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

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00843989%3A_____%2F25%3AE0112022" target="_blank" >RIV/00843989:_____/25:E0112022 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00216224:14110/25:00143362 RIV/00216208:11130/25:10505319 RIV/00216208:11140/25:10505319 RIV/61989592:15110/25:73632872 a 2 dalších

Výsledek na webu

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

Jazyk výsledku

angličtina

Název v původním jazyce

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

Popis výsledku v původním jazyce

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.

Název v anglickém jazyce

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

Popis výsledku anglicky

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.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30205 - Hematology

Projekt

—

Návaznosti

V - Vyzkumna aktivita podporovana z jinych verejnych zdroju

Rok uplatnění

2025

Kód důvěrnosti údajů

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

Název periodika

Research and Practice in Thrombosis and Haemostasis

ISSN

2475-0379

e-ISSN

—

Svazek periodika

9

Číslo periodika v rámci svazku

7

Stát vydavatele periodika

US - Spojené státy americké

Počet stran výsledku

8

Strana od-do

1-8

Kód UT WoS článku

001620334300002

EID výsledku v databázi Scopus

2-s2.0-105020926250