Incidence and risk factors for inhibitor development in previously untreated severe haemophilia A patients born between 2005 and 2010

Haemophilia ◽  
2014 ◽  
Vol 20 (6) ◽  
pp. 771-776 ◽  
Author(s):  
C. Vézina ◽  
M. Carcao ◽  
C. Infante-Rivard ◽  
D. Lillicrap ◽  
A. M. Stain ◽  
...  
Keyword(s):  
Risk Factors ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Inhibitor Development

Inhibitor development in haemophilia according to concentrate

2015 ◽  
Vol 113 (05) ◽  
pp. 968-975 ◽  
Author(s):  
Riita Lassila ◽  
Flora Peyvandi ◽  
Gabriele Calizzani ◽  
Alex Gatt ◽  
Thierry Lambert ◽  
...  
Keyword(s):  
Calculated Data ◽  
Incidence Rates ◽  
Clotting Factor ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Inhibitor Development ◽  
Haemophilia B ◽  
Number Of Patients ◽  
Recombinant Fviii ◽  
Set Up

SummaryInhibitor development represents the most serious side effect of haemophilia treatment. Any difference in risk of inhibitor formation depending on the product used might be of clinical relevance. It was this study’s objective to assess inhibitor development according to clotting factor concentrate in severe haemophilia A and B. The European Haemophilia Safety Surveillance (EUHASS) was set up as a study monitoring adverse events overall and according to concentrate. Since October 2008, inhibitors were reported at least quarterly. Number of treated patients was reported annually, specifying the number of patients completing 50 exposure days (Previously Untreated Patients, PUPs) without inhibitor development. Cumulative incidence, incidence rates and 95 % confidence intervals (CI) were calculated. Data from October 1, 2008 to December 31, 2012 were analysed for 68 centres that validated their data. Inhibitors developed in 108/417 (26 %; CI 22–30 %) PUPs with severe haemophilia A and 5/72 (7 %; CI 2–16%) PUPs with severe haemophilia B. For Previously Treated Patients (PTPs), 26 inhibitors developed in 17,667 treatment years [0.15/100 treatment years; CI 0.10–0.22) for severe haemophilia A and 1/2836 (0.04/100; (CI 0.00–0.20) for severe haemophilia B. Differences between plasma-derived and recombinant concentrates, or among the different recombinant FVIII concentrates were investigated. In conclusion, while confirming the expected rates of inhibitors in PUPs and PTPs, no class or brand related differences were observed.

scholarly journals Inhibitor development in previously untreated patients with severe haemophilia: A comparison of included patients and outcomes between a clinical study and a registry‐based study

Haemophilia ◽  
2020 ◽  
Vol 26 (5) ◽  
pp. 809-816
Author(s):  
Carla J. Jonker ◽  
Katrien Oude Rengerink ◽  
Arno W. Hoes ◽  
Peter G. M. Mol ◽  
H. Marijke Berg
Keyword(s):  
Clinical Study ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Inhibitor Development

Improved prediction of inhibitor development in previously untreated patients with severe haemophilia A

Haemophilia ◽  
2014 ◽  
Vol 21 (2) ◽  
pp. 227-233 ◽  
Author(s):  
S. M. Hashemi ◽  
K. Fischer ◽  
K. G. M. Moons ◽  
H. M. Berg ◽  
Keyword(s):  
Haemophilia A ◽  
Severe Haemophilia ◽  
Inhibitor Development

Influence of the type of F8 gene mutation on inhibitor development in a single centre cohort of severe haemophilia A patients

Haemophilia ◽  
2010 ◽  
Vol 17 (2) ◽  
pp. 275-281 ◽  
Author(s):  
S. C. GOUW ◽  
J. G. VAN DER BOM ◽  
H. M. VAN DEN BERG ◽  
R. A. ZEWALD ◽  
J. K. PLOOS VAN AMSTEL ◽  
...  
Keyword(s):  
Gene Mutation ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Single Centre ◽  
Inhibitor Development

Haemophilia and thrombophilia

2003 ◽  
Vol 23 (01) ◽  
pp. 36-40 ◽  
Author(s):  
C. Escuriola ◽  
K. Kurnik ◽  
R. Schobess ◽  
S. Horneff ◽  
A. Kosch ◽  
...  
Keyword(s):  
Risk Factors ◽  
Factor Viii ◽  
Protein C ◽  
Methylenetetrahydrofolate Reductase ◽  
Haemorrhagic Stroke ◽  
Type I ◽  
Haemophilia A ◽  
Factor V ◽  
Severe Haemophilia ◽  
Prothrombotic Risk Factors

SummaryFor the study presented here 135 pediatric PUP patients with haemophilia consecutively admitted to German pediatric haemophilia treatment centers were investigated. In addition to factor VIII activity, the factor V (FV) G1691A mutation, the factor II (FII) G20210A variant, methylenetetrahydrofolate reductase (MTHFR) T677T genotype, elevated lipoprotein a (Lp a), antithrombin, protein C, and protein S were investigated. 103 out of 122 HA patients (FVIII activity <1%) were suffering from severe HA. The prevalence of prothrombotic risk factors in children with severe haemophilia A (HA) did not differ from previously reported data: FV GA 5.8%, FII GA 3.9%, MTHFR TT 10%, elevated Lp a 7%, protein C type I deficiency 1.1%. The first symptomatic bleeding leading to diagnosis of severe haemophilia occurred with a median age of 1.6 years (range: 0.5-7.1 years) in children carrying prothrombotic risk factors compared to non-carriers (0.9 years (0.1-4.0; p = 0.01). Two patients presenting with neonatal stroke due to elevated Lp a and the FII GA variant showed haemorrhagic stroke transformation triggered by severe haemophilia. In addition, when haemophilia A was corrected by administration of factor VIII concentrates eight out of 25 children with central lines in place developed catheter-related thrombosis. Conclusion: The data of this multicentre cohort study demonstrate that the clinical phenotype of severe haemophilia A in childhood is clearly influenced by the coinheritance of prothrombotic risk factors.

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