scholarly journals The effect of emicizumab prophylaxis on long‐term, self‐reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies

Haemophilia ◽  
2021 ◽  
Author(s):  
Mark W. Skinner ◽  
Claude Négrier ◽  
Ido Paz‐Priel ◽  
Sammy Chebon ◽  
Victor Jiménez‐Yuste ◽  
...  
Keyword(s):  
Physical Health ◽  
Factor Viii ◽  
Haemophilia A

scholarly journals Long‐term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A

Haemophilia ◽  
2015 ◽  
Vol 22 (1) ◽  
pp. 72-80 ◽  
Author(s):  
B. Nolan ◽  
J. Mahlangu ◽  
D. Perry ◽  
G. Young ◽  
R. Liesner ◽  
...  
Keyword(s):  
Fusion Protein ◽  
Factor Viii ◽  
Recombinant Factor Viii ◽  
Haemophilia A ◽  
Safety And Efficacy ◽  
Fc Fusion Protein

The long-term course of factor VIII inhibitors in patients with congenital haemophilia A without immune tolerance induction

2011 ◽  
Vol 105 (01) ◽  
pp. 59-65 ◽  
Author(s):  
Camila Caram ◽  
Roberta Grazielle de Souza ◽  
Júlio Carepa de Sousa ◽  
Tatiana Araújo Pereira ◽  
Ana Maria do Amaral Cerqueira ◽  
...  
Keyword(s):  
Confidence Interval ◽  
Factor Viii ◽  
Immune Tolerance ◽  
Tolerance Induction ◽  
Clotting Factor ◽  
Haemophilia A ◽  
Immune Tolerance Induction ◽  
Inhibitor Titre ◽  
Very High

SummaryThe development of alloantibodies that inhibit or neutralise the function of factor VIII is considered the most serious complication of the treatment of congenital haemophilia A. In order to describe their course without immune tolerance induction (ITI), we documented data on all performed inhibitor tests with dates as well as on clotting factor infusions of all consecutive patients who were treated in our centre between 1993 and 2006. Patients were tested every 7.1 months (95% confidence interval [CI], 6.6–7.8). A ‘sustained negative inhibitor status’ was defined as consistent non-positive inhibitor measurements for two years or longer. A total of 60/486 (12%) patients tested had a positive inhibitor titre in two or more occasions. Most of the patients (56%) with a maximum inhibitor titre of < 5 Bethesda unit (BU)/ml (named “low titre inhibitor”) developed a sustained negative inhibitor status. Among patients with high (5–9.9 BU/ml) and very high (≥ 10 BU/ ml) inhibitor titres, the proportions were 50% and 3%, respectively. Our findings suggest that ITI might not be needed for all patients with non-transient inhibitors, especially when their maximum inhibitor titre is below 10 BU/ml. Further studies in countries where ITI is not available are needed to examine predictors of the natural sustained negative inhibitor status.

Adult severe haemophilia A patients under long-term prophylaxis with factor VIII in routine clinical practice

2015 ◽  
Vol 26 (5) ◽  
pp. 509-514 ◽  
Author(s):  
María E. Mingot-Castellano ◽  
Laura González-Díaz ◽  
Rocio Tamayo-Bermejo ◽  
Anabel I. Heiniger-Mazo
Keyword(s):  
Clinical Practice ◽  
Factor Viii ◽  
Routine Clinical Practice ◽  
Haemophilia A ◽  
Severe Haemophilia

scholarly journals Successful immune tolerance in two children with severe haemophilia A

2013 ◽  
Vol 1 (1) ◽  
pp. 5-7
Author(s):  
Bert Leenders
Keyword(s):  
Factor Viii ◽  
Case Studies ◽  
Immune Tolerance ◽  
Bleeding Disorder ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Igg Antibodies

Abstract Haemophilia A is an X-linked congenital bleeding disorder resulting from a deficiency of factor VIII (FVIII). Therapy to prevent or treat bleeding is by replacement of the missing FVIII. However, as a consequence of treatment, patients with HA may develop inhibitory IgG antibodies to FVIII, termed “inhibitors”. When this occurs, treatment becomes more complex and costly and morbidity increases. Inhibitor formation, occurring in up to 36% of patients with severe HA, is currently one of the most significant complications affecting patients with HA [1,2]. In these two case studies, the management of bleeds in patients with inhibitors, and the long-term inhibitor eradication will be discussed.

scholarly journals Long-term efficacy and safety of a pasteurized, plasma-derived factor VIII concentrate (Beriate® P) in patients with haemophilia A

2014 ◽  
Vol 134 ◽  
pp. S38-S42 ◽  
Author(s):  
Robert Klamroth ◽  
Saskia Gottstein ◽  
Marija Orlovic ◽  
Christl Heinrichs
Keyword(s):  
Factor Viii ◽  
Haemophilia A ◽  
Efficacy And Safety ◽  
Term Efficacy
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