The European Cystic Fibrosis Society Patient Registry (ECFSPR) data validation programme: accuracy and consistency of data

Background: The ECFSPR database for 2016 contains data of 44,719 patients from 31 countries. Data of high quality is essential for use in annual reports and epidemiological research. Methods: A validation programme was introduced to quantify consistency and accuracy of data-input at source level, with on-site visits to countries entering data directly in the ECFSTracker software. Data fields to verify: demographic, diagnostic and transplantation, anthropometric and best lung function measurement, bacterial infections, medications and complications. Accuracy was defined as the proportion of values entered in ECFSTracker matching the medical record, and definitions used by the ECFSPR (consistency) for randomly selected cases. Results: Ten out of 41 centres (24%) in 4 countries (Austria, Portugal, Slovakia, Switzerland), reporting 6550% of all patients in their countries, were selected. Demographic, diagnostic and transplant data were checked for 489 patients (21%*), clinical data for 463 patients (20%*) (2016 data). Data on birth, gender, and transplantation exceeded 98.8% accuracy. Anomalies on reported mutations was 0.9%; reliable source data based on genetic reports, were available in 3 out of 4 countries in 95,9%- 91,9% of all patients, 55,5% in one country. Antropometry (92,2%), lung function (86,4%), inhaled antibiotics (96.1%), DNase (89.1%), pancreatic enzyme use (97.6%) were accurate and consistent with the ECFSPR definitions, so were chronic Pseudomonas (95.0%), Burkholderia infection (97.0%), and hemoptysis (94.6%). Liver disease was reported inconsistently due to different interpretation of the definition and resulted in an accuracy of 86.8%. Conclusions: The ECFSPR dataset is highly accurate for most data verified at source level. To further optimize we recommend centres to use a reliable source for genetic information, adhere to the definition of best lung function, and the ECFSPR to redefine liver disease. *of the total patients in these countries

Epidemiology of European adults with Cystic Fibrosis

The European Cystic Fibrosis Society Patient Registry (ECFSPR) collects anonymised demographic and clinical data from consenting people with CF in Europe. The aim of this study is to describe the adult population with CF in Europe. We considered patients of 18 years or older in the 2017 data, the latest year of follow-up available. Countries with a coverage below 80% were not considered when reporting figures by country. Percentages are computed for categorical variables; quartiles for numerical variables. For the year 2017, the ECFSPR database contains data of 48,204 patients from 35 countries. 24,491 (51.3%) are adults. The percentage of adults varies from 6.5% in Albania to 62.8% in Sweden. The proportion of adult females varies from 37.1% in North Macedonia to 59.6% in Slovenia. The highest percentage of patients living with lung transplant is 22.7% in Slovenia. Considering patients of 18-29 years, the lowest percentage of chronic Pseudomonas Aeruginosa is 30.6% in France, the highest is 79.4% in Serbia. For chronic Burkholderia cepacia complex species the lowest percentage of infected patients is 2.5% in Italy, the highest 20.6% in Serbia. The lowest percentage of CF related diabetes is 7.3% in Slovak Republic, the highest 34.2% in Czech Republic. Median of BMI changes from 18.8 in the Russian Federation to 22.4 kg/m2 in Luxembourg. Considering patients not transplanted, we observe a percentage of patients with FEV1% below 40% that varies from 3.0% in Denmark to 28.5% in Russian Federation. We considered only countries with a coverage of 80% or higher, for a realistic reflection of CF and observed variation among those countries. Data show that the percentage of adult population is higher in North Europe, infections are more frequent in Eastern Europe and growth and lung function are worse in Eastern Europe. Further investigation is required to investigate differences in healthcare systems to plan an adequate development of CF care services for adult CF people