Cystic Fibrosis in Adults

Univ. of North Carolina, Chapel Hill. Comprehensive text for the diagnosis and care of adults with cystic fibrosis. Text is organized by the organ systems most affected by CF: molecular and cellular bases of disease pathogenesis, pulmonary disease, gastrointestinal system, and other organ systems. Extensively referenced. DNLM: Cystic Fibrosis--in adulthood.

Forty years ago, prospects were bleak for a patient with cystic fibrosis, which was then an invariably fatal disease of early childhood. Patients seldom survived beyond infancy; they succumbed to malnutrition and pulmonary complications. Nonetheless, a few centers initiated an aggressive, comprehensive approach to the care of patients with cystic fibrosis that introduced symptomatic treatments designed to slow the progression of the disease. Since that time, their clinical condition has improved. Although some patients still die early in life, many others live well into adulthood. Better diagnostic techniques and refinements of therapeutic strategies have combined to increase the median age of survival beyond 30 years. More than a third of all patients in the United States who are registered with the Cystic Fibrosis Foundation are older than 18 years of age. Cystic fibrosis has "grown up" and become a disease treated by internists as well as pediatricians.

Cystic Fibrosis in Adults discusses the advances and reviews extensively the clinical issues that are pertinent to patients with this disease. The first several chapters serve as an introduction and review the pathogenic mechanisms that may be potential targets of therapy. Along with improvements in the clinical care of patients with cystic fibrosis, our understanding of the molecular and cellular biology of the disease has expanded rapidly since the identification of the gene for cystic fibrosis 10 years ago. The physiologic and genetic information about the basic defect has provided valuable insight into the structure and function of the cystic fibrosis transmembrane conductance regulator and how mutations in the gene for this protein may contribute to the pathophysiology of the disease. This knowledge has led to a shift in the therapeutic approach to cystic fibrosis. Whereas previous therapies were directed at the downstream effects of the disease, new therapies are being designed to correct or bypass the basic defect.

Because more than 90 percent of patients with cystic fibrosis die from associated lung disease, much of the book is devoted to the pulmonary manifestations of cystic fibrosis. Several chapters discuss the current management of sinopulmonary disease in cystic fibrosis, which is directed primarily at the consequences of infection and inflammation, as well as the role of lung transplantation in end-stage disease. New approaches to treatment of pulmonary disease are also discussed, with a focus on the status of gene therapy for cystic fibrosis. Although this information may be useful to researchers, many of the details included in the discussion are probably irrelevant to clinicians. Less space is devoted to some of the newer, exciting pharmacologic therapies that may correct or circumvent the defective cystic fibrosis transmembrane conductance regulator. Many of these new drugs are now being used in patients, and I suspect that they would be of great interest to clinicians who care for adults with this disease. Indeed, these therapeutic strategies may deserve a chapter of their own.

The remainder of Cystic Fibrosis in Adults covers many of the extrapulmonary manifestations, with particular emphasis on the pancreatic and gastrointestinal complications of the disease. It also includes chapters on clinical topics that are neglected in other textbooks, such as reproductive issues in patients with cystic fibrosis.

Cystic Fibrosis in Adults clearly shows the symbiosis between research and clinical care with regard to the disease. The book is well organized, and the individual topics are covered thoroughly. Inevitably, as in any multiauthored book, there is some duplication of the information presented. Nevertheless, the editors have produced a useful reference book, and this comprehensive textbook integrates information on the care of patients with new insight into the pathogenesis of cystic fibrosis. As our understanding of the molecular and cellular basis of the disease advances, new therapeutic strategies will emerge that ideally will increase the life expectancy of patients even further. With luck, future editions will need to include a section on the geriatric care of patients with cystic fibrosis.

Reviewed by Thomas Ferkol, M.D.
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