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Haemophilia. 2019 Jul;25(4):676-684. doi: 10.1111/hae.13762. Epub 2019 Apr 29.

The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab.

Author information

1
Division of Haematology/Oncology, Department of Paediatrics and Child Health Evaluative Sciences, Research Institute, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
2
Haemophilia Centre Rhine Main, Möerfelden-Walldorf, Germany.
3
Maggiore Hospital Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.
4
Institute of Experimental Haematology and Transfusion Medicine, University of Bonn, Bonn, Germany.
5
Haemophilia Centre, Great Ormond Street Hospital for Children, NHS Trust Haemophilia Centre, London, UK.
6
Our Lady's Children's Hospital Crumlin, Dublin, Ireland.
7
Department of Haematology and Transfusion Medicine and National Haemophilia Centre, University Hospital, Comenius University, Bratislava, Slovakia.
8
Unit for Congenital Bleeding Disorders, Hospital Universitario y Politécnico La Fe, Valencia, Spain.
9
Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, California.

Abstract

INTRODUCTION:

As a result of the new treatment paradigm that the haemophilia community will face with the availability of novel (non-factor) therapies, an updated consensus on ITI recommendations and inhibitor management strategies is needed.

AIM:

The Future of Immunotolerance Treatment (FIT) group was established to contemplate, determine and recommend the best management options for patients with haemophilia A and inhibitors.

DISCUSSION AND CONCLUSIONS:

Despite the considerable success of emicizumab in the management of inhibitor patients, the FIT group still sees the importance of eradicating inhibitors. However, the availability of emicizumab and other non-factor therapies in the future might impact greatly on how ITI is undertaken. Theoretically, concomitant use of emicizumab and FVIII might allow emicizumab to effectively prevent bleeding with lower dose ITI regimens. This might allow for the greater adoption of low-dose/low-frequency FVIII ITI regimens, which may result in a reduced need for central venous access devices while still maintaining a reasonable likelihood of ITI success. The FIT group proposes a new management algorithm for current ITI (without emicizumab) and a hypothetical new approach with the availability of emicizumab. As there are no published data regarding the concomitant use of emicizumab and FVIII for ITI, the FIT Expert group encourages the undertaking of properly conducted prospective studies to explore these approaches further.

KEYWORDS:

bypassing agents; emicizumab; factor VIII; haemophilia A; immune tolerance induction; inhibitor

PMID:
31033112
DOI:
10.1111/hae.13762

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