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

J 2025

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

ZÁPOTOCKÁ, Ester; Petra OVESNÁ; Bohumir BLAZEK; Zuzana CERMAKOVA; Zdenka HAJSMANOVA et al.

Základní údaje

Originální název

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

Autoři

ZÁPOTOCKÁ, Ester; Petra OVESNÁ ; Bohumir BLAZEK; Zuzana CERMAKOVA; Zdenka HAJSMANOVA; Radomira HRDLICKOVA; Petr BIRKE; Daniela PROCHAZKOVA; Petr SMEJKAL a Jan BLATNÝ

Vydání

RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS, AMSTERDAM, ELSEVIERRADARWEG, 2025, 2475-0379

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30205 Hematology

Stát vydavatele

Nizozemské království

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 3.400 v roce 2024

Označené pro přenos do RIV

Ano

Organizační jednotka

Lékařská fakulta

Klíčová slova anglicky

annual bleeding rate; consumption; emicizumab; haemophilia A; prophylaxis; zero bleeds

Štítky

14110321, 14110616

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 16. 1. 2026 12:34, Mgr. Tereza Miškechová

Anotace

V originále

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 < .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 < .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.

Citovat

ZÁPOTOCKÁ, Ester; Petra OVESNÁ; Bohumir BLAZEK; Zuzana CERMAKOVA; Zdenka HAJSMANOVA; Radomira HRDLICKOVA; Petr BIRKE; Daniela PROCHAZKOVA; Petr SMEJKAL a Jan BLATNÝ. Emicizumab prophylaxis in current haemophilia A care in the Czech Republic-data from the Czech National Haemophilia Programme Registry. RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS. AMSTERDAM: ELSEVIERRADARWEG, 2025, roč. 9, č. 7, s. 1-8. ISSN 2475-0379. Dostupné z: https://doi.org/10.1016/j.rpth.2025.103215.

@article{2547610,
   author = {Zápotocká, Ester and Ovesná, Petra and Blazek, Bohumir and Cermakova, Zuzana and Hajsmanova, Zdenka and Hrdlickova, Radomira and Birke, Petr and Prochazkova, Daniela and Smejkal, Petr and Blatný, Jan},
   article_location = {AMSTERDAM},
   article_number = {7},
   doi = {https://doi.org/10.1016/j.rpth.2025.103215},
   keywords = {annual bleeding rate; consumption; emicizumab; haemophilia A; prophylaxis; zero bleeds},
   language = {eng},
   issn = {2475-0379},
   journal = {RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS},
   title = {Emicizumab prophylaxis in current haemophilia A care in the Czech Republic-data from the Czech National Haemophilia Programme Registry},
   url = {https://www.rpthjournal.org/article/S2475-0379(25)00539-4/fulltext},
   volume = {9},
   year = {2025}
}

TY  - JOUR
ID  - 2547610
AU  - Zápotocká, Ester - Ovesná, Petra - Blazek, Bohumir - Cermakova, Zuzana - Hajsmanova, Zdenka - Hrdlickova, Radomira - Birke, Petr - Prochazkova, Daniela - Smejkal, Petr - Blatný, Jan
PY  - 2025
TI  - Emicizumab prophylaxis in current haemophilia A care in the Czech Republic-data from the Czech National Haemophilia Programme Registry
JF  - RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS
VL  - 9
IS  - 7
SP  - 1-8
EP  - 1-8
PB  - ELSEVIERRADARWEG
SN  - 24750379
KW  - annual bleeding rate
KW  - consumption
KW  - emicizumab
KW  - haemophilia A
KW  - prophylaxis
KW  - zero bleeds
UR  - https://www.rpthjournal.org/article/S2475-0379(25)00539-4/fulltext
N2  - 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 < .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 < .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.
ER  -

ZÁPOTOCKÁ, Ester; Petra OVESNÁ; Bohumir BLAZEK; Zuzana CERMAKOVA; Zdenka HAJSMANOVA; Radomira HRDLICKOVA; Petr BIRKE; Daniela PROCHAZKOVA; Petr SMEJKAL a Jan BLATNÝ. Emicizumab prophylaxis in current haemophilia A care in the Czech Republic-data from the Czech National Haemophilia Programme Registry. \textit{RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS}. AMSTERDAM: ELSEVIERRADARWEG, 2025, roč.~9, č.~7, s.~1-8. ISSN~2475-0379. Dostupné z: https://doi.org/10.1016/j.rpth.2025.103215.

Přehled o publikaci