Antiinflammatory Therapies for Cystic Fibrosis: Past, Present, and Future

William A. Prescott, Jr., Pharm.D.; Cary E. Johnson, Pharm.D.


Pharmacotherapy. 2005;25(4):555-573. 

In This Article

Abstract and Introduction

Inflammation is a major component of the vicious cycle characterizing cystic fibrosis pulmonary disease. If untreated, this inflammatory process irreversibly damages the airways, leading to bronchiectasis and ultimately respiratory failure. Antiinflammatory drugs for cystic fibrosis lung disease appear to have beneficial effects on disease parameters. These agents include oral corticosteroids and ibuprofen, as well as azithromycin, which, in addition to its antimicrobial effects, also possesses antiinflammatory properties. Inhaled corticosteroids, colchicine, methotrexate, montelukast, pentoxifylline, nutritional supplements, and protease replacement have not had a significant impact on the disease. Therapy with oral corticosteroids, ibuprofen, and fish oil is limited by adverse effects. Azithromycin appears to be safe and effective, and is thus the most promising antiinflammatory therapy available for patients with cystic fibrosis. Pharmacologic therapy with antiinflammatory agents should be started early in the disease course, before extensive irreversible lung damage has occurred.

Cystic fibrosis is the most common lethal, genetically inherited disorder among Caucasians.[4,6,8,11] The clinical features of cystic fibrosis occur as a result of mutations in the cystic fibrosis trans-membrane conductance regulator gene.[4,6,8,11] Consequently, the cystic fibrosis airway is exposed to a vicious cycle of obstruction, infection, and inflammation, all intimately linked.[3] Airway inflammation begins early in the disease course, with notable neutrophil infiltration present in infants as young as 4 weeks.[4–11] The inflammatory process persists throughout the patient's life and, if left unchecked, irreversibly damages the airways, leading to bronchiectasis and progressive decline in lung function.[10] Pulmonary disease accounts for most of the morbidity and mortality in patients with cystic fibrosis. Therefore, interrupting this cycle with antiinflammatory agents may slow disease progression, improve quality of life, and delay respiratory failure.[12] However, less than 50% of pulmonary practitioners prescribe specific antiinflammatory agents, and only 25% of patients with cystic fibrosis receive such therapy.[13]

The safety and efficacy of numerous anti-inflammatory therapies have been studied for treatment of cystic fibrosis. Several agents have proved beneficial, particularly in younger patients with mild disease. However, safety concerns have limited the utility of certain drugs.


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