When should experimental surgery end?
When should experimental surgery end?
Experts divided over motives behind lung study
A study intended to examine an experimental surgery has instead ignited a debate over the ethics of withholding medical treatment to critically ill patients.
The large, multicenter National Emphysema Treatment Trial (NETT), cosponsored by the Health Care Financing Administration (HCFA), and the National Institutes of Health (NIH), is designed to evaluate a relatively new surgical procedure that holds the promise of improved quality of life for emphysema sufferers.
The lung study will last five years, but in just the first year of enrollment, the trial has yielded significant information in the larger debate over whether medical treatment should be withheld from critically ill patients for the sake of scientific scrutiny.
NETT's genesis began as a result of HCFA's discovery that surgeons had been billing an experimental procedure known as lung-volume reduction surgery using a variety of lung surgery codes. Some clinical studies showed promising benefits for patients, but others showed mortality rates as high as 26%. In response to conflicting results, HCFA cut off all payments for the procedure in 1995, announcing it would pay for the surgery only for those patients participating in the large-scale clinical trial that began enrolling participants last year.
The trial, expected to enroll more than 4,000 emphysema patients, randomly assigns participants to either receive aggressive pulmonary rehabilitation and nonsurgical medical treatment or lung-volume reduction. Participants will be followed for five years, with data on survival, quality of life, exercise capacity, and pulmonary function collected every six months.
Ethical concerns, however, have overshadowed the design of the study. Some prominent investigators, including a surgeon who pioneered the lung-reduction procedure, withdrew from the trial citing ethical objections to withholding the procedure from patients they feel would certainly benefit.
In fact, one managed care organization decided not to wait for the government results and initiated a study of its own. Blue Cross Blue Shield of Massachusetts has sponsored its own trial, the Overholt-Blue Cross Emphysema Surgery Trial (OBEST), which has a similar design to NETT but will allow participants in the nonsurgical group to opt for surgery after six months. (For more on the OBEST study, see related story, p. 100.)
Experts on both sides of the ethical debate agree that lung-volume reduction surgery itself needs further clinical examination. Patients with severe emphysema have thin oversized inelastic lungs that lack the ability to expel air after each breath. The operation involves removing lung tissue to reduce patients' lungs back to a more normal size.
In 1994, St. Louis surgeon Joel Cooper, MD, announced success in treating some emphysema patients with the procedure, which was first performed by surgeon Otto Brantigan 30 years ago. Shortly afterward, surgeons across the country began operating to reduce patients' lung size, often without a clear understanding of what they were doing.
"It was being done helter-skelter throughout the country, often with terrible results, by people who had no idea what they were doing. People died," says Robert Keenan, MD, vice chief of cardio-thoracic surgery at the University of Pittsburgh and the center's principal investigator for the NETT trial.
Keenan argues that the trial is both ethical and necessary to prevent critically ill patients from spending thousands of dollars to undergo a dangerous procedure that may not help them and could end their lives.
Keenan believes the surgery improves the condition of certain emphysema patients, but that most doctors can't reliably discern which patients will do well with the procedure and which patients won't.
"I've done over 300 lung-reduction procedures," he states. "All of us who have experience with lung-reduction surgery, we all know many individuals who have clearly been helped by this procedure. We have also had individuals who, upfront, we felt would do well with surgery, and they didn't."
The trial, Keenan says, will determine which type of patient should be offered the surgery and under what conditions the surgery should be performed.
"We need guidelines or parameters on the patients who could be offered the surgery, the sort of patients who shouldn't be offered the surgery, and the conditions under which you should be allowed to do the surgery, in terms of anesthetic care, respiratory care, nursing care, and those kinds of things," Keenan adds.
"If this is a procedure that can be done only in a couple of hospitals across the country, then that's important to know."
Richard Fischel, MD, formerly the co-principal investigator for the University of California-Los Angeles' arm of NETT, resigned from the study due to concerns about the patient population and about randomizing patients who he feels would expect do well with the surgery.
Fischel, who has published studies on the procedure along with Joel Cooper, says that researchers for HCFA and NIH compiled his data along with that of other, dissimilar studies of lung-volume reduction and concluded there was not enough evidence to show a benefit for the procedure. Cooper also resigned from the study.
There is evidence to show benefit for a very select group of patients, contends Fischel. And, there is also evidence that some emphysema patients will not do well with the procedure.
"Then, there are some patients we just don't know about," he says. "For those patients, we need more data."
However, Fischel says, the NETT trial did not adequately limit its inclusion criteria for the study, and consequently, he believes, the end result will be that there is insufficient evidence to support performing the procedure.
Some experts argue that HCFA is predispositioned to limit access to the surgery because it is so expensive. The operation costs at least $30,000. If it were performed on every emphysema patient that some estimates say will benefit, the tab could exceed $6 billion.
Participating doctors hesitant to criticize
Fischel also argues that many surgeons at centers participating in the trial are reluctant to criticize the study's design because they don't want to lose funding for the trial or the procedure if it is approved. Others contend that hospitals not participating in the study can now offer the surgery to self-pay and private pay patients, while Medicare patients must wait.
Unlike randomized clinical trials involving new drugs, patients involved in the trial of a new surgical procedure know immediately which group they are in.
Patients in both trials are briefed extensively on the design and intent of the study and the risks and benefits of both surgery and nonsurgical treatment, says Keenan. Then all patients receive a course of pulmonary rehabilitation and medical treatment.
Participants are then randomized to either receive surgery or continue with pulmonary rehab and medical management. A primary concern among investigators is the impact to the studies if a significant number of participants drop out of the study after being assigned to a particular group.
"That is a concern, but it is a risk in any trial," Keenan states. "You just hope to weed those people out beforehand."
Another sticking point for some investigators is the unbiased way in which they are required to present information to prospective enrollees.
"Basically, I have to look patients straight in the eye and tell them I don't know which will be better for them, surgery or nonsurgery," explains Fischel. "With many patients, I simply cannot do that."
Overall care should improve
Keenan emphasizes that, in many cases across the country, patients have not had access to accurate information about the real risks and benefits of the surgery. A brochure he keeps in his office from another facility proclaimed, "Now there is a cure."
"There is no cure," he states. "This may make your life better, but it will not necessarily make it longer What if I promise to do this procedure, promise that it will improve your lung capacity, improve your ability to function, and what if it doesn't? What if we don't make you better? What if, in fact, we make you dead? Then, we have not helped. It's not the same thing as someone with a ruptured bowel, and he or she is going to die if you don't operate."
Some critics of the lung study have unfairly compared the favorable results of some patients who have undergone the lung procedure with emphysema patients who have had little or no pulmonary rehabilitation, says Keenan. "In many states, pulmonary rehab was not covered by Medicare, either," he states.
The NETT trial also should provide investigators with more complete information about which patients will do well with aggressive, nonsurgical treatment, he adds.
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