Predictive factors of survival in people with Cystic fibrosis from a Spanish registry of rare diseases.

Abstract

OBJECTIVE: In Spain, several studies describe the population with cystic fibrosis (CF) and analyze mortality trends, but few determine factors that influence survival. The objective of this study was to analyze survival in people with CF included in a population-based registry of rare diseases and determine factors that influence it. METHODS: An observational study was carried out in people with a confirmed diagnosis of CF until December 31, 2018 registered in the Rare Diseases Information System of the Region of Murcia (SIERrm). The Kaplan-Meier method and the log-rank test were used to estimate and compare survival curves, and the Cox proportional hazards model was used to calculate survival predictors. RESULTS: Of 192 patients registered in SIERrm on the date of the study, 39 died, with the median age of survival being 26 years. The high-risk genotype (HR: 5.8 [95%CI: 1.97-16.94]), pancreatic insufficiency (HR: 5.4 [95%CI: 1.24-23.48]), colonization chronic by Pseudomonas aeruginosa (HR: 6.8 [95%CI: 1.21-38.54]), CF-related liver disease (HR: 5.0 (95%CI: 1.61-15.61]), and bone abnormalities (HR: 6.4 [95%CI: 1.91-21.61]) were associated with decreased survival (p<0.05). Adjusting the model for genotype, the association with high-risk genotype, CF-related liver disease, and bone changes remained (p<0.05). CONCLUSIONS: Population registries are useful to identify patients at risk of serious disease due to their genetic and clinical characteristics, who may benefit from specific treatments and health resources that influence factors that reduce survival.