"How it is," for someone with cystic fibrosis, is that each breath is a small triumph. The genetic conditio churns out gluey mucus that will clog and inexorably shut down the lungs if not jarred loose and hocked up at least twice a day. That entails half-hour sessions of gentle thumps (parents of CFbabies call them "pitty pats") at more than a dozen prescribed locations on the back and chest, or wearing a vibrating vest powered by an air hose, or using a handheld "positive expiratory pressure" device. The sessions are punctuated with vigorous "huff coughs" to expel the sticky stuff. CF children learn the technique as toddlers.
There's more. The mucus blocks the ducts in the pancreas that release vital digestive enzymes generated by the organ. To prevent malnutrition, downing handfuls of enzyme capsules is mandatory before eating so much as a cracker or drinking a glass of milk. Many children still can't absorb the amount of nutrients they need, and must have a high-calorie supplement dripped into their stomach at night through a port in their belly or a tube down their nose. About half of those with the condition, including Lang, develop CF-related diabetes because of gummed-up ducts in the pancreas and have to take insulin injections. A who's who of deadly microorganisms love to multiply in the built-up mucus and cause lung infections. For patients who get repeat lung infections—like Lang, who spends a couple of weeks in the hospital for every six outside it—30-minute daily sessions at home, inhaling an antibiotic and tolerating the unpleasant aftertaste, are encouraged.
Even after 12 years of caring for CF children, Jamie Wooldridge, a pulmonologist in the Cystic Fibrosis Center at Cincinnati Children's, purses her lips and shakes her head in disbelief when she talks about the grueling regimen. "I can't think of another chronic illness that asks so much of families," she says. "Three to four hours. Every day."
The daily grind can never stop because it does not defeat the disease; it only blunts its force so that life can go on for CF children—or most of them. Every year scores die, falling short of adulthood however diligently their families work to keep them alive. And while several drugs now in clinical trials hold out the promise of a cure, their approval could be several years off.
Yet the larger picture also shows encouraging progress. The grip of the disease, which affects an estimated 30,000 U.S. children and adults, has loosened: In the early 1960s, children typically died before they were 10 years old. New drugs and improved care have pushed the median age above 37. The children are healthier, too. They play soccer. They join tumbling teams. They surf.
The pace of progress, moreover, is picking up. Over the past decade, CF specialists have increasingly embraced evidence-based medicine, "best practices," and other health reform tools. Several years ago, the Cystic Fibrosis Foundation, which for more than 50 years has funded basic research and development of drugs like those currently being tested, began bringing together teams from some of its 115 accredited centers, forming collaboratives to exchange information and encourage the spread of successful approaches. In 2006, the foundation took the almost unheard-of step of going public with outcomes data. Anyone can now look up average lung function and body mass index (BMI), the two key CF indicators, for every center's patients at the foundation's website.
When the numbers were released in 2006, the differences from center to center were immediately apparent. Cincinnati Children's, however, had known five years earlier that it had to improve. In 2001, the hospital persuaded the foundation to hand over performance data from a few top-performing centers, expecting that its own numbers would compare favorably. Instead, the figures described a troubling portrait of care that was mediocre or worse. Cincinnati Children's launched an unflinchingly critical self-examination. Top administrators and clinicians invited the parents of the hospital's CF children to a meeting. They displayed the stark data in PowerPoint slides, pledged to do much better, and asked the families to help. "We laid our dirty laundry on the table," says James Acton, director of the cystic fibrosis center at Cincinnati Children's.
Less than 10 years later—a blink in the life of a large institution—the hospital is now among the small handful of elite CFcenters. "They became some of the deepest students of the study of perfection," says Don Berwick, who as CEO of the Institute for Healthcare Improvement in Cambridge, Mass., which promotes practices that enhance safety and quality, frequently advised Cincinnati Children's as the hospital reinvented itself. (This month Berwick took over as newly appointed head of the federal Centers for Medicare and Medicaid Services.)
Cincinnati Children's is not alone in recognizing inadequate performance and vowing to do better; it was one of many hospitals that sought a "persuing perfection" grant from the Robert Wood Johnson Foundation to support its turnaround effort, for example. But it is telling that years before health reform became law in March, the hospital built its road to improvement using many of the same tools Congress wrote into the final package:
Evidence-based medicine. Detractors of EBM worry that it means studies and data and not a physician's accumulated clinical wisdom should dictate care. It doesn't, or shouldn't. The idea, rather, is that well-done research should help guidetreatment. For example, Pseudomonas aeruginosa is a nasty bacterium that has a particular affinity for the mucus in the lungs of CF patients and is responsible for a disproportionate share of their hospitalizations and deaths. Many children with cystic fibrosis show no symptoms of infection but repeatedly test positive for the bug. Given its lethal nature, it makes sense to damp it down to the lowest possible level, and many studies have shown that routinely inhaling the antibiotic Tobramycin keeps the bug at bay.
When Cincinnati Children's began to dissect its own performance and plot out how it could improve, the CF care team and quality analysts saw that the evidence for using Tobi (no one uses the drug's full name) with chronically infected kids was powerful and that the hospital wasn't even in the top quarter of centers in keeping patients on it. It's not an easy drug to use, eating up an hour or more a day with no obvious benefit because of the absence of symptoms. The hospital's doctors intensified their work to educate families on the benefits of Tobi even for children who did not appear ill. Parents were enlisted as partners, reaching out to other parents. (One of them, Tracey Blackwelder, was the mother of four CF children, all under the hospital's care. She is now on staff to help the hospital find similarly motivated parents to join "improvement teams.")
Progress in Tobi use was charted. Now prescriptions are written for about 80 percent of eligible children, says Acton. That compares with fewer than half of patients at about 1 in 7 centers nationally.
Nor had the hospital previously been especially diligent in making sure children with low BMIs received extra calories by mouth or through a tube or port. In 2004, 61 percent of its low-BMI children, almost exactly the national average, got supplemental feeding. The national average now stands at 71 percent. The Cincinnati Children's rate is 91 percent.
Collecting and sharing data. Berwick likes to say that if you want to do better, you first have to know how you're doing. You start tracking the percentage of CF children year by year who get a flu shot, for example, and their average lung function. Then, he says, you make the data available to the public and to other care providers so the top and bottom of the range of performance are identified. That allows you to set realistic benchmarks and goals in a way for all to see.
Gerald O'Connor, a professor of medicine at Dartmouth Medical School and chair of the Cystic Fibrosis Foundation committee that drew up the 2006 plan to display centers' clinical performance, says that opening up the data was "a game-changer," because the centers could observe for themselves that the ranges were too large to explain other than by differences in each center's approach to care. "What people were doing was wildly variable," he says. Lung function figures, for example, showed that children at a few aggressive centers were breathing, on average, as well as children without the disease. These centers hounded families to schedule outpatient visits and reminded them when one was approaching.Lung infections were reduced by broad use of proven medications and successful fluimmunization campaigns.
When the staff at Cincinnati Children's realized in 2001 that average lung function for its CF kids from 6 to 17 was less than 80 percent of normal, trailing the national average of about 84 percent and the top center's 93 percent, they increased the frequency of lung infection checks to every three months instead of once a year. Parents were taught new and better ways to clear their child's airway. Reminders to get flu shots went out. Today, national average lung function has improved to about 93 percent. At Cincinnati Children's it has rocketed to a better-than-normal 102.5 percent.
Learning from other centers. CF center director James Acton examines 8-year-old outpatient Raegan Holley and asks her mother, Dawn, 15 questions displayed on the nearby monitor. Raegan will receive a "pulmonary exacerbation score," or PES, of 0 to 38 according to the number and severity of her currentrespiratory symptoms (such as increased cough for a week or more) and objective measurements (such as blood oxygen level). A perfect PES is 0. Five or higher suggests that she needs a change in her lung-clearing technique or a more effective antibiotic.
The PES score sheet is Acton's favorite example of how the hospital worked with another center to benefit patients. The sheet, developed at Akron Children's Hospital, caught Acton's eye when the director of its CF center presented it at the annual meeting of the North American Cystic Fibrosis Conference conference. When Acton returned, he shared it with the hospital's CF team. The Akron center then helped its Cincinnati counterpart document the extent to which evaluations of outpatients varied from physician to physician, resulting in more- or less-aggressive care depending on each doctor's clinical preferences. "They didn't believe it at first when I showed them the results," says Acton. By standardizing assessments, he argued, care would be improved. And that, he says, is what happened.
Defining ambitious goals. Although Cincinnati Children's now outpaces most other CF centers in the nation, its clinicians and quality specialists are not satisfied. "There's incremental improvement every year," says Bruce Marshall, the Cystic Fibrosis Foundation's vice president for clinical affairs. The hospital's 2011 goals include pushing lung function up a point—to 106.3 percent for kids from ages 6 to 12 and to a lesser but still ambitious 100 percent for those 13 to 17. (As children reach adolescence, many become less faithful to the critical daily routines.)
Nutritional status, CF's other key indicator, now stands at 60 percent for the hospital's CF patients, meaning that 60 percent of the children are at or above the 50th percentile, or average, for BMI. The current national average is slightly above 48 percent; Cincinnati Children's is aiming for 62 percent in 2011.
Improved performance for children with an elevated PES aims even higher. At the moment, a tally of 5 or higher on a clinic visit produces a documented change in the child's care plan about 40 percent of the time. The 2011 target is to double that to 80 percent.
Collaborating with families. The healthcare reform law calls for providers of medical care to involve patients, parents, and other caregivers in "shared decision making," with each a full partner in charting a course of action. That families should actively take part, with their views on appropriate care considered as important as those of the doctors, remains a foreign notion at most hospitals. Not at this one.
Annelise Page was labeled an urgent nutritional risk when she was 7 years old; her BMI was in the lowest 10th percentile. The family and the center's physicians and dietitians had "many difficult discussions" about the best way to help her, says Acton. They "were very clear about balancing quality of life with the need for sometimes intrusive interventions" such as a feeding-tube port into her stomach, which Annelise rejected. ("That won't look good with a bikini.")
Meanwhile she remained at risk. "She tried to consume three to four thousand calories a day but she just couldn't do it," says her mother, Honor Page. "She got so sick of butter and ice cream. She would plead with me for a salad. People really have no idea how hard it is."
About two years ago her doctor, Jamie Wooldridge, brought up the possibility of a nasogastric feeding tube, which would have to be snaked through the girl's nose every night to drip predigested nutrients directly into her stomach. Annelise agreed to give it a try. She mastered the technique, which soon became just another part of her nightly routine. And she gained 30 pounds, topping the 50th percentile. Now 16 and on her school tumbling team, Annelise tracks her BMI on a chart on her bedroom wall. "They listened to her," says her mother. "They went into their bag of tricks and thought, 'What can we offer this family?' "
Adopting electronic records. Health information technology has benefited Cincinnati Children's patients most directly in the form of My Care Connection, a secure Web page that lets families track a child's progress and stay on top of the latest medical plan. There are word descriptions and graphs of the child's most recent lung function data, results of lab and imaging tests, and an updated list of medications and dosages. Parents can put questions to a child's care team and expect answers within a day. "It is a critical philosophical step forward," says Acton, "in transferring control of their healthcare from us to them."