J 2023

Bleeding pattern and consumption of factor VIII concentrate in adult patients with haemophilia A without inhibitors in the Czech Republic between 2013 and 2021 (Czech National Haemophilia Programme registry data)

ROMANOVÁ, Gabriela, Petr SMEJKAL, Petra OVESNÁ, Eva DRBOHLAVOVA, Petr DULICEK et. al.

Základní údaje

Originální název

Bleeding pattern and consumption of factor VIII concentrate in adult patients with haemophilia A without inhibitors in the Czech Republic between 2013 and 2021 (Czech National Haemophilia Programme registry data)

Autoři

ROMANOVÁ, Gabriela (203 Česká republika, domácí), Petr SMEJKAL (203 Česká republika, domácí), Petra OVESNÁ (203 Česká republika), Eva DRBOHLAVOVA (203 Česká republika), Petr DULICEK (203 Česká republika), Zdenka HAJSMANOVA (203 Česká republika), Antonin HLUSI (203 Česká republika), Radka HRDLICKOVA (203 Česká republika), Jana ULLRYCHOVA (203 Česká republika), Ivan VONKE (203 Česká republika), Jan BLATNÝ (203 Česká republika, domácí), Zuzana CERMAKOVA (203 Česká republika), Ester ZAPOTOCKA (203 Česká republika) a Miroslav PENKA (203 Česká republika, domácí)

Vydání

Annals of hematology, New York, Springer Verlag, 2023, 0939-5555

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30205 Hematology

Stát vydavatele

Spojené státy

Utajení

není předmětem státního či obchodního tajemství

Odkazy

Impakt faktor

Impact factor: 3.500 v roce 2022

Kód RIV

RIV/00216224:14110/23:00132991

Organizační jednotka

Lékařská fakulta

UT WoS

001071705000001

Klíčová slova anglicky

Haemophilia; Bleeding; Prophylaxis; On-demand; Consumption of factor VIII/IX concentrate; CNHP (the Czech National Haemophilia Programme); EHL (extended half-life)

Příznaky

Mezinárodní význam, Recenzováno
Změněno: 12. 1. 2024 14:45, Mgr. Tereza Miškechová

Anotace

V originále

The manuscript provides an overview of treatment and its changes in adult patients with haemophilia A without inhibitors in the Czech Republic between 2013 and 2021 using data from the registry of the Czech National Haemophilia Programme (CNHP). Over a 9-year period, we focused on the reduction in the annual bleeding rate (ABR), joint bleeding rate (AJBR) and factor VIII consumption when patients with severe haemophilia A switched from on-demand treatment to prophylaxis. The ABR and AJBR include both patient-reported home treatment and treated hospitalisation episodes. All adult patients with severe haemophilia A were categorised into three groups according to the therapeutic regimen. The first group was patients on prophylaxis during the follow-up period, the second group consisted of patients on on-demand treatment, and the third group was patients who received both treatment regimens during follow-up. With an increase in the proportion of patients with severe haemophilia A on prophylaxis from 37 to 74% between 2013 and 2021, the ABR for all patients with severe haemophilia A decreased approximately 6.9-fold, and the AJBR decreased 8.7-fold. Expectedly, the factor consumption increased by approximately 68.5%. In the group of patients with severe haemophilia A who had switched from an on-demand to a prophylactic regimen, the total number of bleeding events decreased 3.5-fold, and the number of joint bleeding episodes decreased 3.9-fold. Factor VIII consumption increased by 78.4%. Our study supports a previously reported positive effect of prophylaxis on bleeding control. We believe that the substantial improvement in ABR justifies the increased treatment costs.