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Observational Study
. 2015 Aug;50(8):763-70.
doi: 10.1002/ppul.23217. Epub 2015 Jun 9.

Risk factors for lung function decline in a large cohort of young cystic fibrosis patients

Jonathan Cogen  1 , Julia Emerson  1 , Don B Sanders  2 , Clement Ren  3 , Michael S Schechter  4 , Ronald L Gibson  1 , Wayne Morgan  5 , Margaret Rosenfeld  1 ; EPIC Study Group
Collaborators, Affiliations
Observational Study

Risk factors for lung function decline in a large cohort of young cystic fibrosis patients

Jonathan Cogen et al. Pediatr Pulmonol. 2015 Aug.

Abstract

Objective: To identify novel risk factors and corroborate previously identified risk factors for mean annual decline in FEV1% predicted in a large, contemporary, United States cohort of young cystic fibrosis (CF) patients.

Methods: Retrospective observational study of participants in the EPIC Observational Study, who were Pseudomonas-negative and ≤12 years of age at enrollment in 2004-2006. The associations between potential demographic, clinical, and environmental risk factors evaluated during the baseline year and subsequent mean annual decline in FEV1 percent predicted were evaluated using generalized estimating equations.

Results: The 946 participants in the current analysis were followed for a mean of 6.2 (SD 1.3) years. Mean annual decline in FEV1% predicted was 1.01% (95%CI 0.85-1.17%). Children with one or no F508del mutations had a significantly smaller annual decline in FEV1 compared to F508del homozygotes. In a multivariable model, risk factors during the baseline year associated with a larger subsequent mean annual lung function decline included female gender, frequent or productive cough, low BMI (<66th percentile, median in the cohort), ≥1 pulmonary exacerbation, high FEV1 (≥115% predicted, in the top quartile), and respiratory culture positive for methicillin-sensitive Staphylococcus aureus, methicillin-resistant S. aureus, or Stenotrophomonas maltophilia.

Conclusions: We have identified a range of risk factors for FEV1 decline in a large cohort of young, CF patients who were Pa negative at enrollment, including novel as well as previously identified characteristics. These results could inform the design of a clinical trial in which rate of FEV1 decline is the primary endpoint and identify high-risk groups that may benefit from closer monitoring.

Keywords: EPIC Observational Study; Pseudomonas aeruginosa; cystic fibrosis; decline; lung function; pediatric.

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Figures

Figure 1
Figure 1. Schematic of study flow
Figure 2
Figure 2. Selection of study cohort
Figure 3
Figure 3
Forest plot of effect of risk factors on annual change in FEV1% predicted, from univariate GEE models. For each risk factor, estimates of mean annual change in FEV1% predicted and associated 95% confidence intervals are displayed for participants with the risk factor relative to participants in the reference category. All risk factors were measured during the baseline year. Based on evaluation of quartiles, best baseline FEV1% predicted was dichotomized at the top quartile value (115%), and best baseline weight and BMI percentiles were dichotomized at the median value (50th and 66th percentile, respectively).
See this image and copyright information in PMC

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