An Expensive New Heart Implant Offers Hope to Seriously Ill Patients

By RON WINSLOW  Staff Reporter of THE WALL STREET JOURNAL  Nov 13. 2001

ANAHEIM, Calif. -- A medical device that boosts the heart's pumping capacity can significantly prolong the lives of patients with severe heart failure and act as an alternative to heart transplants, according to a major study.

The findings offer new hope to an estimated 100,000 patients each year who aren't eligible for a donated heart, but whose illness leaves them so debilitated they are short of breath while sitting in a chair or too tired to talk on the telephone. Researchers found that the implantable pump reduced the chance of death by nearly 50% within a year and was associated with significantly improved quality of life when compared with patients who got the best available medical care without the device.

The pump, known as a left ventricular assist device, or LVAD, is one of several devices already on the market that serve as a temporary "bridge to transplant" to keep patients alive while they wait for a heart from a donor. But fewer than 3,000 hearts world-wide are donated for transplant each year and doctors have long wondered whether LVADs could be used as long-term therapy for the tens of thousands of patients who have no other options.

"That's a closed issue now," says Eric Rose, a transplant surgeon at Columbia University's College of Physicians and Surgeons in New York and lead investigator of the trial. "This is a bona fide treatment for these very sick patients." Dr. Rose presented the data at the annual Scientific Sessions of the American Heart Association here. The study is also being published this week in the New England Journal of Medicine.

The new treatment comes with daunting economic challenges for the health-care system. Called the HeartMate VE, and marketed by Thoratec Corp. of Pleasanton, Calif., the device costs about $60,000; when the operation to implant it is added, the entire procedure can run between $100,000 and $200,000. The potential market for the device could reach $6 billion a year while the total costs for treating such patients with LVADs could exceed $10 billion or more. Much of that cost would be financed by the federal Medicare program since most heart-failure patients are 65 years old or older. Heart transplants typically cost $175,000 to $200,000.

A new study shows that the LVAD heart pump, once considered to be a temporary 'bridge' to a transplant, can also be used long-term.

"These results are really magnificent," says Valentin Fuster, director of the Cardiovascular Institute at Mount Sinai Medical Center in New York, who wasn't involved with the study. "But who will pay?"

Even with such concerns, the results are expected to fuel enthusiasm for a variety of new mechanical heart devices now under development at several companies. The most prominent example is the total artificial heart being developed by Abiomed Inc. of Danvers, Mass., which was implanted in a fifth patient this month. Other devices, like the HeartMate VE, essentially take over the function of the left ventricle, the heart's main pumping chamber. Yet other models are intended to assist the heart by taking over some, but not all, of the pumping action.

Over the next several years, researchers say, doctors will learn more about what kind of device works best in which patients and how best to manage the patients' care.

"It's wonderful to have this therapy and to be able to offer it," says Lynne Stevenson, a heart failure expert at Brigham and Women's Hospital in Boston and a co-author of the new study. "But it's our responsibility to carefully choose the best ways in which to offer it."

The study encompassed 129 patients with end-stage heart disease who were randomized to get either the device or optimal standard medical treatment. The results offered sobering evidence of just how sick such patients are. Dr. Rose said 75% of the patients who didn't get the LVAD died within a year, compared with 48% of those who received the device. Just 8% of those getting the standard treatment were alive after two years, compared with 23% of those who received the pump.

Patients given the device had a median survival of 408 days compared with 150 days for those on the best available therapy, the researchers found.

But the survival and other benefits associated with the LVAD came at a price, Dr. Rose pointed out: high complication rates related mostly to infections and failure of the device. They were the most important reasons why 77% of patients who got the device died after two years.

Dr. Rose said early experience during the study, which began in 1997, contributed to alterations in the design of the device as well as adjustments in the care of patients. For instance, doctors learned that many of the patients were malnourished, leaving them less able to heal wounds around wires that connect the implanted device to an external controller that can be worn on a belt. As a result, patients who enrolled later in the study appeared to have a higher survival rate. Dr. Rose said such lessons should continue to lead to better results for patients.

Sharon Hunt, a cardiologist at Stanford University School of Medicine, cautioned that with such a high death rate, it may take both doctors and third-party payers a while to accept the treatment as a reasonable alternative to a heart transplant. Still, she calls the report a "landmark" that will become the benchmark against which future trials of similar devices will be judged.

As part of the study, researchers are conducting a detailed cost analysis of the device. Dr. Rose believes that despite its big-ticket price, the procedure will be comparable to many widely covered treatments when the benefits in terms of prolonged and improved quality of life are taken into account.

The device, developed by a company that Thoratec recently bought, is similar in size to a portable compact disc player. It is implanted into the abdomen under the heart. Tubes attached to the pump connect outside the body to a small battery pack worn as a shoulder holster and a controller worn like a beeper on a belt.

Thoratec announced last month it had submitted the study's results to the Food and Drug Administration, seeking approval of the device for long-term use. Such approval is crucial to getting reimbursement from Medicare and other insurers, says D. Keith Grossman, president and chief executive of Thoratec. Thoratec's stock fell $1.39 a share to $18.50 Monday in Nasdaq Stock Market trading.

About five million Americans suffer from congestive heart failure and 550,000 new cases are diagnosed each year. It already is one of the most expensive ailments for Medicare, with annual treatment costs estimated as high as $40 billion. Those who are likely to be candidates for mechanical heart devices are among the very sickest of these patients

Abstract of article at New England Journal of Medicine

Long-Term Use of a Left Ventricular Assist Device for End-Stage Heart Failure

Notice: This article is being published at www.nejm.org on November 12 to coincide with a presentation at the Scientific Sessions 2001 of the American Heart Association. It will appear in the November 15 issue of the Journal. Click on "PDF of this article" for the full text.

Background Implantable left ventricular assist devices have benefited patients with end-stage heart failure as a bridge to cardiac transplantation, but their long-term use for the purpose of enhancing survival and the quality of life has not been evaluated.

Methods We randomly assigned 129 patients with end-stage heart failure who were ineligible for cardiac transplantation to receive a left ventricular assist device (68 patients) or optimal medical management (61). All patients had symptoms of New York Heart Association class IV heart failure.

Results Kaplan--Meier survival analysis showed a reduction of 48 percent in the risk of death from any cause in the group that received left ventricular assist devices as compared with the medical-therapy group (relative risk, 0.52; 95 percent confidence interval, 0.34 to 0.78; P=0.001). The rates of survival at one year were 52 percent in the device group and 25 percent in the medical-therapy group (P=0.002), and the rates at two years were 23 percent and 8 percent (P=0.09), respectively. The frequency of serious adverse events in the device group was 2.35 (95 percent confidence interval, 1.86 to 2.95) times that in the medical-therapy group, with a predominance of infection, bleeding, and malfunction of the device. The quality of life was significantly improved at one year in the device group.

Conclusions The use of a left ventricular assist device in patients with advanced heart failure resulted in a clinically meaningful survival benefit and an improved quality of life. A left ventricular assist device is an acceptable alternative therapy in selected patients who are not candidates for cardiac transplantation. (N Engl J Med 2001;345:1435-43.)