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Inhibitors in haemophilia A and B : Management of bleeds, inhibitor eradication, and strategies for difficult-to-treat patients

Ljung, Rolf LU ; Auerswald, Guenter; Benson, Gary; Dolan, Gerry; Duffy, Anne; Hermans, Cedric; Jiménez-Yuste, Victor; Lambert, Thierry; Morfini, Massimo and Zupančić-Šalek, Silva, et al. (2019) In European Journal of Haematology 102(2). p.111-122
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... (More)

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 tolerize 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 summarizes 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. This article is protected by copyright. All rights reserved.

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published
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European Journal of Haematology
volume
102
issue
2
pages
111 - 122
publisher
Wiley-Blackwell
external identifiers
  • scopus:85057991589
ISSN
1600-0609
DOI
10.1111/ejh.13193
language
English
LU publication?
yes
id
6c3f6be6-eecc-466f-b260-a9a997613e8c
date added to LUP
2018-11-22 10:07:51
date last changed
2019-02-20 11:37:09
@article{6c3f6be6-eecc-466f-b260-a9a997613e8c,
  abstract     = {<p>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 tolerize 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 summarizes 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. This article is protected by copyright. All rights reserved.</p>},
  author       = {Ljung, Rolf and Auerswald, Guenter and Benson, Gary and Dolan, Gerry and Duffy, Anne and Hermans, Cedric and Jiménez-Yuste, Victor and Lambert, Thierry and Morfini, Massimo and Zupančić-Šalek, Silva and Santagostino, Elena},
  issn         = {1600-0609},
  language     = {eng},
  number       = {2},
  pages        = {111--122},
  publisher    = {Wiley-Blackwell},
  series       = {European Journal of Haematology},
  title        = {Inhibitors in haemophilia A and B : Management of bleeds, inhibitor eradication, and strategies for difficult-to-treat patients},
  url          = {http://dx.doi.org/10.1111/ejh.13193},
  volume       = {102},
  year         = {2019},
}