Severity and burden of sickle cell disease in France: a nationwide realworld study

Abstract

The burden of sickle cell disease (SCD) in France has been difficult to apprehend due to the paucity of reliable nationwide epidemiological data. We aimed to describe the epidemiology of SCD and evaluate its burden and costs. Patients with SCD and most severely affected patients were identified between 2012 and 2018 from the French National Health Data System database (SNDS, Systeme national des donnees de sante). Outcomes of interest included rates of acute and chronic complications, healthcare resource utilisation and associated costs, and were compared in subpopulations of patients before and after Hematopoietic Stem Cell Transplantation (HSCT), initiating hydroxyurea (HU) or a chronic transfusion program (CTP). Between 2012 and 2018, 22,619 patients with SCD were identified, among which 4,270 patients were defined as most severely affected. Rates of vaso-occlusion (VOC) episodes and acute chest syndrome (ACS) were 86.29 [95CI%: 85.75; 86.83] and 12.90 [95%CI: 12.69; 13.11] per 100 person-years in study population and 166.9 [95%CI: 165.4; 168.4] and 22.71 [95%CI: 22.16; 23.27] per 100 person-years in most severely affected patients. Median (Q1-Q3) annualised total costs were €5,073.63 (1,633.74-14,000.94) and €13,295.67 (5,754.67-26,385.23) in study population and most severely affected patients. Median annualised costs were 10 times lower after treatment intensification for HSCT (€29,011.75 vs €2,465.98, p