J 2021

Incidence of inhibitor development in PUPs with severe Haemophilia A in the CEE region between 2005 and 2015

BLATNÝ, Jan, Maria KARDOS, Predrag MILJIC, Ernest BILIC, Majda BENEDIK-DOLNICAR et. al.

Basic information

Original name

Incidence of inhibitor development in PUPs with severe Haemophilia A in the CEE region between 2005 and 2015

Authors

BLATNÝ, Jan (203 Czech Republic, guarantor, belonging to the institution), Maria KARDOS, Predrag MILJIC, Ernest BILIC, Majda BENEDIK-DOLNICAR, Barbara FAGANEL-KOTNIK, Dobrin KONSTANTINOV, Zhanna KOVALOVA and Petra OVESNÁ (203 Czech Republic, belonging to the institution)

Edition

Thrombosis Research, OXFORD, Pergamon-Elsevier Science, 2021, 0049-3848

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30205 Hematology

Country of publisher

United Kingdom of Great Britain and Northern Ireland

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

Impact factor

Impact factor: 10.407

RIV identification code

RIV/00216224:14110/21:00121759

Organization unit

Faculty of Medicine

DOI

UT WoS

000616053200031

Keywords in English

Haemophilia A; Inhibitors; CEE; Cohort studies; Risk factors; PUPs

Tags

Tags

International impact, Reviewed
Změněno: 14/6/2021 08:01, Mgr. Tereza Miškechová

Abstract

V originále

Introduction: This study analyses real-world data on 144 previously untreated patients (PUPs) with severe Haemophilia A, from seven countries in Central and Eastern Europe (CEE: Bulgaria, Croatia, Czech Republic, Hungary, Latvia, Serbia, and Slovenia), over a period of 11 years. It analyses the risk factors associated with development of inhibitors to factor VIII concentrates. Methods: Cox proportional hazard models were used to estimate the hazard risk of factors possibly influencing the development of inhibitors. Patients were followed for up to 100 exposure days (EDs). Results: Cumulative inhibitor incidence at the time of 100 EDs was 18.7%, slightly lower than the 25-35% incidence reported in most studies. Of PUPs who developed inhibitors, a majority (56%) developed them within the first 20 EDs and 88% by the 50th ED. FVIII class (recombinant or plasma-derived) did not influence the inhibitors' incidence rate (p = 0.64). We found a significant protective effect of prophylaxis compared to on demand treatment (p = 0.003). PUPs who had an intensive peak treatment during the first 50 EDs were at significantly higher risk for inhibitor development (HR (95% CI) 5.3 (2.3-12.5), p < 0.001). Conclusion: Inhibitors are and will continue to be the most significant complication of haemophilia treatment with factor concentrates. This is particularly true for haemophilia A. In our cohort, we were able to show that the treatment regimen used during first 50EDs influenced significantly the inhibitor risk, but the class of the factor concentrate did not play an important role. Real world data will remain one of the important resources for improving our knowledge of haemophilia.