Weighing Cost and Pain of Marrow Transplant : Medicine: Bringing a patient to the brink of death to kill cancer raises question of how many patients have to be cured before it’s considered routine treatment.
PHOENIX — Doctors have long lamented a common roadblock to curing cancer: If they give enough medicine to destroy the tumor, they also kill the patient.
Now they are trying a new approach that stops just short of doing exactly that.
If it works, proponents say this technique will achieve one of cancer therapy’s most elusive goals. It will stop cancer that has already spread throughout the body.
But at a price.
The key to the new approach is a bone marrow transplant, one of the most daunting and expensive procedures in medicine. And because of the high cost, both in money and suffering, the health profession is asking some seldom-heard questions about what patients should be expected to endure and what society should be expected to spend.
At its heart, the idea is crude: Just give incredibly high doses of the toxic medicines known as chemotherapy. Over a few days, doctors inject 10, 20, even 30 times more chemotherapy than they ever would have dared in the past. When all goes well, they kill every last lurking cancer cell. But they also push the limit of what their patients’ hearts, livers and lungs can stand. And they destroy their bone marrow, the vital tissue that makes new blood.
After escorting their patients to the brink of death, doctors then “rescue” them, as they so literally put it, by giving back a bit of marrow that they saved ahead of time.
Doctors who have been testing this still-experimental approach in recent years say they hope it will allow them, at least occasionally, to turn back the relentlessly fatal spread of cancer of the breast, colon, skin, brain, lungs and ovaries, among other organs.
“It’s a horrendous procedure,” said psychologist Barrie Cassileth of the University of Pennsylvania. “It’s life-threatening, painful, debilitating and marginally successful, but some people will be cured. And that’s the dilemma. The heart of the matter is: What percentage of patients have to be cured before we consider this a routine treatment after others have failed?”
No one has a precise answer. If virtually everyone with this stage of disease now dies, what small chance of success must doctors be able to offer before they pull out this heaviest of medical artillery?
“There are those who would say, ‘If it’s 5%, you should do it,’ ” said Dr. William P. Vaughan of the University of Nebraska.
Doctors believe they have cured at least some otherwise doomed patients. But the success rate will almost certainly fall as they extend this approach to older and sicker people. It will also vary with the kind and stage of cancer.
Most patients treated so far are women with advanced breast cancer. Rigorously controlled studies are still going on, but Vaughan estimates that 30% of those with the best possible outlook in his institution’s program achieve “multiyear disease-free survival,” though it’s too soon to say how many are cured. An additional 10% die from the treatment itself. The rest go on to die from the cancer that would have killed them anyway.
Costs range from $100,000 to $250,000, depending on the number of complications and how long patients must stay in the hospital. Vaughan figures this works out to $1 million for each life saved.
Is a life worth this much? And what if patients gain, say, an extra two years of reasonably good life and then die of cancer? Are these years worth $250,000--or $1 million?
Although there are exceptions, health insurance companies refuse to pay for bone marrow transplants for breast cancer and other so-called solid tumors, because they consider the benefits unproved. But if studies, including one financed in part by Blue Cross and Blue Shield, show that marrow transplants save lives, this treatment will almost certainly be offered to tens of thousands of Americans. Insurance companies will pick up the bills.
“When you provide very expensive techniques for a relatively small number of people, you then make basic health coverage for many people more expensive,” said Dr. John Cova of the Health Insurance Assn. of America. “That has profound public health implications, because it makes access to the health care system more difficult.”
Just how much widespread use of bone marrow transplants would push up insurance rates is difficult to estimate, in part because the alternative--lingering death in a hospital--also is expensive.
David deCoriolis, research director at BCS Financial Corp., a Chicago health reinsurance firm, said that perhaps 30,000 to 40,000 of the 150,000 women who die each year from breast cancer might be considered for marrow transplants.
“What will it cost for every insured person?” DeCoriolis asked. “Less than one dollar annually. Is it worthwhile? The answer, probably, is yes.”
Bone marrow transplants have long been standard treatment for people with cancer of the blood-forming tissue inside the bones. Doctors kill the cancerous marrow and then put back disease-free marrow taken from a relative. If it works, the patient is cured.
During the early 1980s, doctors began experimenting with marrow transplants for people with other kinds of cancer. The idea was to blast away with as much chemotherapy as the body could stand. The marrow, which is especially sensitive to this treatment, would simply be sacrificed and replaced later. They called the approach autologous bone marrow transplantation, since it uses the patient’s own marrow rather than donor marrow.
One patient, a woman with seemingly terminal breast cancer at the Dana-Farber Cancer Institute in Boston, has been alive for eight years since her pioneering treatment. A few others have survived up to five years. Doctors believe these people almost certainly would have otherwise died.
These successes change the goal of caring for people with advanced cancer, which until now has done little more than delay the inevitable. At a recent meeting of the American Cancer Society in Phoenix, transplant doctors spoke of something far more ambitious.
“You can really talk about cure now, not just putting off the disease for a couple of years,” said Dr. Richard J. O’Reilly of Memorial-Sloan Kettering Cancer Center in New York.
But not without extraordinary sickness for many patients.
“It’s industrial strength,” said Dr. James A. Neidhart of the University of New Mexico. “But about half of the time, people taking the regimen can function rather normally. The rest of it is pure courage.”
First, doctors draw out blood-forming tissue from the patients’ marrow or bloodstream. Then over several days they give them seemingly fatal doses of chemotherapy. Such large amounts produce even worse nausea, vomiting and diarrhea than ordinary cancer treatment.
Now, their bone marrow is dead. They are unable to make platelets to stop bleeding, and without new white blood cells they are completely defenseless against infection. Doctors put back some of the saved bone marrow, but it takes two weeks to a month to grow back enough to restore their blood supply.
In the meantime, patients must stay in isolation. They are at risk of direct damage to their organs from the chemotherapy. Bleeding, especially inside the head, can be fatal. The drugs cause sores in their mouths and ulcers throughout their digestive tracts. These openings allow germs to get into their bodies at just the time when they cannot resist them.
Patients are usually given multiple antibiotics, but most still get infections, which can cause extreme illness. In the worst cases, they require respirators to breathe and dialysis to take over for their kidneys.
While they start to feel better within a few weeks, many complain of lasting fatigue, and doctors say patients often need six months to a year to feel normal again.
“This is a treatment where my patients tell me they didn’t know they could feel so bad,” said Dr. Karen Antman of Dana-Farber.
Dr. Mary B. Daley of the Fox Chase Cancer Center in Philadelphia said that unlike most cancer research, formal studies of this treatment will look at the quality of patients’ extra life as well as the quantity.
“As physicians, we say that people ought to be willing to undergo anything if they get cured of their disease,” Daley said. “That’s why physicians have been so reluctant to think about quality of life. But not everybody feels that way. Some would say that their quality of life was so bad on therapy that they would have been better off without it.”
Besides the rigors of the treatment itself, doctors will look at how patients feel as they recover and whether they have trouble holding onto spouses, friends and jobs.
Vaughan at Nebraska said some people back out when they hear what the treatment involves. “If at least a few patients every year don’t say to me, ‘That’s not for me. Thank you very much. Let me out of here,’ then I’m not explaining it right,” he said.
But with all the risks, the decision for some is easy.
“I know that if I don’t have it, I’m really shortening my life,” said Barbara Howley, 33, who works for a book publisher in suburban Boston. Twelve years ago, she was treated for breast cancer, and now the tumor is back, spread this time through her body.
She hoped to be accepted into the Nebraska program. “This is my only chance to go into complete remission,” she said. “And that’s what I’m hoping for. I figure I have nothing to lose.”
Two years ago, Fanny Gaynes, 45, an advertising writer from Tiburon, Calif., underwent the treatment for spreading breast cancer. She had a relatively easy time of it and is “absolutely, totally, 100% glad” she went through it.
But now she has more cancer, this time in her other breast. Would she have bone marrow transplantation again? Knowing what she knows now, she’s not sure.
“When you’re having trouble sleeping because of the respirator in the room next to you, and you just know what’s going on all over the hall--you see it on the nurses’ faces--you have to really, really want it, and you have to believe in it.”
