OBJECTIVES: Sickle Cell Disease (SCD) is a genetic disease impacting ≈300,000 newborns annually, worldwide. Real-life data on SCD are scarce and its public health impact seems underestimated. This study aimed at describing SCD epidemiology and burden in France, using data from the French representative claims data sample called échantillon généraliste des bénéficiaires (EGB).

METHODS: A descriptive, retrospective, longitudinal study based on secondary data from EGB was done. Patients with SCD were detected based on hospitalizations using ICD-10 codes, and Siklos^® (hydroxycarbamide) dispensation. Those aged ≥12 years between January 1^st, 2016 and December 31^st, 2018 with continuous health insurance affiliation were extracted and followed-up for ≥6 months and up to 3 years for SCD burden description. Patients with sickle-cell trait (ICD-10 D57.3) were excluded. Data were extrapolated to the entire French population. Costs were compared to general population, based on a direct matching with 1:3 ratio.

RESULTS: Among the 732,164 individuals from EGB, 318 had at least one SCD-related event. Of them, 153 patients meeting selection criteria were extracted. In 2018, the number [CI₉₅] of patients with SCD in general population was extrapolated to 19,502 [19,230-19,778]. Mean (SD) age was 37,6 (17,4) years, with 69.5% women. Only 11.9% of patients received hydroxycarbamide (any form). Hospitalization rate per 100 patient-year was 12.88 [9.16-17.60]. When compared to general population, SCD estimated annual mean costs were around 8 times greater (8,535€ and 1,067€, respectively). On French scale, SCD attributable burden was assessed at almost 150 million € over cost annually, mostly (>80%) due to hospitalizations.

CONCLUSIONS: This study estimated the number of patients with SCD in France at nearly 20,000, steadily increasing over the years, and highlighted its important economic burden and public health impact. With the low-use of hydroxycarbamide and only few therapeutic alternatives, an important unmet medical need could be tackled, notably regarding SCD chronic management.