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Cystic Fibrosis

Guidelines

Flume PA, Mogayzel PJ Jr, Robinson KA, et al. Cystic fibrosis pulmonary guidelines: treatment of pulmonary exacerbations. Am J Respir Crit Care Med 2009; 180:802-8. This document offers graded recommendations for management of exacerbations, but is most noteworthy for highlighting the lack of evidence guiding many fundamental aspects of care.
PMID:  19729669
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Flume PA, O’Sullivan BP, Robinson KA, et al. Cystic fibrosis pulmonary guidelines: chronic medications for maintenance of lung health. Am J Respir Crit Care Med 2007; 176:957-969. This document offers graded recommendations for medication use according to patient age and severity of lung disease. It also nicely summarizes areas of clinical uncertainty.
PMID:  17761616
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Therapeutics

Fuchs HJ, Borowitz DS, Christiansen DH, et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis: the Pulmozyme Study Group.  N Engl J Med 1994;331:637-42. Large RCT found patients receiving a 24-week course of Pulmozyme had an improvement in FEV1 of 5% compared to placebo and decreased exacerbation rate (28 vs. 37% in placebo group).
PMID:  7503821
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Ramsey BW, Pepe MS, Quan JM, et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. N Engl J Med 1999;340:23-9. Study found use of TOBI on alternating months improved lung function, decreased bacterial burden, and decreased the relative risk of hospitalization. The rate of acquired tobramycin resistance was about 7% over 24 weeks.
PMID:  9878641
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Saiman L, Marshall BC, Mayer-Hamblett N, et al. Azithromycin in patients with cystic fibrosis chronically infected with pseudomonas aeruginosa.  JAMA 2003;290:1749-56.  Large multicenter RCT of 6 months duration found chronic azithromycin resulted in a 4.4% improvement in FEV1% predicted compared to a 1.8% decline in placebo.  The azithromycin group had fewer exacerbations and gained more weight.
PMID:  14519709
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Balfour-Lynn IM, Lees B, Hall P, et al. Multicenter randomized controlled trial of withdrawal of inhaled corticosteroids in cystic fibrosis.  Am J Respir Crit Care Med  2006; 173:1356-62. This study of 171 children and adults with mean baseline FEV1 of 76% predicted found no difference in to time to 1st exacerbation, or use of rescue bronchodilators and antibiotics over the first 6 months of withdrawing inhaled steroid.  These results suggest that many CF patients may be able to safely discontinue inhaled steroids.
PMID:  16556691
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Elkins MR, Robinson M, Rose BR, et al.  A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis.  N Engl J Med  2006; 354:229-40. This study of 164 children and adults with CF found the combination of bronchodilator and 7% saline neb bid had only a modest impact on pulmonary function but reduced exacerbations (76% exacerbation free vs. 62% placebo, p = .03).  These results may not be applicable to patients on more aggressive baseline regimens than the study population.
PMID:  16421364
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Retsch-Bogart GZ, Quittner AL, Gibson RL, et al.  Efficacy and safety of inhaled aztreonam lysine for airway Pseudomonas in cystic fibrosis.  Chest 2009;135:1223-32.   This is the largest published study of inhaled aztreonam, which was recently FDA-approved. 164 children and adults with moderate-to-severe CF lung disease and pseudomonas infection were randomized to receive inhaled aztreonam (75 mg TID) or placebo for 28 days.  The treatment group had no increased adverse events, but had a reduction in productive cough (25% vs 12.5%) as well as significant improvement in multiple CFQ-R scales.  No difference in treatment emergent infections was observed.  This study demonstrated short term safety and benefits.  Results of longer-duration studies are forthcoming. 
PMID:  19420195
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Aaron SD, Vandemheen KL, Ferris W, et al. Combination antibiotic susceptibility testing to treat exacerbations of cystic fibrosis associated with multiresistant bacteria: a randomised, double-blind, controlled clinical trial.  Lancet  2005; 366:463-71.  This study of 132 CF patients found selection of intravenous antibiotics based on multiple combination bactericidal susceptibility testing did not reduce the time to next exacerbation compared to antibiotic selection based on conventional culture and sensitivity tests and there was no difference in the rate of treatment failure. 
PMID:  16084254
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