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

Rolf Ljung, Guenter Auerswald, Gary Benson, Gerry Dolan, Anne Duffy, Cedric Hermans, Victor Jiménez-Yuste, Thierry Lambert, Massimo Morfini, Silva Zupančić-Šalek, Elena Santagostino

Research output: Contribution to journalArticle

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.

Original languageEnglish
Pages (from-to)111-122
JournalEuropean Journal of Haematology
Volume102
Issue number2
DOIs
Publication statusPublished - 2019

Keywords

  • coagulation disorders
  • paediatric haematology
  • quality of life

ASJC Scopus subject areas

  • Hematology

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    Ljung, R., Auerswald, G., Benson, G., Dolan, G., Duffy, A., Hermans, C., Jiménez-Yuste, V., Lambert, T., Morfini, M., Zupančić-Šalek, S., & Santagostino, E. (2019). Inhibitors in haemophilia A and B: Management of bleeds, inhibitor eradication and strategies for difficult-to-treat patients. European Journal of Haematology, 102(2), 111-122. https://doi.org/10.1111/ejh.13193