rFVIIIFc in treatment of haemophilia A from perspective of real clinical practice in three centers for treatment of haemophilia in the Czech Republic

Abstract

Introduction: In haemophilia A, prophylaxis with factor VIII with extended biological half-life (EHL) are gradually promoted over factors with standard half-life (SHL). One of representatives of EHL is efmoroctocog alfa - rFVIIIFc.

Objective: Retrospective evaluation of transition from SHL to rFVIIIFc treatment in conditions of routine clinical practice of three comprehensive haemophilia centers in the Czech Republic.

Methods: Retrospective analysis of medical records of patients with severe haemophilia A in two age groups (<12 and ≥12 years) previously treated with prophylactic SHL who were switched to rFVIIIFc. We evaluated bleeding control, injection frequency, factor consumption and trough levels achieved.

Results: 16 patients with average age of 6.1 years were included in the group <12 years, and 8 patients with an average age of 37.8 years were included in the  group ≥12 years. Group < 12 years: Compared with SHL, treatment with rFVIIIFc resulted in statistically significant reduction in the mean annual ABR bleeding rate (2.69 ± 2.24 vs. 0.69 ± 1.02; p= 0.0015) and the number of weekly applications ( 3 ± 0.45 vs. 2.21 ± 0.29; p= 0.0008). Proportion of patients without bleeding increased from 12.5% ​​to 62.5%. Factor consumption was similar and differences in mean trough levels (%) were not statistically significant (1.37 ± 0.85 vs. 1.66 ± 0.79). Group ≥ 12 years: Compared to SHL, treatment with rFVIIIFc led to statistically significant decrease in mean number of applications (2.62 ± 0.62 vs. 1.88 ± 0.13; p= 0.0223) and increase in mean trough levels (1 .75 ± 1.01 vs. 2.7 ± 1.28; p= 0.0223) with same factor consumption. Decrease in ABR was not statistically significant (mean 2.38 ± 1.51 vs. 1.13 ± 1.81; p= 0.0707). Proportion of patients without bleeding was 12.5% ​​for SHL and 62.5% for rFVIIIFc.

Conclusion: Switch from SHL treatment to rFVIIIFc was associated with better bleeding control and reduced application number regardless of age, while factor consumption remained unchanged. Benefits were particularly pronounced in children.