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

Research output: Contribution to journalReview article

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 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.

Details

Authors
  • Rolf Ljung
  • Guenter Auerswald
  • Gary Benson
  • Gerry Dolan
  • Anne Duffy
  • Cedric Hermans
  • Victor Jiménez-Yuste
  • Thierry Lambert
  • Massimo Morfini
  • Silva Zupančić-Šalek
  • Elena Santagostino
Organisations
External organisations
  • Klinikum Bremen-Mitte
  • Belfast City Hospital
  • St Thomas' Hospital
  • Irish Haemophilia Society
  • Saint-Luc University Hospital
  • Autonomous University of Madrid
  • Bicêtre Hospital
  • Italian Association of Haemophilia Centres
  • Maggiore Hospital Policlinico
  • University of Osijek
  • University of Zagreb
  • Skåne University Hospital
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Hematology
Original languageEnglish
Pages (from-to)111-122
JournalEuropean Journal of Haematology
Volume102
Issue number2
Early online date2018 Nov 8
Publication statusPublished - 2019 Feb
Publication categoryResearch
Peer-reviewedYes