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Thromb Haemost 2016; 116(S 01): S10-S17
DOI: 10.1160/TH16-01-0049
5th International Coagulation Meeting 2015 – Barcelona, Spain
Schattauer GmbH

The burden of inhibitors in haemophilia patients

Christopher E. Walsh
1   Division of Hematology and Medical Oncology, Mount Sinai School of Medicine, New York, New York, USA
,
Victor Jiménez-Yuste
2   Department of Haematology, Autónoma University Madrid and La Paz University Hospital, Madrid, Spain
,
Guenter Auerswald
3   Senior Advisor, Comprehensive Care Centre for Haemophilia and Thrombosis, Professor Hess Children’s Hospital, Klinikum Bremen-Mitte, Bremen, Germany
,
Salvador Grancha
4   Research and Development, Grifols S. A., Barcelona, Spain
› Author AffiliationsThe contributions of Maria Isabel Bravo and Montserrat Costa in the in vitro and in vivo studies evaluating the natural protection of FVIII by VWF against inhibitors are acknowledged. The authors also thank David P. Figgitt PhD, Content Ed Net, for providing editorial assistance in the preparation of the article, with funding from Grifols S. A.
Further Information

Publication History

Received: 20 January 2016

Accepted after major revision: 07 July 2016

Publication Date:
15 December 2017 (online)

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Summary

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.

Keywords

Haemophilia - FVIII - immune tolerance induction - inhibitor - von Willebrand factor
 

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