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DURHAM, N.C. - Medical researchers hope that an unusual
clinical trial will prove whether or not removing portions of
lungs can provide long-term benefit to patients severely
disabled by emphysema, a progressive lung disease for which
there is no other cure.

The trial's findings are both medically and economically
important, researchers say, because it is estimated that there
are currently more than 10 million Americans with emphysema and
the procedure costs $50,000 to $60,000. Most of these patients
are usually elderly and disabled and are covered by Medicare,
the federal insurance program.

Medicare had paid for thousands of the procedures in the
early 1990s, when many hospitals began offering it to
enthusiastic patients. Although short-term benefits were
observed in many patients, important unanswered questions
included the duration of benefit, which patients were most
suitable, and what were the potential short- and long-term
complications, researchers said.

The National Emphysema Treatment Trial, which is now
enrolling the first of a planned 4,700 patients, is being
carried out in 18 centers across the country. Patients will be
enrolled for three to five years, and will be followed for at
least five years.

Duke University Medical Center is the only hospital in the
Southeast participating in the trial. Funded jointly by the
National Heart, Lung, and Blood Institute (NHLBI) and the
Health Care Financing Administration (HCFA), the agency that
handles Medicare claims, this is the largest clinical trial the
two federal agencies have combined to sponsor.

"This can be the model of how Medicare and other third-party
payers can work together with researchers to evaluate new
procedures," said pulmonologist Dr. Neil MacIntyre, Duke's
principal investigator in the trial. "Instead of just deciding
arbitrarily whether or not a specific procedure or therapy is
effective and should be covered, the government is funding a
trial to actually find out the answer.

"Because of the large numbers of patients and way the trial
has been designed, we should get definitive answers on the role
of this procedure." In the procedure, known as lung volume
reduction surgery (LVRS), surgeons remove portions of the lung
that have become overinflated from the disease. This allows
undiseased portions of lung - which had been compressed by
diseased lung tissue - to expand and function properly. The
removal of the inflexible portions of lung also improves the
ability of the diaphragm to function in a more normal manner,
MacIntyre said.

The first surgeries were performed in the early 1950s and
although some patients regained a degree of lung function, one
in six patients died. Because surgeons had difficulty
controlling air leaks after surgery, the procedure fell out of
favor. However, with the advent of improved surgical
techniques, surgeons revisited the procedure in the early
1990s.

"Studies involving small numbers of patients in the surgical
literature showed significant improvements in lung function
tests and exercise tolerance, with early mortality rates
between 2 percent and 7 percent," MacIntyre said.

When success stories began appearing in the popular press
about the benefits of the procedure, many emphysema patients
demanded it. By 1995, many hospitals jumped on the bandwagon
and offered the new surgical approach to their emphysema
patients.

As the popularity of LVRS grew, so did the number of claims
submitted to Medicare for reimbursement. In 1992, between
200-300 claims were submitted for the procedure; by 1995, the
number had jumped to more than 2,000.

"For obvious reasons, HCFA began tracking claims," MacIntyre
said. "After studying 711 consecutive claims, they found that
26 percent of the patients were dead after one year, there was
a 40 percent rehospitalization rate, and 16 percent needed
long-term care.

"Clearly, there was quite a difference between what was
being reported in the literature and what HCFA found." So in
January 1996, HCFA halted reimbursements for LVRS, and called
for a large-scale trial to determine the effectiveness of the
procedure and to determine which patients would benefit the
most from surgery. In December of last year, HCFA and the NHLBI
announced the trial and the 18 participating centers, who have
worked for the past year designing the trial.

"It is obvious to us that while many patients may benefit
from the procedure, patients could also be harmed," MacIntyre
continued. "It is important to sort these things out before
making a blanket statement about the benefits of LVRS."

To be eligible for the trial, patients must have moderate to
severe emphysema, have no other complicating medical problems,
and must have quit smoking for at least four months. The vast
majority of these emphysema cases are the result of smoking,
MacIntyre said.

"In general, these patients get short of breath at mild
exertion, some are on oxygen, and most are unable to carry out
the routine activities of everyday life," MacIntyre said. "It
is truly a debilitating disease for which little can be
done."

Patients must be Medicare-eligible to participate in the
trial. Many hospitals, including Duke, continue to offer the
surgery, but patients may have to pay for it out of their own
pocket, because many insurers do not cover the operation. Like
the federal government, they are awaiting the outcome of the
trial, MacIntyre said.

All participants in the trial will receive intensive medical
therapy and rehabilitation and half will be selected by random
for LVRS. Those picked for surgery will be further separated
randomly into two different surgical approaches - either by
median sternotomy (open chest) or by video assisted thoracic
surgery (VATS). At Duke, Dr. Duane Davis will perform the open
surgery, while Dr. David Harpole will use the VATS
approach.

Duke pulmonologist Dr. Victor Tapson and study coordinator
Abby Krichman will evaluate potential patients and will be in
charge of the medical care of study participants, while
MacIntyre serves as director of the Duke pulmonary
rehabilitation program.

Study participants will be evaluated for exercise ability,
lung function, quality of life, illness and survival. Although
it has been estimated that it will take seven years to complete
the trial, treatment recommendations may be made sooner if the
data, which will be reviewed at regular intervals by an
independent panel of experts, show clear-cut benefits or
risks.

"If it is determined that the surgery is clearly beneficial,
those patients who had been randomized to maximum medical care
alone will be offered the surgery immediately," MacIntyre said.
"Likewise, if the surgery is doing more harm than good, the
trial will be stopped."

Current treatment for patients in the final stages of
emphysema is largely supportive, including nutritional
supplementation and exercise rehabilitation. Lung
transplantation may be an option for some patients under age
60, but donor lungs are scarce, and the procedure is
expensive.