rFVIIIFc v léčbě hemofilie A z pohledu reálné klinické praxe tří center pro léčbu hemofilie v České republice
| Title in English | rFVIIIFc in treatment of haemophilia A from perspective of real clinical practise in three centres for treatment of heamophilia in the Czech Republic |
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| Authors | |
| Year of publication | 2023 |
| Type | Article in Periodical |
| Magazine / Source | Transfuze a Hematologie dnes |
| MU Faculty or unit | |
| Citation | |
| Web | https://redakce.carecomm.cz/tahd/article/view/1495/613 |
| Doi | http://dx.doi.org/10.48095/cctahd2023prolekare.cz6 |
| Keywords | haemophilia A; biological half-life; SHL; rFVIIIc; bleeding control; application number |
| Description | Introduction: In haemophilia A, prophylaxis using factor VIII preparations with an extended bio logical half-life (EHL) is gradually gaining ground over standard half-life factors (SHL). One of representative of EHL is efmoroctocog alfa (rFVIIIFc). Objective: Retrospective evaluation of the transition from SHL to rFVIIIFc treatment in the routine clinical practice at three complex haemophilia centres 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 attained trough levels. Results: 16 patients with an 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 a 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). The 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 a statistically significant decrease in the mean number of applications (2.62 ± 0.62 vs. 1.88 ± 0.13; P = 0.0223) and an increase in mean trough levels (1.75 ± 1.01 vs. 2.7 ± 1.28; P = 0.0223) with the same factor consumption. The decrease in ABR was not statistically significant (mean 2.38 ± 1.51 vs. 1.13 ± 1.81; P = 0.0707). The proportion of patients without bleeding was 12.5% for SHL and 62.5% for rFVIIIFc. Conclusion: The 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 important in children. |
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