Prophylactic Efmoroctocog Alfa may improve outcomes in patients with hemophilia A: Study

A recent retrospective study published in the journal of Hematology explored the clinical characteristics and treatment patterns of patients with hemophilia A who were undergoing prophylactic treatment. This comprehensive study utilized data from the Systeme National des Donnees de Sante database which spanned from 2016 to 2019 to highlight the factors such as factor consumption and events of interest in this patient population. This study also evaluated the clinical impact of transitioning these patients to Elocta® which is an extended half-life treatment for hemophilia A.

Among the 545 patients included in the analysis, Elocta® emerged as the most frequently utilized treatment option. The bleeding events affected 15.4% of patients, while 13.9% experienced articular non-bleeding events leading to hospitalization. The surgeries or procedures related to hemophilic arthropathy were reported in 9.9% of cases. The outcomes revealed a mean monthly factor VIII product consumption of 344 IU/kg/month for extended half-life treatment and 331 IU/kg/month for standard half-life products.

The sub-cohort analysis involved a total of 146 patients who transitioned to Elocta® observed promising trends. Although bleeding events did not significantly decrease post-switch, there was a marked reduction in their frequency. Also, bleeding events decreased from a mean of 0.32 events per patient per year to 0.09 events post-switch. Importantly, this reduction did not reach statistical significance. There were no significant differences in factor consumption rates or the occurrence of articular non-bleeding events before and after the initiation of Elocta®. Overall, this study provides valuable insights into the treatment landscape and clinical outcomes for the patients with hemophilia A who are receiving prophylactic regimens in France. 

Source: 

Trossaërt, M., Falk, A., Gautier, L., Kragh, N., Van Hinloopen, O., & Varin, R. (2024). An observational study of haemophilia A patients without inhibitors using the French national claims (SNDS) database. In Hematology (Vol. 29, Issue 1). Informa UK Limited. https://doi.org/10.1080/16078454.2024.2320610