Abstract

To estimate the hemofilia A (HA) prevalence and to analyse among HA patients the drug-utilization patterns and direct healthcare costs. Observational retrospective study based on administrative databases of one Italian region (sample population around 4.9million). Between 01/2010—06/2019, patients were included as “confirmed-HA” if they presented a HA diagnosis (ICD-9-CM=286.0) or prescriptions for coagulation factor (F)VIII (ATC B02BD02); as “potential-HA” if they received von Willebrand factor and FVIII in combination (ATC code B02BD06 or B02BD02 plus B02BD10). Index date corresponded to the date of first match with inclusion criteria. Drug-utilization and costs analysis were performed on confirmed HA patients receiving FVIII during the last 2 years of study period (up-to-date cohort). Prophylaxis and on-demand regimens were identified with FVIII International Unit (IU) respectively higher and lower than defined thresholds set for specific age-groups. HA prevalence was 71/1,000,000 inhabitants: confirmed-HA 60.83/1,000,000, potential-HA 10.17/1,000,000. Overall,387 patients(mean age=45.6±24.3) were included, 334 of which with confirmed-HA (mean age=44.7±24.7). Considering the up-to-date cohort(N=168), 46.8%(mean age=26.1±17.3) of patients received prophylaxis, 53.2%(mean age=40.7±22.3) on-demand. In the last 6-months of study period, 86.3% of patients still received FVIII. Mean annual FVIII IU dispensed was 289,081.1(prophylaxis) and 46,620.8(on-demand). Total IU consumption (prophylaxis and on-demand) lowered with increasing age, in line with a downward trend of prophylaxis in older age-groups. Mean total annual healthcare cost was €99,477(€185,047 for patients in prophylaxis, €32,114 for those on-demand). Alongside with IU consumption results, costs were higher in patients ≤12 years(€50,2010) than ≥65 years(€16,476.). Moreover, a greater consumption of most-expensive therapy was observed in younger patients. This study provided the most up-to-date insights on HA in Italian real-world settings. Prevalence was consistent with the expected one reported by AICE(6.9/100,000), showing the usefulness of administrative databases for HA identification. Moreover, drug-utilization and costs investigation using administrative data could be helpful to inform health decision making.

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