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

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...

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Autores principales: Carcao, Manuel, Escuriola‐Ettingshausen, Carmen, Santagostino, Elena, Oldenburg, Johannes, Liesner, Ri, Nolan, Beatrice, Bátorová, Angelika, Haya, Saturnino, Young, Guy
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6850066/
https://www.ncbi.nlm.nih.gov/pubmed/31033112
http://dx.doi.org/10.1111/hae.13762
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author Carcao, Manuel
Escuriola‐Ettingshausen, Carmen
Santagostino, Elena
Oldenburg, Johannes
Liesner, Ri
Nolan, Beatrice
Bátorová, Angelika
Haya, Saturnino
Young, Guy
author_facet Carcao, Manuel
Escuriola‐Ettingshausen, Carmen
Santagostino, Elena
Oldenburg, Johannes
Liesner, Ri
Nolan, Beatrice
Bátorová, Angelika
Haya, Saturnino
Young, Guy
author_sort Carcao, Manuel
collection PubMed
description 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.
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spelling pubmed-68500662019-11-15 The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab Carcao, Manuel Escuriola‐Ettingshausen, Carmen Santagostino, Elena Oldenburg, Johannes Liesner, Ri Nolan, Beatrice Bátorová, Angelika Haya, Saturnino Young, Guy Haemophilia Original Articles 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. John Wiley and Sons Inc. 2019-04-29 2019-07 /pmc/articles/PMC6850066/ /pubmed/31033112 http://dx.doi.org/10.1111/hae.13762 Text en © 2019 The Authors. Haemophilia Published by John Wiley & Sons Ltd. This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
spellingShingle Original Articles
Carcao, Manuel
Escuriola‐Ettingshausen, Carmen
Santagostino, Elena
Oldenburg, Johannes
Liesner, Ri
Nolan, Beatrice
Bátorová, Angelika
Haya, Saturnino
Young, Guy
The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab
title The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab
title_full The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab
title_fullStr The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab
title_full_unstemmed The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab
title_short The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab
title_sort changing face of immune tolerance induction in haemophilia a with the advent of emicizumab
topic Original Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6850066/
https://www.ncbi.nlm.nih.gov/pubmed/31033112
http://dx.doi.org/10.1111/hae.13762
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