Longevity of Patients With Cystic Fibrosis in 2000 to 2010 and Beyond: Survival Analysis of the Cystic Fibrosis Foundation Patient Registry

Abstract

Background Advances in treatments for cystic fibrosis (CF) continue to extend survival. An updated estimate of survival is needed for better prognostication and to anticipate evolving adult care needs. Objective To characterize trends in CF survival between 2000 and 2010 and to project survival for children born and diagnosed with the disease in 2010. Design Registry-based study. Setting 110 Cystic Fibrosis Foundation–accredited care centers in the United States. Patients All patients represented in the Cystic Fibrosis Foundation Patient Registry (CFFPR) between 2000 and 2010. Measurements Survival was modeled with respect to age, age at diagnosis, gender, race or ethnicity, F508del mutation status, and symptoms at diagnosis. Results Between 2000 and 2010, the number of patients in the CFFPR increased from 21 000 to 26 000, median age increased from 14.3 to 16.7 years, and adjusted mortality decreased by 1.8% per year (95% CI, 0.5% to 2.7%). Males had a 19% (CI, 13% to 24%) lower adjusted risk for death than females. Median survival of children born and diagnosed with CF in 2010 is projected to be 37 years (CI, 35 to 39 years) for females and 40 years (CI, 39 to 42 years) for males if mortality remains at 2010 levels and more than 50 years if mortality continues to decrease at the rate observed between 2000 and 2010. Limitations The CFFPR does not include all patients with CF in the United States, and loss to follow-up and missing data were observed. Additional analyses to address these limitations suggest that the survival projections are conservative. Conclusion Children born and diagnosed with CF in the United States in 2010 are expected to live longer than those born earlier. This has important implications for prognostic discussions and suggests that the health care system should anticipate greater numbers of adults with CF. Primary Funding Source Cystic Fibrosis Foundation.

Changing Epidemiology of the Respiratory Bacteriology of Patients With Cystic Fibrosis

“…CFFPR data demonstrate that more recent cohorts have better lung function than previously reported age-matched cohorts, thanks to numerous advances in CF-specific therapies, suggesting that children and adolescents have less structural lung disease and, therefore, less risk for infection. 2 The 21 which may also improve overall lung health. For over a decade, CF centers have been progressively implementing more rigorous infection prevention and control strategies to reduce the risk for patient-to-patient transmission of CF pathogens and acquisition from the natural and/or health-care environment.…”

“…1,2 However, progressive lung disease associated with chronic respiratory infections and inflammation remains the most common cause of morbidity and mortality in CF. 3 Monitoring changes in the epidemiology of CF pathogens is essential to optimally manage CF lung disease and to understand the potential impact of therapeutic interventions on CF microbiology.…”

Minorities are Underrepresented in Clinical Trials of Pharmaceutical Agents for Cystic Fibrosis

“…By 2014, the fraction of 18-year-olds with normal lung function had increased to 72% (1). Median predicted survival improved from 14 years in the 1960s to 32 years in 2000 and to 39.3 years in 2014 (1,2). Improvements in morbidity and mortality are attributable, at least in part, to the development and approval of new pharmaceutical agents targeting pulmonary infections, airway inflammation, and the cystic fibrosis transmembrane conductance regulator (CFTR) abnormality in CF.…”

The Analysis of Cystic Fibrosis in the Federation of Bosnia and Herzegovina

“…Survival of patients with CF has dramatically improved since the disease was discovered, 70 years ago. Progressive pulmonary disease is leading to morbidity and pulmonary arrest, and the average life expectancy of people with 37.4 years, 37 years for women and 40 years for men (13,14). Progress in the length of life of patients is the result of many new improvement in treating disease.…”