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Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A

The next frontier in hemophilia A management has arrived. However, questions remain regarding the broader applicability of new and emerging hemophilia A therapies, such as the long-term safety and efficacy of non-factor therapies and optimal regimens for individual patients. With an ever-evolving cl...

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Autores principales: Batorova, Angelika, Boban, Ana, Brinza, Melen, Lissitchkov, Toshiko, Nemes, Laszlo, Zupan Preložnik, Irena, Smejkal, Petr, Zozulya, Nadezhda, Windyga, Jerzy
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Carol Davila University Press 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9126455/
https://www.ncbi.nlm.nih.gov/pubmed/35646171
http://dx.doi.org/10.25122/jml-2022-0103
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author Batorova, Angelika
Boban, Ana
Brinza, Melen
Lissitchkov, Toshiko
Nemes, Laszlo
Zupan Preložnik, Irena
Smejkal, Petr
Zozulya, Nadezhda
Windyga, Jerzy
author_facet Batorova, Angelika
Boban, Ana
Brinza, Melen
Lissitchkov, Toshiko
Nemes, Laszlo
Zupan Preložnik, Irena
Smejkal, Petr
Zozulya, Nadezhda
Windyga, Jerzy
author_sort Batorova, Angelika
collection PubMed
description The next frontier in hemophilia A management has arrived. However, questions remain regarding the broader applicability of new and emerging hemophilia A therapies, such as the long-term safety and efficacy of non-factor therapies and optimal regimens for individual patients. With an ever-evolving clinical landscape, it is imperative for physicians to understand how available and future hemophilia A therapies could potentially be integrated into real-life clinical practice to improve patient outcomes. Against this background, nine hemophilia experts from Central European countries participated in a pre-advisory board meeting survey. The survey comprised 11 multiple-choice questions about current treatment practices and future factor and non-factor replacement therapies. The survey questions were developed to reflect current unmet needs in hemophilia management reflected in the literature. The experts also took part in a follow-up advisory board meeting to discuss the most important unmet needs for hemophilia management as well as the pre-meeting survey results. All experts highlighted the challenge of maintaining optimal trough levels with prophylaxis as their most pressing concern. Targeting trough levels of ≥30–50 IU/L or even higher to achieve less bleeding was highlighted as their preferred strategy. However, the experts had an equal opinion on how this could be achieved (i.e., more efficacious non-factor therapies or factor therapy offering broader personalization possibilities such as targeting trough levels to individual pharmacokinetic data). In summary, our study favors personalized prophylaxis to individual pharmacokinetic data rather than a "one-size-fits-all" approach to hemophilia A management to maintain optimal trough levels for individual patients.
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spelling pubmed-91264552022-06-01 Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A Batorova, Angelika Boban, Ana Brinza, Melen Lissitchkov, Toshiko Nemes, Laszlo Zupan Preložnik, Irena Smejkal, Petr Zozulya, Nadezhda Windyga, Jerzy J Med Life Original Article The next frontier in hemophilia A management has arrived. However, questions remain regarding the broader applicability of new and emerging hemophilia A therapies, such as the long-term safety and efficacy of non-factor therapies and optimal regimens for individual patients. With an ever-evolving clinical landscape, it is imperative for physicians to understand how available and future hemophilia A therapies could potentially be integrated into real-life clinical practice to improve patient outcomes. Against this background, nine hemophilia experts from Central European countries participated in a pre-advisory board meeting survey. The survey comprised 11 multiple-choice questions about current treatment practices and future factor and non-factor replacement therapies. The survey questions were developed to reflect current unmet needs in hemophilia management reflected in the literature. The experts also took part in a follow-up advisory board meeting to discuss the most important unmet needs for hemophilia management as well as the pre-meeting survey results. All experts highlighted the challenge of maintaining optimal trough levels with prophylaxis as their most pressing concern. Targeting trough levels of ≥30–50 IU/L or even higher to achieve less bleeding was highlighted as their preferred strategy. However, the experts had an equal opinion on how this could be achieved (i.e., more efficacious non-factor therapies or factor therapy offering broader personalization possibilities such as targeting trough levels to individual pharmacokinetic data). In summary, our study favors personalized prophylaxis to individual pharmacokinetic data rather than a "one-size-fits-all" approach to hemophilia A management to maintain optimal trough levels for individual patients. Carol Davila University Press 2022-04 /pmc/articles/PMC9126455/ /pubmed/35646171 http://dx.doi.org/10.25122/jml-2022-0103 Text en ©2022 JOURNAL of MEDICINE and LIFE https://creativecommons.org/licenses/by/3.0/This article is distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0/ (https://creativecommons.org/licenses/by/3.0/) ), which permits unrestricted use and redistribution provided that the original author and source are credited.
spellingShingle Original Article
Batorova, Angelika
Boban, Ana
Brinza, Melen
Lissitchkov, Toshiko
Nemes, Laszlo
Zupan Preložnik, Irena
Smejkal, Petr
Zozulya, Nadezhda
Windyga, Jerzy
Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A
title Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A
title_full Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A
title_fullStr Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A
title_full_unstemmed Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A
title_short Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A
title_sort expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia a
topic Original Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9126455/
https://www.ncbi.nlm.nih.gov/pubmed/35646171
http://dx.doi.org/10.25122/jml-2022-0103
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