Project Manager stève consultants, Oullins, France, France
Background: SCD is a severe hematological disease leading to several complications that significantly impact patients’ quality of life and result in a shorter life expectancy. Epidemiological and disease burden data are however scarce.
Objectives: The study objectives were to determine the prevalence of SCD and to assess its clinical burden in France.
Methods: This was a population-based observational study using secondary data from the French National Health Data System (Système National des Données de Santé). Patients alive on or born after January 1st, 2012, with at least one SCD-related record (i.e. hospital diagnosis and/or long-term disease and/or specific treatment) between 2009 and 2018 were included. Patients cover with general scheme were followed from index date (i.e. date of SCD identification or January 1st, 2012 for existing cases) to death or December 1st, 2018, whichever occurred first; and classified as non-severe patients or most severely affected patients (i.e. with history of vaso-occlusive pain events [VOEs] or dispensations of hydroxycarbamide [HU] or transfusion procedures).
Results: Over the study period, 22,619 patients with SCD were identified. The number of SCD patients increased on average of 7.1% per year, from 15,203 in 2012 to 21,668 in 2018. 20,412 patients were followed, among which 4,270 (20.9%) were defined as most severely affected patients. 15,954 patients (78.2%) experienced at least one acute SCD-related event, notably vaso-occlusion (VOC) episodes and acute chest syndromes (ACS) whose rates were respectively 86.29 [95CI%: 85.75; 86.83] and 12.90 [95%CI: 12.69; 13.11] per 100 person-years in whole SCD 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. 5,470 patients (26.8%) experienced at least one chronic SCD-related event, with higher figures for most severely affected patients compared to whole SCD population: 15.4% (n=656) vs. 8.1% (n=1,651) for cardiovascular diseases, 15.5% (n=662) vs. 6.6% (n=1,351) for osteonecrosis, 13.9% (n=593) vs 4.0% (n=819) for iron overload, 7.9% (n=338) vs. 6.6% (n=1,350) for kidney disease, and 9.3% (n=399) vs. 4.7% (n=961) for pulmonary thrombotic events. During follow-up, 1,062 (5.2%) SCD patients died, among whom 248 (23.4%) were most severely affected.
Conclusions: Prevalence of SCD steadily increases, and the disease is associated with high morbidity and mortality, especially for most severely affected patients. To our knowledge, this is the first study to cover the entire French population, allowing to precisely characterize the burden of SCD and its evolution in the context of new SCD treatments.
