Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?

The development of inhibitory antibodies is a severe complication of clotting factor replacement therapy in patients with severe haemophilia A (HA). Current World Federation of Hemophilia (WFH) guidelines for haemophilia care indicate that eradication of inhibitors is best achieved through immune to...

Full description

Bibliographic Details
Authors: Holstein, Katharina, Quellec, Sandra Le, Klamroth, Robert, Batorova, Angelika, Holme, Pål Andre, Jiménez Yuste, Víctor Manuel, Astermark, Jan
Format: article
Publication Date:2021
Country:España
Institution:Universidad Autónoma de Madrid
Repository:Biblos-e Archivo. Repositorio Institucional de la UAM
Language:English
OAI Identifier:oai:repositorio.uam.es:10486/711213
Online Access:http://hdl.handle.net/10486/711213
https://dx.doi.org/10.1111/hae.14470
Access Level:Open access
Keyword:emicizumab
factorVIII
haemophilia A
immune tolerance induction
inhibitors
prophylaxis
Medicina
id ES_73594710b6fde8d33ea1e4e7ed792491
oai_identifier_str oai:repositorio.uam.es:10486/711213
network_acronym_str ES
network_name_str España
repository_id_str
spelling Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?Holstein, KatharinaQuellec, Sandra LeKlamroth, RobertBatorova, AngelikaHolme, Pål AndreJiménez Yuste, Víctor ManuelAstermark, JanemicizumabfactorVIIIhaemophilia Aimmune tolerance inductioninhibitorsprophylaxisMedicinaThe development of inhibitory antibodies is a severe complication of clotting factor replacement therapy in patients with severe haemophilia A (HA). Current World Federation of Hemophilia (WFH) guidelines for haemophilia care indicate that eradication of inhibitors is best achieved through immune tolerance induction (ITI) therapy. Aim: The European Collaborative Haemophilia Network conducted a survey to determine whether ITI is still used in the routine management of patients with HA, and whether the availability of emicizumab prophylaxis has influenced treatment decisions. Methods: The survey was conducted in late 2020/early 2021 in 18 centres representing 17 countries in the Europe/Middle East region treating a total of 4955 patients, and included sections specific to patient and centre demographics, treatment protocols (both ITI and prophylactic), inhibitor development and initiation of ITI, treatment success, and the incidence of adverse events. Results: While our results indicate that ITI can still be considered a mainstay of treatment for patients with HA with inhibitors, less than daily dosing of ITI in combination with emicizumab prophylaxis is becoming commonplace across the spectrum of disease severity, with initiation being guided by bleeding patterns. The most frequently cited reasons for not initiating emicizumab prophylaxis were availability or reimbursement issues. Conclusion: ITI remains a mainstay for haemophilia treatment of patients with HA with inhibitors, but emicizumab has become a preferred first-line approach to protect against bleeds and represents an alternative to burdensome ITI in certain patient groupsWileyDepartamento de MedicinaFacultad de Medicina20212021-12-17research articlehttp://purl.org/coar/resource_type/c_2df8fbb1VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10486/711213https://dx.doi.org/10.1111/hae.14470reponame:Biblos-e Archivo. Repositorio Institucional de la UAMinstname:Universidad Autónoma de MadridInglésengopen accesshttp://purl.org/coar/access_right/c_abf2info:eu-repo/semantics/openAccessoai:repositorio.uam.es:10486/7112132026-06-23T12:46:27Z
dc.title.none.fl_str_mv Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?
title Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?
spellingShingle Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?
Holstein, Katharina
emicizumab
factorVIII
haemophilia A
immune tolerance induction
inhibitors
prophylaxis
Medicina
title_short Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?
title_full Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?
title_fullStr Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?
title_full_unstemmed Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?
title_sort Immune tolerance induction in the era of emicizumab – still the first choice for patients with haemophilia A and inhibitors?
dc.creator.none.fl_str_mv Holstein, Katharina
Quellec, Sandra Le
Klamroth, Robert
Batorova, Angelika
Holme, Pål Andre
Jiménez Yuste, Víctor Manuel
Astermark, Jan
author Holstein, Katharina
author_facet Holstein, Katharina
Quellec, Sandra Le
Klamroth, Robert
Batorova, Angelika
Holme, Pål Andre
Jiménez Yuste, Víctor Manuel
Astermark, Jan
author_role author
author2 Quellec, Sandra Le
Klamroth, Robert
Batorova, Angelika
Holme, Pål Andre
Jiménez Yuste, Víctor Manuel
Astermark, Jan
author2_role author
author
author
author
author
author
dc.contributor.none.fl_str_mv Departamento de Medicina
Facultad de Medicina
dc.subject.none.fl_str_mv emicizumab
factorVIII
haemophilia A
immune tolerance induction
inhibitors
prophylaxis
Medicina
topic emicizumab
factorVIII
haemophilia A
immune tolerance induction
inhibitors
prophylaxis
Medicina
description The development of inhibitory antibodies is a severe complication of clotting factor replacement therapy in patients with severe haemophilia A (HA). Current World Federation of Hemophilia (WFH) guidelines for haemophilia care indicate that eradication of inhibitors is best achieved through immune tolerance induction (ITI) therapy. Aim: The European Collaborative Haemophilia Network conducted a survey to determine whether ITI is still used in the routine management of patients with HA, and whether the availability of emicizumab prophylaxis has influenced treatment decisions. Methods: The survey was conducted in late 2020/early 2021 in 18 centres representing 17 countries in the Europe/Middle East region treating a total of 4955 patients, and included sections specific to patient and centre demographics, treatment protocols (both ITI and prophylactic), inhibitor development and initiation of ITI, treatment success, and the incidence of adverse events. Results: While our results indicate that ITI can still be considered a mainstay of treatment for patients with HA with inhibitors, less than daily dosing of ITI in combination with emicizumab prophylaxis is becoming commonplace across the spectrum of disease severity, with initiation being guided by bleeding patterns. The most frequently cited reasons for not initiating emicizumab prophylaxis were availability or reimbursement issues. Conclusion: ITI remains a mainstay for haemophilia treatment of patients with HA with inhibitors, but emicizumab has become a preferred first-line approach to protect against bleeds and represents an alternative to burdensome ITI in certain patient groups
publishDate 2021
dc.date.none.fl_str_mv 2021
2021-12-17
dc.type.none.fl_str_mv research article
http://purl.org/coar/resource_type/c_2df8fbb1
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv http://hdl.handle.net/10486/711213
https://dx.doi.org/10.1111/hae.14470
url http://hdl.handle.net/10486/711213
https://dx.doi.org/10.1111/hae.14470
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Wiley
publisher.none.fl_str_mv Wiley
dc.source.none.fl_str_mv reponame:Biblos-e Archivo. Repositorio Institucional de la UAM
instname:Universidad Autónoma de Madrid
instname_str Universidad Autónoma de Madrid
reponame_str Biblos-e Archivo. Repositorio Institucional de la UAM
collection Biblos-e Archivo. Repositorio Institucional de la UAM
repository.name.fl_str_mv
repository.mail.fl_str_mv
_version_ 1869410803451428864
score 15,301629

Similar Items