Pharmacologic management of cystic fibrosis

Standard pharmacologic management of cystic fibrosis is discussed and the role of new agents in the treatment of this disease is explored. Cystic fibrosis is a recessive, fatal genetic disease involving multiple organ systems, in which patients develop pancreatic insufficiency, malabsorption, and repeated pulmonary infections. Pharmacotherapy to date has included broad-spectrum antimicrobials and aggressive nutritional management with microencapsulated pancreatic enzymes. Acute pulmonary exacerbations, caused by Pseudomonas aeruginosa, require combination i.v. antimicrobial therapy for 14 to 21 days. With the recent discovery of the genetic defect responsible for cystic fibrosis, as well as the cellular mechanism, new pharmacologic approaches are being explored to improve treatment. Aerosolized amiloride is being tested to modify the basic defect in the chloride channel. Dornase, a new mucolytic, is used to decrease sputum viscosity and increase mucociliary clearance. Leukoprotease inhibitors are currently being evaluated for decreasing the acute inflammatory reaction in the lung. Gene therapy has been promising, but its role in the management of cystic fibrosis is many years away. Drug therapy for cystic fibrosis has been primarily directed at treating infections with antibiotics and supplementing digestive enzymes and vitamins. New agents and gene therapy may substantially change the morbidity and mortality of this disease.