The burden of cystic fibrosis in Belgium: a registry-based study

Background: Cystic fibrosis is the most common life-limiting autosomal recessive disease among people of European heritage. This rare disease is known to cause substantial distress on patient level. However, the specific impact on individual health as well as the national impact on public health remains to be elucidated. The aim of this dissertation is to quantify and elaborate the disease burden of cystic fibrosis in Belgium.

Methodology: The Belgian Cystic Fibrosis Registry provides data concerning cystic fibrosis patients in Belgium. These data are used as the primary input to calculate the burden of cystic fibrosis in 2014 in Belgium. Disability-adjusted life years (DALYs), the sum of years of life lost (YLLs) and years lived with disability (YLDs), are calculated using a prevalence approach (hybrid model) in a multi-aspect disease model.

Results: In 2014, patients with cystic fibrosis in Belgium are accountable for 484.8 YLLs and 437.6 YLDs, corresponding with 922.4 DALYs in total (0.75 DALYs per patient). The YLD component consists of primary lung dysfunction (242.7 YLDs), non-transplantation-related complications (143.2 YLDs) and transplantation-related complications (51.6 YLDs). Non-transplantation-related complications are further divided into respiratory complications (11.1 YLDs), digestive/endocrine complications (79.5 YLDs) and other complications (52.6 YLDs). On population level, each complication category is largely explainable by one type of complication, respectively nasal polyps (10.5 YLDs), pancreatic dysfunction (44.2 YLDs) and psychological/psychiatric diseases (25.2 YLDs). The latter complication is found to have the biggest burden on the level of the patient.

Conclusions: Cystic fibrosis is the paragon of rare diseases. As a separate entity, its impact on public health is unpretentious. Nonetheless, this burden of disease study strongly suggests that cystic fibrosis should receive sufficient public health support in order to decrease the disease burden. Public health policies should emphasize certain disease-related topics because of their impact on public health and/or individual health.