Standard care for outpatients with CF is intensive, with regimens that include exogenous pancreatic enzymes taken with meals, nutritional supplementation, anti-inflammatory medication, bronchodilators, and chronic or periodic administration of oral or aerosolized antibiotics (e.g., as maintenance therapy for patients with P. aeruginosa). Recombinant DNAse aerosols (degraded DNA strands that contribute to mucus viscosity) and nebulized hypertonic saline (serves to augment PCL depth, activate mucociliary clearance, and mobilize inspissated airway secretions) are administered routinely. Chest physiotherapy several times each day is a standard means to promote clearance of airway mucus. Among older individuals with CF, malabsorption, chronic inflammation, and endocrine abnormalities can lead to poor bone mineralization, requiring treatment with vitamin D, calcium, and other measures. The time, complexity, and expense of home care are considerable and take a significant toll on patients and their families.
Severe respiratory exacerbation is commonly managed by hospital admission for frequent chest physiotherapy and parenteral antibiotics directed against serious (and often multiply resistant) bacterial pathogens. Aggressive intervention in this setting can restore a large component of lung function, but ongoing and cumulative loss of pulmonary reserve reflects the natural history of the disease. Poor prognostic indicators such as sputum culture containing B. cepacia, mucoid P. aeruginosa, or atypical mycobacteria are rigorously monitored in the CF patient population. An increasing incidence of methicillin-resistant S. aureus has also been observed, although the clinical significance of this finding has not been fully elucidated. Typical inpatient antibiotic coverage includes combination drug therapy with an aminoglycoside and β-lactam for up to 14 days. Maximal improvement in lung function is often achieved by 8–10 days in this setting. Many families elect parenteral antibiotic treatment at home, and additional studies are needed to evaluate specific drug combinations, duration of therapy, and home versus inpatient management. Other CF respiratory sequelae that may require hospitalization include hemoptysis and pneumothorax. Hypersensitivity to Aspergillus (allergic bronchopulmonary aspergillosis) occurs in approximately 5% of individuals with the disease and should be suspected in the absence of a response to conventional treatment.
Lung transplantation remains a viable therapeutic option in the setting of end-stage CF pulmonary failure, with 5-year postoperative survival rates on the order of 50–60%. Determining the optimal timing for surgery presents a substantial challenge, particularly because overall prognosis for individuals with severe lung disease is sometimes difficult to predict, and mortality associated with transplantation is significant (1-year survival rates of approximately 80%). Forced expiratory volume in 1 s (FEV1) measurements less than 30% predicted, together with an assortment of other clinical features, are often used as thresholds for entry onto transplantation lists, although waiting periods for healthy donor lungs can be quite protracted. Based on clinical outcome and limited access to healthy donor lungs, many CF patients and their families do not pursue this option.