Management of the Child With Cystic Fibrosis

Abstract

Cystic fibrosis is one of the most common lethal inherited diseases among the Caucasian population, with an incidence of 1 in 2,000. With the progress made in the management of the disease, a once-regarded childhood illness has now an improved survival rate of up to 30 years. It is a multifaceted disease affecting a number of organ systems primarily pulmonary and gastrointestinal tract, with the former leading to most of the mortality. Therefore, good pulmonary toilet, including daily chest physiotherapy and appropriate antibiotic treatment for acute pulmonary exacerbations, remains the cornerstone of therapy. Disease-specific pharmacokinetics seen in these patients require special dosing considerations specifically for antibiotics to ensure adequate serum concentrations. In addition, bronchodilators, steroids, and mucolytics also play a role. With respect to the gastrointestinal tract, pancreatic insufficiency occurs and requires enzyme replacement. Intestinal obstruction may occur as early as the neonatal period, as "meconium ileus," and recur throughout the patient's lifespan. More recent modalities including chloride-channel facilitators, antiproteases, and gene therapy may hold promise to further improve the survival and quality of life in these individuals. The pharmacists' role is vital, especially with the unique pharmacokinetic considerations specific to this population and the complexity of medications necessary for appropriate management of the disease. Copyright © 1996 by W.B. Saunders Company