Researchers reported that both emicizumab and Fc fusion protein (rFVIIIFc) recorded lower bleeding rates compared to prior factor VIII (FVIII) therapies for patients with hemophilia A, as presented at the 63rd American Society for Hematology Annual Meeting & Exposition.
Hemophilia A is a bleeding disorder characterized by FVIII deficiency. Patients with hemophilia A suffer from bleeding episodes that either occur spontaneously or following trauma, surgery, or injury.
Cockerham et al wanted to investigate the efficacy of other forms of therapy besides FVIII replacement therapy in treating hemophilia A. They used specialty pharmacy data to conduct a retrospective observational descriptive study between November 2014 and January 2021. The database contained over 5000 patients with hemophilia A in the US.
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The researchers defined the index date as the first dispense of emicizumab after October 2018 or the first dispense of rFVIIIFc after May 2015.
“Patient reported and claims-based treatment outcomes assessed pre- and post-treatment included: annualized bleeding rate (ABR, calculated from patient bleed logs), any factor usage including on-demand FVIII use, prescribed and dispensed dosage of treatment, and frequency,” they wrote.
In total, the researchers included in their analysis 118 patients treated with emicizumab and 55 patients treated with rFVIIIFc. The results demonstrated that the mean change in overall ABR pre- and post-treatment was -0.91 in the emicizumab group and -1.14 in the rFVIIIFc group.
Mean changes in spontaneous ABR and spontaneous joint ABR were -0.40 in the pretreatment emicizumab group and -0.37 in the post-treatment cohort. As for the rFVIIIFc group, the mean changes in spontaneous ABR and spontaneous joint ABR were -0.36 in the pretreatment group and -0.16 in the post-treatment group.
“This descriptive analysis indicated that both treatments remained effective with lower bleed rates than prior FVIII therapies,” Cockerham et al concluded.
Reference
Cockerham A, Wilson A, Frick A, Dasmahapatra P. A retrospective observational descriptive study on the effectiveness and usage of emicizumab and antihemophilic factor (recombinant), Fc fusion protein in patients with hemophilia A in the US. Poster presented at: 63rd American Society for Hematology Annual Meeting & Exposition: December 12, 2021; Atlanta, Georgia.