Cargando…

Management of haemophilia A with inhibitors: A regional cross‐talk

INTRODUCTION: The development of inhibitors with factor VIII (FVIII) replacement therapy is one of the most common and challenging complications of haemophilia A (HA) treatment, jeopardising treatment efficacy and predisposing patients to high risks of morbidity and mortality. The management of pati...

Descripción completa

Detalles Bibliográficos
Autores principales: Peyvandi, Flora, Kavakli, Kaan, El‐Beshlawy, Amal, Rangarajan, Savita
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9796719/
https://www.ncbi.nlm.nih.gov/pubmed/35868021
http://dx.doi.org/10.1111/hae.14638
_version_ 1784860550058999808
author Peyvandi, Flora
Kavakli, Kaan
El‐Beshlawy, Amal
Rangarajan, Savita
author_facet Peyvandi, Flora
Kavakli, Kaan
El‐Beshlawy, Amal
Rangarajan, Savita
author_sort Peyvandi, Flora
collection PubMed
description INTRODUCTION: The development of inhibitors with factor VIII (FVIII) replacement therapy is one of the most common and challenging complications of haemophilia A (HA) treatment, jeopardising treatment efficacy and predisposing patients to high risks of morbidity and mortality. The management of patients with inhibitors is particularly challenging in countries where resources are limited. AIM: To provide a comprehensive summary of the management of HA with inhibitors while focusing on differences in practice between Western and non‐Western countries and how resource scarcity can impact HA management, leading to suboptimal outcomes in patients with inhibitors. METHODS: Summary of key evidence and regional expert opinion. RESULTS: We address, particularly, the diagnosis of and testing for inhibitors, as well as the epidemiology of inhibitors, including incidence, prevalence and disease burden. Secondly, we provide an overview of the current treatment landscape in HA with inhibitors regarding the eradication of inhibitors with immune tolerance induction and the treatment and prevention of bleeding with bypassing agents, non‐factor replacement agents and other experimental therapies. This is complemented with insights from the authors around the applicability of, and challenges associated with, such therapies in their settings of practice. CONCLUSIONS: We conclude by proposing some key steps towards bridging the gaps in the management of HA with inhibitors in resource‐limited countries, including: (1) the collection of quality data that can inform healthcare reforms and policies; (2) improving disease knowledge among healthcare practitioners and patients with the aim of standardising disease management across centres and (3) working towards promoting equal access to HA care and therapies for everyone.
format Online
Article
Text
id pubmed-9796719
institution National Center for Biotechnology Information
language English
publishDate 2022
publisher John Wiley and Sons Inc.
record_format MEDLINE/PubMed
spelling pubmed-97967192023-01-04 Management of haemophilia A with inhibitors: A regional cross‐talk Peyvandi, Flora Kavakli, Kaan El‐Beshlawy, Amal Rangarajan, Savita Haemophilia Reviews INTRODUCTION: The development of inhibitors with factor VIII (FVIII) replacement therapy is one of the most common and challenging complications of haemophilia A (HA) treatment, jeopardising treatment efficacy and predisposing patients to high risks of morbidity and mortality. The management of patients with inhibitors is particularly challenging in countries where resources are limited. AIM: To provide a comprehensive summary of the management of HA with inhibitors while focusing on differences in practice between Western and non‐Western countries and how resource scarcity can impact HA management, leading to suboptimal outcomes in patients with inhibitors. METHODS: Summary of key evidence and regional expert opinion. RESULTS: We address, particularly, the diagnosis of and testing for inhibitors, as well as the epidemiology of inhibitors, including incidence, prevalence and disease burden. Secondly, we provide an overview of the current treatment landscape in HA with inhibitors regarding the eradication of inhibitors with immune tolerance induction and the treatment and prevention of bleeding with bypassing agents, non‐factor replacement agents and other experimental therapies. This is complemented with insights from the authors around the applicability of, and challenges associated with, such therapies in their settings of practice. CONCLUSIONS: We conclude by proposing some key steps towards bridging the gaps in the management of HA with inhibitors in resource‐limited countries, including: (1) the collection of quality data that can inform healthcare reforms and policies; (2) improving disease knowledge among healthcare practitioners and patients with the aim of standardising disease management across centres and (3) working towards promoting equal access to HA care and therapies for everyone. John Wiley and Sons Inc. 2022-07-22 2022-11 /pmc/articles/PMC9796719/ /pubmed/35868021 http://dx.doi.org/10.1111/hae.14638 Text en © 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Reviews
Peyvandi, Flora
Kavakli, Kaan
El‐Beshlawy, Amal
Rangarajan, Savita
Management of haemophilia A with inhibitors: A regional cross‐talk
title Management of haemophilia A with inhibitors: A regional cross‐talk
title_full Management of haemophilia A with inhibitors: A regional cross‐talk
title_fullStr Management of haemophilia A with inhibitors: A regional cross‐talk
title_full_unstemmed Management of haemophilia A with inhibitors: A regional cross‐talk
title_short Management of haemophilia A with inhibitors: A regional cross‐talk
title_sort management of haemophilia a with inhibitors: a regional cross‐talk
topic Reviews
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9796719/
https://www.ncbi.nlm.nih.gov/pubmed/35868021
http://dx.doi.org/10.1111/hae.14638
work_keys_str_mv AT peyvandiflora managementofhaemophiliaawithinhibitorsaregionalcrosstalk
AT kavaklikaan managementofhaemophiliaawithinhibitorsaregionalcrosstalk
AT elbeshlawyamal managementofhaemophiliaawithinhibitorsaregionalcrosstalk
AT rangarajansavita managementofhaemophiliaawithinhibitorsaregionalcrosstalk