• Kathleen J Ramos, Hee WaiTravisTDivision of Pulmonary, Critical Care, and Sleep Medicine, Department of Medicine, University of Washington, Seattle, WA., Anne L Stephenson, Jenna Sykes, Sanja Stanojevic, Patricia J Rodriguez, Aasthaa Bansal, Nicole Mayer-Hamblett, Christopher H Goss, and Siddhartha G Kapnadak.
• Division of Pulmonary, Critical Care, and Sleep Medicine, Department of Medicine, University of Washington, Seattle, WA. Electronic address: ramoskj@uw.edu.
• Chest. 2022 Oct 1; 162 (4): 757767757-767.

BackgroundImproved methods are needed to risk-stratify patients with cystic fibrosis (CF) and reduced FEV1.Research QuestionsWhat are the predictors of death or lung transplantation (LTx) within 2 years among patients with CF whose FEV1 ≤ 50% predicted? Do these markers similarly predict outcomes among G551D patients taking ivacaftor since 2012?Study Design And MethodsPatients with CF, age ≥ 6 years with FEV1 ≤ 50% predicted as of December 31, 2014, were identified in a data set that merged Cystic Fibrosis Foundation and United Network for Organ Sharing (UNOS) registries. The least absolute shrinkage and selection operator (LASSO) method was applied to a randomly selected training set to select important prognostic variables. Accuracy and association of the model with death or LTx with 2 years (2-year death or LTx) were validated via logistic regression on an independent test set. Sensitivity analyses explored predictors for patients with UNOS data.ResultsFEV1 percent predicted (OR, 1.51 for 5% decrease; 95% CI, 1.27-1.81), number of pulmonary exacerbations treated with IV antibiotics (OR, 1.35; 95% CI, 1.11-1.65), and continuous or nocturnal oxygen (OR, 3.71; 95% CI, 1.81-7.59) were significantly associated with 2-year death or LTx. Our model predicted outcomes with greater sensitivity (ratio of sensitivity, 1.26; 95% CI, 1.02-1.54), ratio of positive predictive value (1.25; 95% CI, 1.05-1.51), and ratio of negative predictive value (1.04; 95% CI, 1.01-1.07) than FEV1 < 30% predicted. Among those taking ivacaftor in 2014, only FEV1 remained associated with 2-year death or LTx. For patients with UNOS data, LASSO identified additional covariates of interest, including noninvasive ventilation use, low hemoglobin, pulmonary arterial systolic pressure, supplemental oxygen, mechanical ventilation, FEV1 percent predicted, and cardiac index.InterpretationAmong individuals with CF and FEV1 ≤ 50% predicted, FEV1 percent predicted, oxygen therapy, and number of pulmonary exacerbations predicted 2-year death or LTx. Although limited by small sample size, only FEV1 remained predictive in patients receiving highly effective modulator therapy. Additional physiologic variables could improve prognostication in CF.Copyright © 2022 American College of Chest Physicians. Published by Elsevier Inc. All rights reserved.

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