Emicizumab prophylaxis in current haemophilia A care in the Czech Republic—data from the Czech National Haemophilia Programme Registry

Result code in IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00098892%3A_____%2F25%3A10159542" target="_blank" >RIV/00098892:_____/25:10159542 - isvavai.cz</a>

Result on the web

<a href="https://www.sciencedirect.com/science/article/pii/S2475037925005394?via%3Dihub" target="_blank" >https://www.sciencedirect.com/science/article/pii/S2475037925005394?via%3Dihub</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1016/j.rpth.2025.103215" target="_blank" >10.1016/j.rpth.2025.103215</a>

Result language

angličtina

Original language name

Emicizumab prophylaxis in current haemophilia A care in the Czech Republic—data from the Czech National Haemophilia Programme Registry

Original language description

Background: Nonfactor therapy with emicizumab has become an important part of the haemophilia A treatment landscape recently. Objectives: We aimed to analyze data on the transition from previous treatment regimens to emicizumab in routine clinical practice in the Czech Republic. Methods: Retrospective analysis of data from the Czech National Haemophilia Programme (CNHP) registry of all consecutive pediatric and adult persons treated with emicizumab prophylaxis. We evaluated bleeding control, injection frequency, and drug consumption in the period before and after emicizumab initiation. Results: Seventy-three persons with severe haemophilia A, median (IQR) age 4 (1-18) years, treated with emicizumab were reported in the CNHP registry up to the end of 2023. Two age categories were used for further stratification, 0-11 (n = 45) and 12+ (n = 28) years. For the whole group, we observed a significant reduction in total annualized bleeding rate (ABR) from a mean of 3.44 to 0.46 (P &lt; .001), in joint ABR from 0.70 to 0.27 (P = .004), and in spontaneous ABR from 1.18 to 0.19 (P = .002) after starting emicizumab. The proportion of persons with no reported bleeding increased from 26.0% to 60.3% (P &lt; .001). A sub-analysis, focused on inhibitors presence (present in 27 cases) and age, showed a more significant effect of emicizumab switch in observed parameters in persons with inhibitors and in the younger age group. Conclusion: The retrospective analysis of nation-wide data from the CNHP registry supports the fact that emicizumab has the potential to reduce bleeding and to provide an opportunity for a significant proportion of patients, with and without factor VIII inhibitors, to achieve zero bleeds.

Czech name

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Czech description

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Type

J_imp - Article in a specialist periodical, which is included in the Web of Science database

CEP classification

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OECD FORD branch

30209 - Paediatrics

Project

—

Continuities

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Publication year

2025

Confidentiality

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Name of the periodical

Research and Practice in Thrombosis and Haemostasis

ISSN

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e-ISSN

2475-0379

Volume of the periodical

9

Issue of the periodical within the volume

7

Country of publishing house

NL - THE KINGDOM OF THE NETHERLANDS

Number of pages

8

Pages from-to

103215

UT code for WoS article

001620334300002

EID of the result in the Scopus database

2-s2.0-105020926250