment of high-risk youth patients
a worthwhile long-term initiative."
Study
Findings Were Significant
The study involved
214 children, ages eight to 18, who were followed for two
years. The researchers encouraged every participant to exercise
and eat a fat-restricted diet. Half of the children were given
either 20 milligrams or 40 milligrams of pravastatin, depending
on the child's age, and the other half received a placebo (inactive
substance).
Before-and-after ultrasound
images were taken to measure the change in the thickness of
the walls of patients' carotid arteries, a marker for the fatty
plaque build-up that leads to atherosclerosis.
After two years, children
on the placebo had increased arterial wall thickness, while
children on pravastatin showed significant regression.
LDL cholesterol levels
dropped 24.1 percent in the statin group but rose 0.3 percent
in the placebo group.
"That's promising
because the LDL cholesterol-lowering, we could expect, but it's
important that it does something to the vessel walls," Dr. Wiegman
notes.
Although the study
is believed to be the most extensive yet, longer studies are
needed to demonstrate the long-term safety and efficacy of statin
therapy in children, the authors said.
Always consult your
child's physician for more information.
Online
Resources
(Our Organization
is not responsible for the content of Internet sites.)
American
Academy of Pediatrics
American
Heart Association
Centers
for Disease Control and Prevention (CDC)
Centers
for Disease Control and Prevention (CDC) 5 A Day
National
Health and Nutrition Examination Survey
National
Institute of Child Health & Human Development
National
Institutes of Health (NIH)
US
Food and Drug Administration |
September 2004
Cholesterol
Drugs Address Childhood Disorder
Study
Looks At One Statin Medication
Study
Findings Were Significant
Cholesterol
Goals
Online
Resources
Cholesterol
Goals
According to the National
Institutes of Health (NIH), to reach an LDL-cholesterol
goal of less than 100 mg/dL, a person may need to
take a cholesterol-lowering medicine in addition to making life
habit changes.
Heart disease patients
and those at high risk for developing heart disease need to
lower their LDL more than other people.
As a result, medications
are more often used by patients with heart disease and those
at high risk than by those who do not have heart disease or
who have a lower risk of developing it.
If an individual
has an LDL level of 130 mg/dL or greater, an LDL-lowering medicine
is often recommended, the NIH states.
If the LDL level
is 100 to 129 mg/dL, a physician will consider all
the facts of a case in deciding whether to prescribe medication
for further LDL lowering or for high triglycerides and/or low
HDL if they are present.
If a person has been
hospitalized for a heart attack, a physician will
likely start a patient on a medication at discharge if LDL-cholesterol
is 130 mg/dL or greater.
If LDL-cholesterol
is between 100 and 129 mg/dL during hospitalization, the physician
may choose to start you on an LDL-lowering medication before
discharge.
Also, if LDL-cholesterol
is far above the goal level of less than 100 mg/dL when first
measured, the physician may choose to start a cholesterol-lowering
medication together with diet and physical activity right from
the beginning of treatment.
The NIH
points out that if a physician prescribes medication, a person
most likely will need to:
-
Follow a cholesterol-lowering
diet.
-
Be more physically active.
-
Lose weight if overweight.
-
Control all of other heart
disease risk factors, including smoking, high blood pressure,
and diabetes.
Taking all these steps
together may lessen the amount of medicine needed or make the
medicine work better - and that reduces risk for a heart attack.
There are currently
five statin drugs on the market in the US used for adults:
The major effect of
the statins is to lower LDL-cholesterol levels, and they lower
LDL-cholesterol more than other types of drugs. Statins inhibit
an enzyme, HMG-CoA reductase, that controls the rate of cholesterol
production in the body.
These medications
lower cholesterol by slowing down the production of cholesterol
and by increasing the liver's ability to remove the LDL-cholesterol
already in the blood.
The statins are usually
given in a single dose at the evening meal or at bedtime. It
is important that these medications be given in the evening
to take advantage of the fact that the body makes more cholesterol
at night than during the day.
A person often sees
results from the statins after several weeks, with a maximum
effect in four to six weeks. After about six
to eight weeks, a physician can do the first check of LDL-cholesterol
while a person is on the medication. A second measurement of
LDL-cholesterol level will have to be averaged with the first
for a physician to decide whether a dose of medication
should be changed to help meet a goal.
Always consult your
child's physician for more information. |
