Hypercholesterolemia

"When diet, exercise, and cholesterol-lowering medications don't work, what can one do to reduce ,the high risk of cardiovascular disease, heart attack, and stroke? For individuals suffering from a genetically inherited disorder called familial hypercholesterolemia (FH), there is new hope, An emerging technology offers an alternative to families with this genetic predisposition to high cholesterol, or FH.

"Lipid apheresis is a way to selectively remove the bad, or LDL, cholesterol," said Mark Deeg, M.D., Ph.D., director of the IU Vascular Health Program, professor of medicine at the Indiana University School of Medicine, and investigator in the Indiana Center for Vascular Biology and Medicine. "It functions like a dialysis machine in terms of what happens. You put a needle in one arm vein, the blood comes out, a series of filters takes out the fat, and the blood returns to the patient's other arm. It is that simple."

The FDA-approved treatment--used widely in Europe for the past 20 years--is now available at nearly 20 medical centers across the United States.

When the trials for the new therapy first began in the United States, Oklahoma native Patti Hatley was eager to enter the trial. Both of Patti's parents suffered from hypercholesterolemia and passed the inherited trait onto their daughter. Despite lifestyle and medications, Patti's total cholesterol reached as high as 789, with an LDL in the 500 range, leaving her future seriously in question.

"I have no lipid receptors at all in my body. I produce mass quantities of cholesterol, but then I can't get rid of it," Patti relates. "At 42, I had my first heart double bypass and left carotid surgeries. I was on all of the statin drugs as they were coming out, but none of those worked for me."

A year and one-half after Patti's surgery, the head of the research center in Oklahoma City called and told her about a new study coming to the United States from Germany. They thought Patti an ideal candidate for the trial, so she began treatment in 1989, becoming the second person in the United States to undergo the new therapy. For 14 years, Patti has undergone twice-a-month lipid apheresis treatments and remains grateful for the opportunity.

"It's given me a life. Without it, I would not be here," Patti says. "Usually people with my type of hypercholesterolemia with no receptors do not live past their 40s. I'm 58 and still kicking. I am the biggest advocate and poster child for lipid apheresis in the United States. I know that there are people out there who would die without this treatment."

To learn more about the benefits of lipid apheresis, the Post interviewed Dr. Deeg, associate professor of biochemistry and molecular biology at Indiana University and director of the new LDL Apheresis Program at Indiana University School of Medicine.

Post: Who is eligible for lipid apheresis?

Deeg: There are two major FDA-approved indications for this machine. The first includes people with LDL levels of 300 or more, despite medical therapy. Most of these people will have familial hypercholesterolemia. Another is therapy for patients with heart disease who are unable to reduce their LDL cholesterol levels below 200 with diet and medications. Most of these candidates also have familial hypercholesterolemia.

Post: Does insurance cover the treatment?

Deeg: Most private insurance companies cover the treatment. It is approved for Medicare coverage as well.

Post: Would individuals with FH then have to undergo lifelong therapy?

Deeg: Yes, unless a gene therapy becomes available someday. Patients are treated with apheresis every two weeks. We begin by inserting a needle in the arms. After a year or two, we place a small shunt in the forearm. After treatment with apheresis, cholesterol levels drop acutely, then rebound. By continuing on the statin or other cholesterol-controlling medications, people can limit how fast the levels come back up.

Post: How much does apheresis lower the LDL level?

Deeg: One treatment will acutely lower LDL level some 60 percent. Not only does the treatment lower LDL, it also lowers two other important risk factors for cardiovascular events. One is the inflammatory marker called C-reactive protein. The treatment also reduces fibrinogen and plasma viscosity. When you have a heart attack, a clot forms. By lowering fibrinogen levels, you reduce the ability to form a clot.

Post: How do people otherwise control hypercholesterolemia without

this new treatment?

Deeg: They pray and take many medications--the mainstay is statin drug therapy. In hypercholesterolemia, the problem is a defect in the LDL receptor in the liver. People with FH can make the LDL, but they can't get rid of it because of the defective receptor, a condition that is called reduced affinity. Most of the medications that we use are focused on up-regulating the receptors; however, they are up-regulating a defective receptor. So rather than being 100 percent efficient, the medications might be one or two percent efficient. There is still some positive effect, so statins, niacin, and other drugs that inhibit cholesterol absorption are used. Without apheresis, drug therapy is pretty much all you can do.

Post: Are researchers investigating the potential of this therapy to treat other conditions?

Deeg: Yes. Some very exciting research was recently published in the Lancet in regard to acute hearing loss. Another trial in process will help determine whether one-time apheresis improves outcome in patients with heart attacks and unstable angina. In addition, we are going to try to get a trial going at Indiana University using the therapy in cardiac transplantation patients who experience accelerated atherosclerosis.

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