Literature DB >> 27528280

The burden of inhibitors in haemophilia patients.

Christopher E Walsh, Víctor Jiménez-Yuste1, Guenter Auerswald, Salvador Grancha.   

Abstract

The burden of disease in haemophilia patients has wide ranging implications for the family and to society. There is evidence that having a current inhibitor increases the risk of morbidity and mortality. Morbidity is increased by the inability to treat adequately and its consequent disabilities, which then equates to a poor quality of life compared with non-inhibitor patients. The societal cost of care, or `burden of inhibitors', increases with the ongoing presence of an inhibitor. Therefore, it is clear that successful eradication of inhibitors by immune tolerance induction (ITI) is the single most important milestone one can achieve in an inhibitor patient. The type of factor VIII (FVIII) product used in ITI regimens varies worldwide. Despite ongoing debate, there is in vitro and retrospective clinical evidence to support the use of plasma-derived VWF-containing FVIII concentrates in ITI regimens in order to achieve early and high inhibitor eradication success rates.

Entities:  

Keywords:  FVIII; Haemophilia; immune tolerance induction; inhibitor; von Willebrand factor

Mesh:

Substances:

Year:  2016        PMID: 27528280     DOI: 10.1160/TH16-01-0049

Source DB:  PubMed          Journal:  Thromb Haemost        ISSN: 0340-6245            Impact factor:   5.249


  16 in total

Review 1.  Gene therapy for hemophilia: what does the future hold?

Authors:  Bhavya S Doshi; Valder R Arruda
Journal:  Ther Adv Hematol       Date:  2018-08-27

2.  Induction of activated T follicular helper cells is critical for anti-FVIII inhibitor development in hemophilia A mice.

Authors:  Weiqing Jing; Juan Chen; Yuanhua Cai; Yingyu Chen; Jocelyn A Schroeder; Bryon D Johnson; Weiguo Cui; Qizhen Shi
Journal:  Blood Adv       Date:  2019-10-22

Review 3.  Novel alternate hemostatic agents for patients with inhibitors: beyond bypass therapy.

Authors:  Margaret V Ragni
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2017-12-08

4.  Gene Delivery of Activated Factor VII Using Alternative Adeno-Associated Virus Serotype Improves Hemostasis in Hemophiliac Mice with FVIII Inhibitors and Adeno-Associated Virus Neutralizing Antibodies.

Authors:  Junjiang Sun; Baolai Hua; Xiaojing Chen; Richard J Samulski; Chengwen Li
Journal:  Hum Gene Ther       Date:  2017-05-05       Impact factor: 5.695

5.  Impact of the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET) study and its post hoc analyses on clinical practice in the United States: A survey of Haemophilia and Thrombosis Research Society members.

Authors:  Christopher M Sande; Ahmad Al-Huniti; Patrick Ten Eyck; Anjali A Sharathkumar
Journal:  Haemophilia       Date:  2019-07-02       Impact factor: 4.287

Review 6.  Hemophilia Care in the Pediatric Age.

Authors:  Marta Bertamino; Francesca Riccardi; Laura Banov; Johanna Svahn; Angelo Claudio Molinari
Journal:  J Clin Med       Date:  2017-05-19       Impact factor: 4.241

7.  Health-related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non-interventional study (NIS).

Authors:  Johnny Mahlangu; Johannes Oldenburg; Michael U Callaghan; Midori Shima; Maria Elisa Mancuso; Peter Trask; Michael Recht; Claudia Garcia; Renchi Yang; Michaela Lehle; Harrison Macharia; Elina Asikanius; Gallia G Levy; Rebecca Kruse-Jarres; Sylvia von Mackensen
Journal:  Haemophilia       Date:  2019-04-24       Impact factor: 4.287

Review 8.  Tolerance to FVIII: Role of the Immune Metabolic Enzymes Indoleamine 2,3 Dyoxigenase-1 and Heme Oxygenase-1.

Authors:  Davide Matino; Sajjad Afraz; George Zhao; Paul Tieu; Marco Gargaro; Francesca Fallarino; Alfonso Iorio
Journal:  Front Immunol       Date:  2020-04-15       Impact factor: 7.561

9.  The severe spontaneous bleeding phenotype in a novel hemophilia A rat model is rescued by platelet FVIII expression.

Authors:  Qizhen Shi; Jeremy G Mattson; Scot A Fahs; Aron M Geurts; Hartmut Weiler; Robert R Montgomery
Journal:  Blood Adv       Date:  2020-01-14

10.  Predicting the Development of Anti-Drug Antibodies against Recombinant alpha-Galactosidase A in Male Patients with Classical Fabry Disease.

Authors:  Sanne J van der Veen; Wytze J Vlietstra; Laura van Dussen; André B P van Kuilenburg; Marcel G W Dijkgraaf; Malte Lenders; Eva Brand; Christoph Wanner; Derralynn Hughes; Perry M Elliott; Carla E M Hollak; Mirjam Langeveld
Journal:  Int J Mol Sci       Date:  2020-08-12       Impact factor: 5.923

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