Thursday, January 13, 2005
Cystic fibrosis is a genetic disease. Only a thousand American children per year are diagnosed as having it. Some ten million people in the United States carry the defective gene, but the disorder is recessive: a child will develop the condition only if both parents are carriers and both pass on a copy. The gene-which was discovered, in 1989, sitting out on the long arm of chromosome No. 7-produces a mutant protein that interferes with cells' ability to manage chloride. This is what makes sweat from people with CF so salty. (Salt is sodium chloride, after all.) The chloride defect thickens secretions throughout the body, turning them dry and gluey. In the ducts of the pancreas, the flow of digestive enzymes becomes blocked, making a child less and less able to absorb food. This was why Annie had all but stopped growing. The effects on the lungs, however, are what make the disease lethal. Thickened mucus slowly fills the small airways and hardens, shrinking lung capacity. Over time, the disease leaves a child with the equivalent of just one functioning lung. Then half a lung. Then none at all.
From my experience at Children's Hospital I am quite familiar with the reputation of Cincinnati. When I started in my current position six years ago my boss told me to go there to find out how to run a quality radiology department. So although the family described in the article was devastated by the news of their daughter's malady, they felt they were fortunate to be living so close to this fine facility. Mr. Gawande recalls their experience when they first went in for evaluation:
"We were there for hours, meeting with all the different members of the team," Honor recalled. "They took Annie's blood pressure, measured her oxygen saturation, did some other tests. Then they put us in a room, and the pediatrician sat down with us. He was very kind, but frank, too. He said, "Do you understand it's a genetic disease? That it's nothing you did, nothing you can catch?" He told us the median survival for patients was thirty years. In Annie's lifetime, he said, we could see that go to forty. For him, he was sharing a great accomplishment in CF care. And the news was better than our worst fears. But only forty! That's not what we wanted to hear."
The team members reviewed the treatments. The Pages were told that they would have to give Annie pancreatic-enzyme pills with the first bite of every meal. They would have to give her supplemental vitamins. They also had to add calories wherever they could-putting tablespoons of butter on everything, giving her ice cream whenever she wanted, and then putting chocolate sauce on it.
A respiratory therapist explained that they would need to do manual chest therapy at least twice a day, half-hour sessions in which they would strike-"percuss"-their daughter's torso with a cupped hand at each of fourteen specific locations on the front, back, and sides in order to loosen the thick secretions and help her to cough them up. They were given prescriptions for inhaled medicines. The doctor told them that Annie would need to come back once every three months for extended checkups. And then they went home to start their new life. They had been told almost everything they needed to know in order to give Annie her best chance to live as long as possible.
And then the author drops the bombshell:
The one thing that the clinicians failed to tell them, however, was that Cincinnati Children's was not, as the Pages supposed, among the country's best centers for children with cystic fibrosis. According to data from that year, it was, at best, an average program. This was no small matter. In 1997, patients at an average center were living to be just over thirty years old; patients at the top center typically lived to be forty-six. By some measures, Cincinnati was well below average. The best predictor of a CF patient's life expectancy is his or her lung function. At Cincinnati, lung function for patients under the age of twelve-children like Annie-was in the bottom twenty-five per cent of the country's CF patients. And the doctors there knew it.
The Page's do not learn of this fact until four years later.
In the winter of 2001, the Pages and twenty other families were invited by their doctors at Cincinnati Children?s to a meeting about the CF program there. Annie was seven years old now, a lively, brown-haired second grader. She was still not growing enough, and a simple cold could be hellish for her, but her lung function had been stable. The families gathered in a large conference room at the hospital. After a brief introduction, the doctors started flashing PowerPoint slides on a screen: here is how the top programs do on nutrition and respiratory performance, and here is how Cincinnati does. It was a kind of experiment in openness. The doctors were nervous. Some were opposed to having the meeting at all. But hospital leaders had insisted on going ahead. The reason was Don Berwick.
Berwick runs a small, nonprofit organization in Boston called the Institute for Healthcare Improvement. The institute provided multimillion-dollar grants to hospitals that were willing to try his ideas for improving medicine. Cincinnati's CF program won one of the grants. And among Berwick's key stipulations was that recipients had to open up their information to their patients-to "go naked," as one doctor put it.
The reaction of the family is interesting in that once they are presented with the facts they decide to stay with treatment at Cincinnati. As a parent I do not know what I would have done in their situation. But besides the obvious discussion the article has generated about how best to care for a child with a chronic lethal disease, the piece is generating calls for hospitals and physicians to be share data regarding their success rates caring for a wide variety of conditions. Insurance companies are already starting to base reimbursement on whether a facility follows accepted treatment plans. As far as the need for healthcare institutions to be able to provide quanitative data to patients regarding outcomes, it looks like the genie is out of the bottle.