Cystic Fibrosis Mortality in Childhood. Data from European Cystic Fibrosis Society Patient Registry

Anna Zolin,Rita Padoan,Natalia Cirilli,Nataliya Kashirskaya,Anna Bossi

doi:10.3390/ijerph15092020

Abstract

Data collected in the European Cystic Fibrosis Society Patient Registry (ECFSPR) database were used to investigate whether risk factors for death in childhood and adolescents CF patients have different impact in countries of different income. In this way, it is possible to recognize where interventions could improve the quality of care and survival in these patients. We matched deceased and alive patients by age, country, year of follow-up. Multivariable logistic models were developed. In the years of this study, the ECFSPR collected information on 24,416 patients younger than 18 years: 7830 patients were from countries with low/middle income and 16,586 from countries with high income; among these the dead are 102 and 107 (p < 0.001), respectively. The use of oxygen, forced expiratory volume in one second (FEV1) below 40% and BMI standard deviation score (SDS) below −2 represent risk factors for death. However, some patients from countries with high income remain alive even if their values of FEV1% and BMI-SDS were low, and some deceased patients from countries with high income had high values of FEV1% (>60%). Evaluation of mortality in pediatric age may reflect the availability of resources for CF diagnosis and treatment in some countries.

Highlights

Recent reports from Australian, U.S and European cystic fibrosis (CF) registries [1,2,3] show that adult patients represent more than 50% of the CF population, and the mean age of CF population is slowly but steadily increasing
The aim of this study is to investigate whether the risk factors for death reported in literature have different impact in countries of different income, so as to recognize the areas where interventions could improve the quality of care and survival of CF patients
(16.7%, this category can include rare complications: only a limited number of cause of death is collected by the European Cystic Fibrosis Society Patient Registry (ECFSPR)), transplantation (10.2%), non-CF related causes (10.2%), liver or gastro-intestinal disease (4.3%), trauma (1.6%). 11.83% of patients died before their second birthday and 67.75% died between 10 and 17 years. 65.59% of the deceased patients were female: both for males and females more than 60% of the patients died between 10 and 17 years

Summary

Introduction

Recent reports from Australian, U.S and European cystic fibrosis (CF) registries [1,2,3] show that adult patients represent more than 50% of the CF population, and the mean age of CF population is slowly but steadily increasing. 2014 data from registries report a proportion of death in paediatric age ranging from 10% to 20% [Australia = 10.5% (two out of 19 patients); U.S = 11.9%. Kerem et al [4] investigated the associations between forced expiratory volume in one second (FEV1 ) and clinical outcomes in the European Cystic Fibrosis Society Patient Registry (ECFSPR). Showed a statistically significant and clinically important effect on FEV1. McColley et al [5]

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