Clinical Care of the CF Patient

Richard Moss, M.D. Professor of Pediatrics and Director of the Cystic Fibrosis Center at Lucile Salter Packard Children's Hospital at Stanford

Fall 1997

We are at a transitional time in CF care, Dr. Richard Moss explained in an overview of clinical care for the CF patient. For decades, CF care has been "empirical," that is, doctors have observed what treatments help patients and have devised regimens based on these observations. This approach has extended the lives of CF patients: in 1960, the median survival age was 10; in 1990, it was 29; and by 1995, it had reached 30.

In 1989, the CF gene was cloned. This meant basic understanding of cystic fibrosis was greatly extended and scientists hoped they could devise "rational" care that would interrupt the molecular and cellular chain of events leading to disease. This research also led to the discovery of over 600 different CF mutations. These are being studied and cataloged, showing a wide variety of symptoms and severity in the disease. For example, ten percent of adults with CF have normal lung function, male infertility with no other symptoms may signal a case of undiagnosed CF, some CF patients have liver disease while others do not, and a small percentage are pancreatic sufficient (they do not need enzymes). This information has been fundamental in our understanding of the basic defect, however, it has not led to proven increases in longevity or quality of life. And it may take longer than had previously been thought for this route to provide a cure. Until it does, doctors will continue to employ and improve upon empirical care.

Our clinical care needs to consider the whole person. Nutrition is one key; lung clearance is also vital. No one treatment is right for everyone. Often patients pick from a smorgasbord of airway clearance treatments ranging from CPT to the flutter to autogenic drainage. Pseudomonas aeruginosa appears in 80 percent of people with CF who are teenaged or older. Studies are underway to find the best way to deal with this. In Denmark, people with CF are living to be 50 years old. They use anti-pseudomonas drugs early and aggressively, eliminating it when it first appears in the lungs. In the United States, studies show that any setback in CF health--sudden loss of weight, sudden exacerbation of a lung infection--is, on the average, not as aggressively treated. Patients and parents of patients must be hyper vigilant in monitoring their health situations, well-informed of their alternatives, persistent once a course of action has been decided upon and aggressive in their treatment. For now, empirical care is all we have. But after observing the Danish model, treatment can be an extremely powerful tool.

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