Imagine the day, envisioned by health reformers, when research about which treatments work best at the most reasonable cost boils medical decision-making down to a science. Doctors tap relevant information into their computers—age, medical history, test results—and presto! they get the best course of action.
What's wrong with this picture? Granted, it's painted in overly simplified terms. But the people on the front lines do worry about what could be lost if treatment choices in a reformed health system rely too heavily on data about what works for the average person and ignore the individual patient: whether he's a construction worker or a music therapist, whether a risk-taker or risk-avoider, whether she's lived a long life or is just reaching her prime. Certainly, many medical decisions are no-brainers—you won't hear much argument over the removal of a near-bursting appendix. And evidence-based medicine may, indeed, cut down on the unnecessary tests and procedures doctors order to increase reimbursements or protect themselves from lawsuits. But physicians practicing cutting-edge medicine point out that their judgments and advice often draw as much on art as on science. Should a patient choose continuing seizures over surgery that risks the loss of her musical talents? How far should doctors go to treat a terminal disease in a young newlywed? Should a pregnant woman sacrifice one twin to improve the survival chances of the other?
Such discussions go on hourly at the University of Maryland Medical Center in Baltimore, a 705-bed hospital that handles more than 36,000 admissions a year. Doctors there, as at major hospitals around the country, are all-too-practiced in the agonizing treatment call. Cancer, in particular, leaves them guessing, seldom able to talk of a cure. "There are decision dilemmas involved in every treatment—it's not a multiple-choice test where there's always a correct answer," says Kevin Cullen, director of UMMC's Greenebaum Cancer Center. U.S. News spent several days this spring following doctors and patients at UMMC to see how treatment decisions are made when hard evidence is lacking.
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When 36-year-old Bart Simmons walked into the cancer center in April, he was praying there would be a correct answer for him—or, at the very least, that he would get a few extra years to spend with his new wife, Rhonda. His testicular cancer, initially treated with surgery and chemotherapy in 2001, had metastasized to the lymph nodes near his kidney. This was the second time his cancer had spread. Last year, he traveled to UMMC (thanks to arrangements made by a client affiliated with the hospital) to have a grapefruit-size tumor removed; no surgeons near his Vero Beach, Fla., home had dared attempt to remove the mass, which was entangled in two major blood vessels connected to the heart. For nearly a year since then, Simmons had been the picture of health; free of any signs of cancer, he worked full time at his construction job, attended church with his wife, and gathered regularly with friends at home Monday evenings.
Now, Simmons and his wife were hearing bad news from surgeon Nader Hanna: The two small lymph node metastases were endangering the only functioning kidney he had; the other had stopped working years ago, probably damaged during his first cancer surgery in Florida in 2001. Hanna presented the couple with their options: immediate surgery to remove the metastases, which would very likely result in the loss of his kidney and lifelong dialysis; or experimental chemotherapy, which could spare his kidney but might not be as effective as the surgery. "The treatment at this point is nonstandard," Hanna told Simmons and his wife. "The options are really complex. Do we treat or not? If we do treat, do we go for the technically challenging surgery? Or do we try chemotherapy, which may do nothing for him?"
Simmons, grateful for the honesty, decided to have the surgery after discussing chemotherapy with oncologist Edward Sausville. Both doctors were betting—though it was indeed a gamble—that surgery would give him the best shot at living longer. "Bart has a highly unusual testicular tumor" that transformed from a relatively benign teratoma to a more aggressive adenocarcinoma, says Sausville. "I've seen maybe three or four in my career."
[Photo gallery: Follow along on Bart's hospital journey.]
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Such rare conditions promise to complicate comparative-effectiveness policymaking, experts maintain, because they don't occur often enough to be rigorously studied to determine which, if any, treatments work. Even when the circumstances are not so rare, medicine's various possible responses may have pros and cons that offset one another, says Gregg Zoarski, an interventional radiologist at UMMC who faces such a conundrum when advising patients with small brain aneurysms. The coil procedure he performs to cut off an aneurysm's blood supply causes it to burst in a small percentage of cases, sometimes leading to a stroke or even death. Doing nothing, though, carries a 2 to 3 percent yearly risk of the aneurysm bursting on its own. He can't just turn to studies to tell him how to proceed, he says, because researchers themselves aren't in agreement.
When research can point to a treatment's clear benefits, certain trade-offs may nonetheless be unacceptable to patients. Whenever surgeons delve into the brain, for example, they chance altering the person's unique talents. Where is creativity located within those intricate folds of tissue? Or tone recognition, or pitch? Those are the questions Jennifer Friend, 28, a music therapist with epilepsy, posed after her UMMC neurologist told her that removing brain tissue in her temporal lobe could halt her twice-monthly seizures. A wealth of data shows that the procedure completely alleviates seizures in about two thirds of patients, improving the overall quality and even the length of their lives.
But while neurological testing can adeptly pinpoint regions in the temporal lobe vital for hearing, language, and memory, not so with musical abilities, says neurologist Jennifer Hopp. And a handful of studies suggest that temporal lobe resection surgery may lead to a decline in abilities to perceive meter and tempo and to recognize emotions evoked in music. Though Friend would finally be able to get her driver's license if she had the surgery, she declined. She says the passion she feels about her job—composing guitar tunes to help psychiatric patients express their moods—means more.
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Part of the challenge (and thrill) of practicing medicine comes from handling the unexpected under pressure. While Hanna had promised to try to save Simmons's kidney, he realized upon peering into the open abdominal cavity that one of the masses was much larger and more extensive than it had appeared on the CT scan less than four weeks earlier, which often happens with fast-growing tumors. What's worse, it had completely wrapped itself around the ureter, the duct that connects the kidney to the bladder. Hanna called two urologists and urologic surgeon Michael Phelan into the OR for an on-the-spot consultation. The two surgeons took a break to go tell Rhonda there was a 90 percent chance the kidney would be lost.
Back in surgery, based on evidence that the tumor had spread beyond the lymph nodes into the surrounding abdominal lining, Hanna decided to order a relatively new on-the-table procedure in which a high dose of heated chemotherapy drugs is poured directly into the abdominal cavity. Although Hanna had never used the method for testicular cancer, he'd used it with some success against similar tumors in the appendix, colon, ovary, and stomach that had spread to the abdominal lining.
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To map out the regimen for newly diagnosed cancer patients, the oncology team in each specialty at UMMC meets once a week to pore over medical histories, imaging scans, and biopsy slides and weigh the risks and benefits of various treatments. At a May breast cancer meeting, 17 experts, including surgeons, nurses, pathologists, and medical oncologists, gathered to discuss whether it was wise to initiate chemotherapy to shrink a large breast tumor in a woman who had missed so many appointments in the past that her tumor was now inoperable, or whether they could give the woman some pills she could take at home. Reviewing another patient, they pondered: Was it smarter to first treat her aggressive cervical cancer with radiation or to remove her small breast tumor? "I think at this point her cervical cancer trumps her breast cancer," argued Anil Dhople, a radiation oncologist. "She has a low-grade breast cancer and a bleeding cervical cancer. I think the cervical cancer needs to be addressed first." The breast surgeons responded that an immediate mastectomy would delay the cervical cancer treatments only by a couple of weeks at most. The matter was settled by Katherine Rak Tkaczuk, director of the breast evaluation and treatment program. "We can give her hormonal therapy pills to potentially shrink her breast tumor during the seven weeks she's getting her radiation therapy and chemotherapy for the cervical cancer," she advised.
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About 45 minutes after speaking with Rhonda, Hanna realized the kidney had to go; the tumor was attached to the kidney wall. Even so, it wasn't an easy call. "If Bart's cancer comes back anyway in three months, his quality of life might be impaired more from taking the kidney out," Hanna admitted later. "But I think this will give him the best chance he has at survival."
Before starting chemo, Hanna called a colleague and asked him to research what dose of the drug, which normally is cleared by the kidneys and thus could be toxic for Simmons, could be safely administered to a patient in renal failure. Hanna calculated that he should lower the dose by one quarter and administer the drugs for one hour instead of 90 minutes—and monitor Simmons carefully in the days following surgery.
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While unclear and painful decisions occur in all areas of medicine, the choice to end a new life is among the most agonizing. These discussions commonly occur when fetuses are diagnosed with genetic problems or severe organ abnormalities, but they also happen in about 30 percent of identical-twin pregnancies, when a blood-flow problem endangers the placenta-sharing twins. All too often, couples must decide whether to terminate one twin to improve the survival odds of the other. "Sometimes one baby has so little flow that it can't make it," explains Ahmet Baschat, a fetal medicine specialist at UMMC. "If that baby dies, the surviving baby can be hurt as well, since they share the same connections for oxygen and nutrients." In many pregnancies that he monitors, the blood flow improves, and the babies are fine. In some, though, things quickly change, and one or both babies are born severely prematurely or die in the womb.
Vera Mednikova, 32, of Vienna, Va., consulted Baschat last fall after being told she might need to terminate her pregnancy (her first) or abort the twin that was abnormally small. She and her husband decided to keep both babies after Baschat said that they could be monitored via weekly ultrasounds; termination of the smaller fetus would be an option up until the 21st week. After that, "we knew if something happened we could lose both babies," Mednikova recalls. The couple's twin girls were born six weeks early in May, and both are doing fine.
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A month after being released from the hospital, Simmons, back at his home in Florida, was upbeat but introspective, hopeful about returning to work part time in a few weeks and doubtful that he'd try any more cancer treatments. "This is the third time I've had to rebuild my life," he says. "There's only so many times you can punch me before I say that's it, I'm not going in the ring again."
The experience of Thomas Scalea, head of UMMC's shock trauma center, illustrates why doctors hate to be the ones to call anyone down for the count. In his work with young people whose severe head injuries have left them comatose, though not brain dead, he knows that even the best imaging scan can't tell him if they'll wake up or how much of their old selves will remain. "A few years ago, I saw a man in his 30s who had nearly drowned after being struck in the head," he recalls. Through heroic efforts, he'd managed to save the man, who had suffered "the worst brain injury I'd ever seen in a living person." After weeks of heart-wrenching conversations with the family, Scalea advised them to withdraw treatment, since he'd seen so many loved ones bankrupt themselves with years of futile efforts. The family decided to hold off. Six months later, the man, fully recovered, walked into Scalea's office and thanked him for saving his life.