SUMMARY
This report presents the data collected in 2018. It is our hope that the analysis of the registry data will provide readers with information on various aspects of CF and continue to provide an important tool for monitoring the patient’s quality of care and trends.
Since its establishment in 1999, the Belgian CF Registry (BCFR) has grown steadily and had 1320 patients registered in 2018. This number excludes three patients whose diagnosis for CF was revoked and eight others without a confirmed diagnosis. We also miss 1-2% of patients who had not been seen between the introduction of the new GDPR-adapted Informed Consent Form and the data submission. There were 22 newly diagnosed patients in 2018, among them five adults, with a median age at diagnosis of 9.8 months and age range from birth to 70.6 years. All the newly diagnosed patients were genotyped; 18 had sweat chloride values > 60 mmol/L ; 2 had no sweat chloride value yet by December 2018.
Among the patients followed-up in 2018, 51.9% were male and 64.3% adults, and the median age was 23.6 years. At inception of the registry, 18 years ago, 39.0% were adults, and the median age was 14.9 years, showing changes in demographics over the years. 45.8% of the patients are homozygous for the F508del mutation and 38.0% are F508del heterozygous. The main reasons for diagnosis of CF are acute or recurrent respiratory problems (41.9%) and failure to thrive (24.9%). About 19.2% were diagnosed via neonatal screening even though Belgium had no national neonatal screening program at the time. Within the year, sixteen deaths were reported (nine of them among transplanted patients) with age at death ranging from 20.4 to 70.2 years. Sixteen patients benefitted from a lung transplant, and 14.3% of the patients in the registry are living with a transplant.
Among the adults, the proportion of underweight patients (BMI < 18.5 kg/m²), continues to decline from about 31.2% in 1998 to 16.7% in 2010 and 12.0% in 2018. This decline was noted also amongst the F508del homozygous patients; 32.8% in 2000, 21.9% in 2010, and 13.9% in 2018. Amongst the patients up to 20 years, the proportion with BMI below the tenth percentile has also been declining over the years. The results above suggest better nutritional and global management in the patients. The patient population continues to record an improvement in lung function expressed as the mean percentage of predicted FEV1. Among the F508del homozygous patients, 27.6% of the children and 3.5% of the adults had FEV1 ≥ 90.0% of predicted in 1998 compared to 49.5% and 8.4% in 2010 and 52.4% and 16.4% respectively among the children and adults in 2018.
The overall annual prevalence of Pseudomonas aeruginosa reported in 2018 was 37.9%. This is in line with the general decline observed from 42.4% in 2012 to 37.5% in 2016. There has also been a steady increase in the prevalence of Achromobacter xylosoxidans from 5.9% in 2009, stabilizing at prevalence levels of about 10.0% since 2012. The prevalence of the Burkholderia cepacia complex on the other hand has decreased to 3.0% in 2018, after a stable period at about 3.5% over the years since 2014.
Thanks to improved disease management and novel treatments, the life expectancy and the quality of life of patients with CF has improved significantly when compared to CF cohorts a decade or two ago. Cystic Fibrosis is no longer a paediatric disease, however this progress is also accompanied by various challenges, expectations and disease-related complications. In 2018, CF-related diabetes had a prevalence of 4.3% and 29.3% (non-transplanted) in children and adults respectively. Other reported complications include early osteoporosis and CF related arthritis/arthropathy. These require specialized care for the adult CF patients.