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, 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.

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

• Originální 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
• WWW: 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

Anotace

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.