Abstract

Sickle-cell disease (SCD) is an inherited, multisystem disorder characterised by progressive organ failure and limited life expectancy. It is the most common monogenetic disease worldwide. Despite improvements in treatment, SCD is still marked by a high utilization of medical resources. Previous cost studies from the US and UK focussed on one or two categories of care (i.e. hospitalisations and physician visits). The aim was to investigate overall costs of healthcare for paediatric SCD patients in the Netherlands, where a so-called ‘comprehensive care approach’ is practiced (including active follow-up, neonatal screening and research). We conducted a retrospective cohort study (2015 – 2016) in one of the largest sickle cell reference centers in the Netherlands, the Erasmus MC. Costs were based on the comprehensive standard of care SCD guideline and real-world data from Erasmus MC electronic patient records (including complications and inpatient visits at the Erasmus MC [n = 18] and peripheral hospitals [n = 38]). Total mean annual costs per patient were €7,411 (SD: €12,911) and increased with age (0-1 year, mean: €3,267, SD: €560; 13-19 years, mean: €14,998, SD: €24,441). Major cost drivers were diagnostic tests for patients in the first year of life and hospital visits for the older group (13-19 years). Comprehensive standard care costs accounted for 67% (0-1 years) to 14% (13-19 years) of the total costs. This is the first European SCD cost study that combined both comprehensive- and complication-related care. In contrast to data from the US, we observed lower annual costs per patient. This might be due to differences in healthcare organisation; such as a routine chronic care approach with bi-annual check-ups in the Netherlands versus focusing on acute complications in the US. Furthermore, real-world data show an increase in costs for older children, which is in line with increased SCD complications with age.

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