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Evaluation of a five-year predicted survival model for cystic fibrosis in later time periods.

ABSTRACT: We evaluated a multivariable logistic regression model predicting 5-year survival derived from a 1993-1997 cohort from the United States Cystic Fibrosis (CF) Foundation Patient Registry to assess whether therapies introduced since 1993 have altered applicability in cohorts, non-overlapping in time, from 1993-1998, 1999-2004, 2005-2010 and 2011-2016. We applied Kaplan-Meier statistics to assess unadjusted survival. We tested logistic regression model discrimination using the C-index and calibration using Hosmer-Lemeshow tests to examine original model performance and guide updating as needed. Kaplan-Meier age-adjusted 5-year probability of death in the CF population decreased substantially during 1993-2016. Patients in successive cohorts were generally healthier at entry, with higher average age, weight and lung function and fewer pulmonary exacerbations annually. CF-related diabetes prevalence, however, steadily increased. Newly derived multivariable logistic regression models for 5-year survival in new cohorts had similar estimated coefficients to the originals. The original model exhibited excellent calibration and discrimination when applied to later cohorts despite improved survival and remains useful for predicting 5-year survival. All models may be used to stratify patients for new studies, and the original coefficients may be useful as a baseline to search for additional but rare events that affect survival in CF.

SUBMITTER: Liou TG

PROVIDER: S-EPMC7171119 | biostudies-literature | 2020 Apr

REPOSITORIES: biostudies-literature

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Evaluation of a five-year predicted survival model for cystic fibrosis in later time periods.

Liou Theodore G TG Kartsonaki Christiana C Keogh Ruth H RH Adler Frederick R FR

Scientific reports 20200420 1

We evaluated a multivariable logistic regression model predicting 5-year survival derived from a 1993-1997 cohort from the United States Cystic Fibrosis (CF) Foundation Patient Registry to assess whether therapies introduced since 1993 have altered applicability in cohorts, non-overlapping in time, from 1993-1998, 1999-2004, 2005-2010 and 2011-2016. We applied Kaplan-Meier statistics to assess unadjusted survival. We tested logistic regression model discrimination using the C-index and calibrati ...[more]

PMID: 32313191

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Project description:Rationale: Differences in cystic fibrosis (CF) airway microbiota between periods of clinical stability and exacerbation of respiratory symptoms have been investigated in efforts to better understand microbial triggers of CF exacerbations. Prior studies have often relied on a single sample or a limited number of samples to represent airway microbiota. However, the variability in airway microbiota during periods of clinical stability is not well known.Objectives: To determine the temporal variability of measures of airway microbiota during periods of clinical stability, and to identify factors associated with this variability.Methods: Sputum samples (N = 527), obtained daily from six adults with CF during 10 periods of clinical stability, underwent sequencing of the V4 region of the bacterial 16S ribosomal RNA gene. The variability in airway microbiota among samples within each period of clinical stability was calculated as the average of the Bray-Curtis similarity measures of each sample to every other sample within the same period. Outlier samples were defined as samples outside 1.5 times the interquartile range within a baseline period with respect to the average Bray-Curtis similarity. Total bacterial load was measured with droplet digital polymerase chain reaction.Results: The variation in Bray-Curtis similarity and total bacterial load among samples within the same baseline period was greater than the variation observed in technical replicate control samples. Overall, 6% of samples were identified as outliers. Within baseline periods, changes in bacterial community structure occurred coincident with changes in maintenance antibiotics (P < 0.05, analysis of molecular variance). Within subjects, bacterial community structure changed between baseline periods (P < 0.01, analysis of molecular variance). Sample-to-sample similarity within baseline periods was greater with fewer interval days between sampling.Conclusions: During periods of clinical stability, airway bacterial community structure and bacterial load vary among daily sputum samples from adults with CF. This day-to-day variation has bearing on study design and interpretation of results, particularly in analyses that rely on single samples to represent periods of interest (e.g., clinical stability vs. pulmonary exacerbation). These data also emphasize the importance of accounting for maintenance antibiotic use and granularity of sample collection in studies designed to assess the dynamics of CF airway microbiota relative to changes in clinical state.

Project description:The effects of lung transplantation on the survival and quality of life in children with cystic fibrosis are uncertain.We used data from the U.S. Cystic Fibrosis Foundation Patient Registry and from the Organ Procurement and Transplantation Network to identify children with cystic fibrosis who were on the waiting list for lung transplantation during the period from 1992 through 2002. We performed proportional-hazards survival modeling, using multiple clinically relevant covariates that were available before the children were on the waiting list and the interactions of these covariates with lung transplantation as a time-dependent covariate. The data were insufficient in quality and quantity for a retrospective quality-of-life analysis.A total of 248 of the 514 children on the waiting list underwent lung transplantation in the United States during the period from 1992 through 2002. Proportional-hazards modeling identified four variables besides transplantation that were associated with changes in survival. Burkholderia cepacia infection was associated with a trend toward decreased survival, regardless of whether the patient underwent transplantation. A diagnosis of diabetes before the patient was placed on the waiting list decreased survival while the patient was on the waiting list but did not decrease survival after transplantation, whereas older age did not affect waiting-list survival but decreased post-transplantation survival. Staphylococcus aureus infection increased waiting-list survival but decreased post-transplantation survival. Using age, diabetes status, and S. aureus infection status as covariates, we estimated the effect of transplantation on survival for each patient group, expressed as a hazard factor of less than 1 for a benefit and more than 1 for a risk of harm. Five patients had a significant estimated benefit, 283 patients had a significant risk of harm, 102 patients had an insignificant benefit, and 124 patients had an insignificant risk of harm associated with lung transplantation.Our analyses estimated clearly improved survival for only 5 of 514 patients on the waiting list for lung transplantation. Prolongation of life by means of lung transplantation should not be expected in children with cystic fibrosis. A prospective, randomized trial is needed to clarify whether and when patients derive a survival and quality-of-life benefit from lung transplantation.

Project description:BackgroundThe effects of lung transplantation on the survival and quality of life in children with cystic fibrosis are uncertain.MethodsWe used data from the U.S. Cystic Fibrosis Foundation Patient Registry and from the Organ Procurement and Transplantation Network to identify children with cystic fibrosis who were on the waiting list for lung transplantation during the period from 1992 through 2002. We performed proportional-hazards survival modeling, using multiple clinically relevant covariates that were available before the children were on the waiting list and the interactions of these covariates with lung transplantation as a time-dependent covariate. The data were insufficient in quality and quantity for a retrospective quality-of-life analysis.ResultsA total of 248 of the 514 children on the waiting list underwent lung transplantation in the United States during the period from 1992 through 2002. Proportional-hazards modeling identified four variables besides transplantation that were associated with changes in survival. Burkholderia cepacia infection was associated with a trend toward decreased survival, regardless of whether the patient underwent transplantation [corrected]. A diagnosis of diabetes before the patient was placed on the waiting list decreased survival while the patient was on the waiting list but did not decrease survival after transplantation, whereas older age did not affect waiting-list survival but decreased post-transplantation survival. Staphylococcus aureus infection increased waiting-list survival but decreased post-transplantation survival. Using age, diabetes status, and S. aureus infection status as covariates, we estimated the effect of transplantation on survival for each patient group, expressed as a hazard factor of less than 1 for a benefit and more than 1 for a risk of harm. Five patients had a significant estimated benefit, 283 patients had a significant risk of harm, 102 patients had an insignificant benefit, and 124 patients had an insignificant risk of harm associated with lung transplantation [corrected].ConclusionsOur analyses estimated clearly improved survival for only 5 of 514 patients on the waiting list for lung transplantation. Prolongation of life by means of lung transplantation should not be expected in children with cystic fibrosis. A prospective, randomized trial is needed to clarify whether and when patients derive a survival and quality-of-life benefit from lung transplantation.

Dataset Information

Evaluation of a five-year predicted survival model for cystic fibrosis in later time periods.

Publications

Evaluation of a five-year predicted survival model for cystic fibrosis in later time periods.

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