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Development and Internal Validation of a Prognostic Model of the Probability of Death or Lung Transplantation Within 2 Years for Patients With Cystic Fibrosis and FEV 1  ≤ 50% Predicted.

Authors :
Ramos KJ
Hee Wai T
Stephenson AL
Sykes J
Stanojevic S
Rodriguez PJ
Bansal A
Mayer-Hamblett N
Goss CH
Kapnadak SG
Source :
Chest [Chest] 2022 Oct; Vol. 162 (4), pp. 757-767. Date of Electronic Publication: 2022 May 26.
Publication Year :
2022

Abstract

Background: Improved methods are needed to risk-stratify patients with cystic fibrosis (CF) and reduced FEV <subscript>1</subscript> .<br />Research Questions: What are the predictors of death or lung transplantation (LTx) within 2 years among patients with CF whose FEV <subscript>1</subscript>  ≤ 50% predicted? Do these markers similarly predict outcomes among G551D patients taking ivacaftor since 2012?<br />Study Design and Methods: Patients with CF, age ≥ 6 years with FEV <subscript>1</subscript>  ≤ 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.<br />Results: FEV <subscript>1</subscript> 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 FEV <subscript>1</subscript> < 30% predicted. Among those taking ivacaftor in 2014, only FEV <subscript>1</subscript> 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, FEV <subscript>1</subscript> percent predicted, and cardiac index.<br />Interpretation: Among individuals with CF and FEV <subscript>1</subscript>  ≤ 50% predicted, FEV <subscript>1</subscript> percent predicted, oxygen therapy, and number of pulmonary exacerbations predicted 2-year death or LTx. Although limited by small sample size, only FEV <subscript>1</subscript> remained predictive in patients receiving highly effective modulator therapy. Additional physiologic variables could improve prognostication in CF.<br /> (Copyright © 2022 American College of Chest Physicians. Published by Elsevier Inc. All rights reserved.)

Details

Language :
English
ISSN :
1931-3543
Volume :
162
Issue :
4
Database :
MEDLINE
Journal :
Chest
Publication Type :
Academic Journal
Accession number :
35643116
Full Text :
https://doi.org/10.1016/j.chest.2022.05.021