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

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.

Základní údaje

Originální název

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

Autoři

BLATNÝ, Jan (203 Česká republika, garant, domácí), Maria KARDOS, Predrag MILJIC, Ernest BILIC, Majda BENEDIK-DOLNICAR, Barbara FAGANEL-KOTNIK, Dobrin KONSTANTINOV, Zhanna KOVALOVA a Petra OVESNÁ (203 Česká republika, domácí)

Vydání

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

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30205 Hematology

Stát vydavatele

Velká Británie a Severní Irsko

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 10.407

Kód RIV

RIV/00216224:14110/21:00121759

Organizační jednotka

Lékařská fakulta

DOI

http://dx.doi.org/10.1016/j.thromres.2020.12.004

UT WoS

000616053200031

Klíčová slova anglicky

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

Štítky

14110321, 14119612, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 14. 6. 2021 08:01, Mgr. Tereza Miškechová

Anotace

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.

Citovat

BLATNÝ, Jan, Maria KARDOS, Predrag MILJIC, Ernest BILIC, Majda BENEDIK-DOLNICAR, Barbara FAGANEL-KOTNIK, Dobrin KONSTANTINOV, Zhanna KOVALOVA a Petra OVESNÁ. Incidence of inhibitor development in PUPs with severe Haemophilia A in the CEE region between 2005 and 2015. Thrombosis Research. OXFORD: Pergamon-Elsevier Science, 2021, roč. 198, FEB 2021, s. 196-203. ISSN 0049-3848. Dostupné z: https://dx.doi.org/10.1016/j.thromres.2020.12.004.

@article{1775937,
   author = {Blatný, Jan and Kardos, Maria and Miljic, Predrag and Bilic, Ernest and BenedikandDolnicar, Majda and FaganelandKotnik, Barbara and Konstantinov, Dobrin and Kovalova, Zhanna and Ovesná, Petra},
   article_location = {OXFORD},
   article_number = {FEB 2021},
   doi = {http://dx.doi.org/10.1016/j.thromres.2020.12.004},
   keywords = {Haemophilia A; Inhibitors; CEE; Cohort studies; Risk factors; PUPs},
   language = {eng},
   issn = {0049-3848},
   journal = {Thrombosis Research},
   title = {Incidence of inhibitor development in PUPs with severe Haemophilia A in the CEE region between 2005 and 2015},
   url = {https://www.sciencedirect.com/science/article/pii/S0049384820306514?via%3Dihub},
   volume = {198},
   year = {2021}
}

TY  - JOUR
ID  - 1775937
AU  - Blatný, Jan - Kardos, Maria - Miljic, Predrag - Bilic, Ernest - Benedik-Dolnicar, Majda - Faganel-Kotnik, Barbara - Konstantinov, Dobrin - Kovalova, Zhanna - Ovesná, Petra
PY  - 2021
TI  - Incidence of inhibitor development in PUPs with severe Haemophilia A in the CEE region between 2005 and 2015
JF  - Thrombosis Research
VL  - 198
IS  - FEB 2021
SP  - 196-203
EP  - 196-203
PB  - Pergamon-Elsevier Science
SN  - 00493848
KW  - Haemophilia A
KW  - Inhibitors
KW  - CEE
KW  - Cohort studies
KW  - Risk factors
KW  - PUPs
UR  - https://www.sciencedirect.com/science/article/pii/S0049384820306514?via%3Dihub
N2  - 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.
ER  -

BLATNÝ, Jan, Maria KARDOS, Predrag MILJIC, Ernest BILIC, Majda BENEDIK-DOLNICAR, Barbara FAGANEL-KOTNIK, Dobrin KONSTANTINOV, Zhanna KOVALOVA a Petra OVESNÁ. Incidence of inhibitor development in PUPs with severe Haemophilia A in the CEE region between 2005 and 2015. \textit{Thrombosis Research}. OXFORD: Pergamon-Elsevier Science, 2021, roč.~198, FEB 2021, s.~196-203. ISSN~0049-3848. Dostupné z: https://dx.doi.org/10.1016/j.thromres.2020.12.004.

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