Inhibitors in haemophilia A and B: Management of bleeds, inhibitor eradication and strategies for difficult‐to‐treat patients
Entity
UAM. Departamento de Medicina; Instituto de Investigación Sanitaria Hospital Universitario de La Paz (IdiPAZ)Publisher
John Wiley & Sons Ltd.Date
2018Citation
10.1111/ejh.13193
European Journal of Haematology 102 (2019): 111-122
ISSN
0902-4441 (print); 1600-0609 (online)DOI
10.1111/ejh.13193Funded by
Supported by a grant from Novo Nordisk Health Care AG, in compliance with international guidelines for good publication practice.Editor's Version
https://doi.org/10.10.1111/ejh.13193Subjects
Coagulation disorders; Paediatric haematology; Quality of life; MedicinaRights
© 2018 The Author(s)Abstract
The standard therapy for patients with haemophilia is prophylactic treatment with
replacement factor VIII (FVIII) or factor IX (FIX). Patients who develop inhibitors
against FVIII/FIX face an increased risk of bleeding, and the likelihood of early development
of progressive arthropathy, alongside higher treatment‐related costs.
Bypassing agents can be used to prevent and control bleeding, as well as the recently
licensed prophylaxis, emicizumab, but their efficacy is less predictable than that of
factor replacement therapy. Antibody eradication, by way of immune tolerance induction
(ITI), is still the preferred management strategy for treating patients with inhibitors.
This approach is successful in most patients, but some are difficult to tolerise
and/or are unresponsive to ITI, and they represent the most complicated patients to
treat. However, there are limited clinical data and guidelines available to help guide
physicians in formulating the next treatment steps in these patients. This review summarises
currently available treatment options for patients with inhibitors, focussing
on ITI regimens and those ITI strategies that may be used in difficult‐to‐treat patients.
Some alternative, non‐ITI approaches for inhibitor management, are also proposed
Files in this item
Google Scholar:Ljung, Rolf
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Auerswald, Guenter
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Benson, Gary
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Dolan, Gerry
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Duffy, Anne
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Hermans, Cedric
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Jiménez Yuste, Víctor Manuel
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Lambert, Thierry
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Morfini, Massimo
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Zupančić‐Šalek, Silva
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Santagostino, Elena
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