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

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F65269705%3A_____%2F22%3A00076576" target="_blank" >RIV/65269705:_____/22:00076576 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00216224:14110/22:00128517

Výsledek na webu

<a href="https://medandlife.org/wp-content/uploads/JMedLife-15-570.pdf" target="_blank" >https://medandlife.org/wp-content/uploads/JMedLife-15-570.pdf</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.25122/jml-2022-0103" target="_blank" >10.25122/jml-2022-0103</a>

Jazyk výsledku

angličtina

Název v původním jazyce

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

Popis výsledku v původním jazyce

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 GREATER-THAN OR EQUAL TO30-50 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 &quot;one-size-fits-all&quot; approach to hemophilia A management to maintain optimal trough levels for individual patients.

Název v anglickém jazyce

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

Popis výsledku anglicky

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 GREATER-THAN OR EQUAL TO30-50 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 &quot;one-size-fits-all&quot; approach to hemophilia A management to maintain optimal trough levels for individual patients.

Druh

J_SC - Článek v periodiku v databázi SCOPUS

CEP obor

—

OECD FORD obor

30218 - General and internal medicine

Projekt

—

Návaznosti

N - Vyzkumna aktivita podporovana z neverejnych zdroju

Rok uplatnění

2022

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Journal of Medicine and Life

ISSN

1844-122X

e-ISSN

1844-3117

Svazek periodika

15

Číslo periodika v rámci svazku

4

Stát vydavatele periodika

RO - Rumunsko

Počet stran výsledku

9

Strana od-do

570-578

Kód UT WoS článku

—

EID výsledku v databázi Scopus

2-s2.0-85131223365