Data that empower: The success and promise of CF patient registries

In this article, we describe existing CF registries with a focus on US registry data collected through the CF Foundation Patient Registry (CFFPR) and the Epidemiologic Study of CF (ESCF); highlight what registries have taught us regarding epidemiology of CF; showcase the impact of registries on research and clinical care; and discuss future directions. This manuscript complements the plenary address given by Dr Wayne Morgan at the 2016 North American CF Conference by summarizing the key points from the presentation and providing additional detail and information.

[1]  P. Wise,et al.  Assessing Differences in Mortality Rates and Risk Factors Between Hispanic and Non-Hispanic Patients With Cystic Fibrosis in California. , 2016, Chest.

[2]  Garry R. Cutting,et al.  Cystic fibrosis genetics: from molecular understanding to clinical application , 2014, Nature Reviews Genetics.

[3]  Aliza K Fink,et al.  The Cystic Fibrosis Foundation Patient Registry. Design and Methods of a National Observational Disease Registry. , 2016, Annals of the American Thoracic Society.

[4]  Erika D. Lease,et al.  Heterogeneity in Survival in Adult Patients With Cystic Fibrosis With FEV1 < 30% of Predicted in the United States , 2017, Chest.

[5]  A. Elliott,et al.  Gender differences in outcomes of patients with cystic fibrosis. , 2014, Journal of women's health.

[6]  S. Emerson,et al.  Effect of genotype on phenotype and mortality in cystic fibrosis: a retrospective cohort study , 2003, The Lancet.

[7]  N. Morris,et al.  IV-treated pulmonary exacerbations in the prior year: An important independent risk factor for future pulmonary exacerbation in cystic fibrosis. , 2016, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[8]  G. Sawicki,et al.  Rate of Uptake of Ivacaftor Use after U.S. Food and Drug Administration Approval among Patients Enrolled in the U.S. Cystic Fibrosis Foundation Patient Registry. , 2015, Annals of the American Thoracic Society.

[9]  C. Goss,et al.  Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study. , 2017, The Lancet. Respiratory medicine.

[10]  G. Sawicki,et al.  Sustained Benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data. , 2015, American journal of respiratory and critical care medicine.

[11]  M S Pepe,et al.  Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. , 1999, The New England journal of medicine.

[12]  R. Kahn,et al.  Median household income and mortality rate in cystic fibrosis. , 2003, Pediatrics.

[13]  S. McColley,et al.  Risk factors for mortality before age 18 years in cystic fibrosis , 2017, Pediatric pulmonology.

[14]  Charles A. Johnson,et al.  Epidemiologic study of cystic fibrosis: Design and implementation of a prospective, multicenter, observational study of patients with cystic fibrosis in the U.S. and Canada , 1999, Pediatric pulmonology.

[15]  P. Bye,et al.  Cystic fibrosis in Australia, 2009: results from a data registry , 2011, The Medical journal of Australia.

[16]  S. Butler,et al.  Estimating effectiveness in an observational study: a case study of dornase alfa in cystic fibrosis. The Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis. , 1999, The Journal of pediatrics.

[17]  K. Rothman,et al.  Mortality of Cystic Fibrosis Patients Treated with Tobramycin Solution for Inhalation , 2003, Epidemiology.

[18]  J. Abbott,et al.  International Committee on Mental Health in Cystic Fibrosis: Cystic Fibrosis Foundation and European Cystic Fibrosis Society consensus statements for screening and treating depression and anxiety , 2015, Thorax.

[19]  W. Morgan,et al.  Forced Expiratory Volume in 1 Second Variability Helps Identify Patients with Cystic Fibrosis at Risk of Greater Loss of Lung Function. , 2016, The Journal of pediatrics.

[20]  Margaret Rosenfeld,et al.  Early anti-pseudomonal acquisition in young patients with cystic fibrosis: rationale and design of the EPIC clinical trial and observational study'. , 2009, Contemporary clinical trials.

[21]  C. Goss,et al.  Predictors of non-referral of patients with cystic fibrosis for lung transplant evaluation in the United States. , 2016, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[22]  I. Nathanson,et al.  Association between practice patterns and body mass index percentile in infants and young children with cystic fibrosis. , 2008, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[23]  J. Neglia,et al.  Cancer risk in nontransplanted and transplanted cystic fibrosis patients: a 10-year study. , 2003, Journal of the National Cancer Institute.

[24]  D. Sanders,et al.  Risk factors for lung function decline in a large cohort of young cystic fibrosis patients , 2015, Pediatric pulmonology.

[25]  C. Goss,et al.  CFTR genotype as a predictor of prognosis in cystic fibrosis. , 2006, Chest.

[26]  M. Rieder,et al.  Exome sequencing of extreme phenotypes identifies DCTN4 as a modifier of chronic Pseudomonas aeruginosa infection in cystic fibrosis , 2012, Nature Genetics.

[27]  N. Dreyer,et al.  Registries for Evaluating Patient Outcomes: A User’s Guide , 2010 .

[28]  M. Rieder,et al.  Exome Sequencing of Phenotypic Extremes Identifies CAV2 and TMC6 as Interacting Modifiers of Chronic Pseudomonas aeruginosa Infection in Cystic Fibrosis , 2015, PLoS genetics.

[29]  Y. Castier,et al.  Survival benefit of lung transplant for cystic fibrosis since lung allocation score implementation. , 2013, American journal of respiratory and critical care medicine.

[30]  W. Morgan,et al.  Relationship of Antibiotic Treatment to Recovery after Acute FEV1 Decline in Children with Cystic Fibrosis , 2017, Annals of the American Thoracic Society.

[31]  Emily A. Knapp,et al.  Longevity of Patients With Cystic Fibrosis in 2000 to 2010 and Beyond: Survival Analysis of the Cystic Fibrosis Foundation Patient Registry , 2014, Annals of Internal Medicine.

[32]  Nancy A Dreyer,et al.  Registries for robust evidence. , 2009, JAMA.

[33]  J. Adjemian,et al.  Epidemiology of nontuberculous mycobacterial infections and associated chronic macrolide use among persons with cystic fibrosis. , 2013, American journal of respiratory and critical care medicine.

[34]  Emily A. Knapp,et al.  Changing Epidemiology of the Respiratory Bacteriology of Patients With Cystic Fibrosis. , 2016, Chest.

[35]  C. Goss,et al.  Disparities in access to lung transplantation for patients with cystic fibrosis by socioeconomic status. , 2012, American journal of respiratory and critical care medicine.

[36]  Emily A. Knapp,et al.  Children and young adults with CF in the USA have better lung function compared with the UK , 2014, Thorax.

[37]  C. Goss,et al.  The yin and yang of indoor airborne exposures to endotoxin. , 2013, American journal of respiratory and critical care medicine.

[38]  S. Stanojevic,et al.  Effect of pulmonary exacerbations on long-term lung function decline in cystic fibrosis , 2011, European Respiratory Journal.

[39]  A. Berlinski,et al.  Redesigning care to meet national recommendation of four or more yearly clinic visits in patients with cystic fibrosis , 2014, BMJ quality & safety.

[40]  A. Chakravarti,et al.  Relative contribution of genetic and nongenetic modifiers to intestinal obstruction in cystic fibrosis. , 2006, Gastroenterology.

[41]  C. Merlo,et al.  Association between respiratory tract methicillin-resistant Staphylococcus aureus and survival in cystic fibrosis. , 2010, JAMA.

[42]  W. Morgan,et al.  Probability of treatment following acute decline in lung function in children with cystic fibrosis is related to baseline pulmonary function. , 2013, The Journal of pediatrics.

[43]  R. Lewis,et al.  Confounding by Indication in Clinical Research. , 2016, JAMA.

[44]  Margaret Rosenfeld,et al.  Pulmonary exacerbations are associated with subsequent FEV1 decline in both adults and children with cystic fibrosis , 2011, Pediatric pulmonology.

[45]  Margaret Rosenfeld,et al.  Developing cystic fibrosis lung transplant referral criteria using predictors of 2-year mortality. , 2002, American journal of respiratory and critical care medicine.

[46]  J. Emerson,et al.  Pseudomonas aeruginosa and other predictors of mortality and morbidity in young children with cystic fibrosis , 2002, Pediatric pulmonology.

[47]  C. Goss,et al.  An official American Thoracic Society research statement: comparative effectiveness research in pulmonary, critical care, and sleep medicine. , 2013, American journal of respiratory and critical care medicine.

[48]  W. Warwick,et al.  Survival patterns in cystic fibrosis , 1975 .

[49]  S. Stanojevic,et al.  A standardized approach to estimating survival statistics for population-based cystic fibrosis registry cohorts. , 2016, Journal of clinical epidemiology.

[50]  L. Saiman,et al.  Respiratory microbiology of patients with cystic fibrosis in the United States, 1995 to 2005. , 2009, Chest.

[51]  T. Liou,et al.  Predictive 5-year survivorship model of cystic fibrosis. , 2001, American journal of epidemiology.

[52]  H. Quinton,et al.  Better nutritional status in early childhood is associated with improved clinical outcomes and survival in patients with cystic fibrosis. , 2013, The Journal of pediatrics.

[53]  C. Goss,et al.  The Cystic Fibrosis Foundation Patient Registry as a tool for use in quality improvement , 2014, BMJ quality & safety.

[54]  P. Margolis,et al.  The association of socioeconomic status with outcomes in cystic fibrosis patients in the United States. , 2001, American journal of respiratory and critical care medicine.

[55]  P. Burgel,et al.  Future trends in cystic fibrosis demography in 34 European countries , 2015, European Respiratory Journal.

[56]  S. Stanojevic,et al.  Survival Comparison of Patients With Cystic Fibrosis in Canada and the United States , 2017, Annals of Internal Medicine..

[57]  Charles A. Johnson,et al.  Risk factors for rate of decline in forced expiratory volume in one second in children and adolescents with cystic fibrosis. , 2007, The Journal of pediatrics.

[58]  S. Paranjape,et al.  Baseline Ultrasound and Clinical Correlates in Children with Cystic Fibrosis. , 2015, The Journal of pediatrics.

[59]  J. Emerson,et al.  Risk factors for age at initial Pseudomonas acquisition in the cystic fibrosis epic observational cohort. , 2012, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[60]  J. Emerson,et al.  Impact of Sustained Eradication of New Pseudomonas aeruginosa Infection on Long-term Outcomes in Cystic Fibrosis. , 2015, Clinical infectious diseases : an official publication of the Infectious Diseases Society of America.

[61]  M. Davidian,et al.  Survival Benefit of Lung Transplantation in the Modern Era of Lung Allocation , 2016, Annals of the American Thoracic Society.

[62]  Charles A. Johnson,et al.  Factors influencing outcomes in cystic fibrosis: a center-based analysis. , 2003, Chest.

[63]  F R Adler,et al.  Survival effect of lung transplantation among patients with cystic fibrosis. , 2001, JAMA.

[64]  David Rodman,et al.  Cystic fibrosis adult care: consensus conference report. , 2004, Chest.

[65]  G. Cutting,et al.  Quantification of the relative contribution of environmental and genetic factors to variation in cystic fibrosis lung function. , 2010, The Journal of pediatrics.

[66]  G. Sawicki,et al.  Reduced mortality in cystic fibrosis patients treated with tobramycin inhalation solution , 2012, Pediatric pulmonology.

[67]  M. Corey,et al.  A comparison of survival, growth, and pulmonary function in patients with cystic fibrosis in Boston and Toronto. , 1988, Journal of clinical epidemiology.