{Reference Type}: Observational Study
{Title}: An observational study of haemophilia A patients without inhibitors using the French national claims (SNDS) database.
{Author}: Trossaërt M;Falk A;Gautier L;Kragh N;Van Hinloopen O;Varin R;
{Journal}: Hematology
{Volume}: 29
{Issue}: 1
{Year}: 2024 Dec
{Factor}: 2.264
{DOI}: 10.1080/16078454.2024.2320610
{Abstract}: <B>UNASSIGNED: </B>To describe clinical characteristics, factor consumption, and events of interest in patients with haemophilia A without inhibitors receiving prophylaxis in France, and the clinical impact of switching to Elocta® in this population.<BR><B>UNASSIGNED: </B>This retrospective, observational study using the Système National des Données de Santé database, analysed data from patients with haemophilia A without inhibitors using prophylactic factor VIII (FVIII) replacement therapy during 2016-2019. Clinical characteristics, treatment patterns and switches, factor consumption, and rate of events of interest were determined. In a sub-cohort of patients treated with Elocta®, clinical characteristics, factor consumption, and rate of events of interest before and after switching to Elocta® were compared.<BR><B>UNASSIGNED: </B>For 545 patients, with mean age (standard deviation [SD]) 25.4 (17.8) years, Elocta® was the most used treatment. Bleeding events and articular non-bleeding events leading to hospitalization occurred in 15.4% and 13.9% of patients, respectively, and 9.9% of patients had surgeries or procedures related to haemophilic arthropathy. The mean (SD) FVIII product consumption was 344 (93) IU/kg/month for extended half-life treatment, and 331 (98) IU/kg/month for standard half-life products. For the sub-cohort of 146 patients, bleeding events (SD) decreased from 0.32 (2.2) to 0.09 (0.42) events/patient/year (p = 0.227) after switching to Elocta®. There was no statistically significant difference in rates of factor consumption or articular non-bleeding events before and after initiation of Elocta®.<BR><B>UNASSIGNED: </B>This study provides real-world insights that advance the understanding of treatment patterns and events of interest in patients with haemophilia A on prophylactic regimens in France.