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Bad times for breast cancer weapon

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Two years after studies showed no survival benefit for women who underwent bone marrow transplants for advanced breast cancer, the arduous treatment has become so unpopular that researchers cannot find enough patients for studies aimed at improving it and showing a benefit.

What’s more, some health insurers are refusing coverage for the few breast cancer patients who do want to participate in studies of new bone marrow transplant procedures. The treatment typically costs $60,000 to $100,000.

One woman who decided to participate in a transplant study is Pamela McEwan, 47, of Blackwood, Camden County. After discovering in July that her breast cancer had spread to her lungs and liver, she felt that a transplant technique being tested at the University of Pennsylvania was her best hope. But in early December, shortly before she was scheduled for treatment, Independence Blue Cross denied coverage.

The company granted an exception this month after appeals by McEwan’s physician, lawyer and others. Still, the bureaucratic fight took a toll.

“Their denial was so crushing and unexpected,” McEwan, a Philadelphia high school biology teacher, said last week before entering the hospital for the three-week-long transplant procedure. “I fell into a deep depression. They say half the battle is your attitude, so I’m fighting hard to get my optimism back.”

The rise and fall of transplants reflects the desperation of patients with late-stage breast cancer, confronting the limits of conventional surgery, radiation and chemotherapy.

In the 1990s, such women and their physicians became convinced, despite a lack of scientific proof, that transplants offered longer survival than standard chemotherapy. Health insurers were forced by lawsuits and legislation to pay for the treatment before it was proven.

The procedure involves removing healthy bone marrow (or marrow stem cells), then giving the patient near-lethal doses of chemotherapy. Because the drugs kill both cancer cells and healthy cells, the patient’s bone marrow is put back to rebuild the immune system. About 2 percent to 5 percent of patients die from complications of the treatment itself.

Despite the risks, most transplant patients during the 1990s refused to participate in transplant studies for fear of being randomly assigned to get conventional treatment. The shortage of patients dragged out the research.

In 1999, results of four major studies finally showed that transplants, while as effective as conventional chemotherapy, did not improve patients’ grim prognosis. One of the studies, led by Edward Stadtmauer of the University of Pennsylvania Cancer Center, found that patients with metastatic cancer fared the same regardless of treatment: After three years, more than 60 percent had died.

Transplant research took another public relations hit a year ago with the disclosure of fraud by a South African study - the only trial that had showed a survival advantage for transplant patients.

Experts urged women and doctors not to lose faith because refinements of the procedure might yet show a survival advantage. Researchers were testing promising new drug regimens, monoclonal antibodies, and techniques for killing undetectable cancer cells in the bone marrow before replacing it in the patient.

“The negative results . . . should not suggest any lack of enthusiasm for . . . evaluation of genuinely different methods,” Marc E. Lippman wrote a year ago in a New England Journal of Medicine editorial.

Despite this, three ongoing trials approved by the National Cancer Institute (NCI), as well as several studies abroad, are seeking patients. This includes Stadtmauer’s latest trial at Penn, in which McEwan is enrolled.

NCI director Richard Klausner said last May in a letter to the country’s oncologists that the largest NCI-sponsored study was in “dire need” of patients, and enrollment was far behind schedule. Results from the trial, originally expected next year, might not be available “for many years,” Klausner warned.

The NCI, medical societies, breast cancer organizations, and bone marrow transplant groups all have urged health insurers to keep paying for patients in transplant studies.

Aetna/US Healthcare, the nation’s biggest health insurer, adopted that policy last February. A few companies, notably UnitedHealth Care in Minneapolis, still offer blanket coverage for the procedure.

But the Blue Cross Blue Shield Association, which licenses the nation’s 47 independent plans bearing that trademark, reevaluated the procedure in February 1999 and recommended against any coverage.

Apparently, the recommendations were heeded. Capital Blue Cross in Harrisburg, which serves central Pennsylvania and the Lehigh Valley, stopped paying for transplants in late 1999, spokesman Joseph Butera said.

So did Independence Blue Cross, said spokesman Dick Doran. Neither patients nor doctors were notified of the policy change.

McEwan, the first patient denied under the Independence Blue Cross change, was as shocked to be turned down as she was to be told that her cancer had returned.

The disease was first diagnosed at such an early stage two years ago that her long-term prognosis was excellent. Despite a strong family history of breast cancer, she hoped a mastectomy, radiation and chemotherapy would wipe out the cancer.

But less than a year after that treatment, a routine chest X-ray showed a tumor in her lungs.

She decided to enter a transplant trial in hope of not only gaining time, but gaining better time. With standard chemotherapy, her life would revolve around long courses of often-debilitating treatment.

“The average remission with a transplant is three years,” she said. “Well, three years of living in my home and working and feeling healthy is better than having to stop working, sell my home, and move in with someone who can help take care of me.”

What women like her really need, she said, are therapies that are more targeted and innovative than transplants. “But if insurance companies just stop supporting trials, there will be no research,” she said.

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April 2014
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