An observational study of haemophilia A patients without inhibitors using the French national claims (SNDS) database

Authors: Trossaërt, Marc; Falk, Aletta; Gautier, Laurène; Kragh, Nana; van Hinloopen, Olivia; Varin, Remi

Affiliations: Hemophilia Treatment Centre, University Hospital of Nantes, Nantes, France. Global Medical Affairs and Clinical Science, Sobi, Stockholm, Sweden. Real World Evidence Data and Analytics, Cerner Enviza, Paris, France. Global Health Economics and Outcomes Research, Sobi, Stockholm, Sweden. Patient Access, Sobi, Paris, France. Department of Pharmacy, Rouen University Hospital, Rouen, France. UNIROUEN, INSERM U1234, Pathophysiology, Autoimmunity, Neuromuscular Diseases and Regenerative THERapies, Normandie University, Rouen, France.

Publication: Hematology (Amsterdam, Netherlands). 2024. 29.

ABSTRACT: OBJECTIVES: 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. METHODS: This retrospective, observational study using the Système National des Données de Santé database, analyzed 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. RESULTS: 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®. CONCLUSION: 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.