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

J 2022

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

BATOROVA, Angelika, Ana BOBAN, Melen BRINZA, Toshiko LISSITCHKOV, Laszlo NEMES et. al.

Základní údaje

Originální název

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

Autoři

BATOROVA, Angelika, Ana BOBAN, Melen BRINZA, Toshiko LISSITCHKOV, Laszlo NEMES, Irena Zupan PRELOŽNIK, Petr SMEJKAL (203 Česká republika, domácí), Nadezhda ZOZULYA a Jerzy WINDYGA (garant)

Vydání

Journal of Medicine and Life, Bucharest, Carol Davila University Press, 2022, 1844-122X

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30205 Hematology

Stát vydavatele

Rumunsko

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Kód RIV

RIV/00216224:14110/22:00128517

Organizační jednotka

Lékařská fakulta

DOI

http://dx.doi.org/10.25122/jml-2022-0103

Klíčová slova anglicky

hemophilia; extended half-life; prophylaxis

Štítky

rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 3. 2. 2023 09:02, Mgr. Tereza Miškechová

Anotace

V originále

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.

Citovat

BATOROVA, Angelika, Ana BOBAN, Melen BRINZA, Toshiko LISSITCHKOV, Laszlo NEMES, Irena Zupan PRELOŽNIK, Petr SMEJKAL, Nadezhda ZOZULYA a Jerzy WINDYGA. Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A. Journal of Medicine and Life. Bucharest: Carol Davila University Press, 2022, roč. 15, č. 4, s. 570-578. ISSN 1844-122X. Dostupné z: https://dx.doi.org/10.25122/jml-2022-0103.

@article{2252389,
   author = {Batorova, Angelika and Boban, Ana and Brinza, Melen and Lissitchkov, Toshiko and Nemes, Laszlo and Preložnik, Irena Zupan and Smejkal, Petr and Zozulya, Nadezhda and Windyga, Jerzy},
   article_location = {Bucharest},
   article_number = {4},
   doi = {http://dx.doi.org/10.25122/jml-2022-0103},
   keywords = {hemophilia; extended half-life; prophylaxis},
   language = {eng},
   issn = {1844-122X},
   journal = {Journal of Medicine and Life},
   title = {Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A},
   url = {https://medandlife.org/all-issues/2022/issue-4-2022/original-article-issue-4-2022/expert-opinion-on-current-and-future-prophylaxis-therapies-aimed-at-improving-protection-for-people-with-hemophilia-a/},
   volume = {15},
   year = {2022}
}

TY  - JOUR
ID  - 2252389
AU  - Batorova, Angelika - Boban, Ana - Brinza, Melen - Lissitchkov, Toshiko - Nemes, Laszlo - Preložnik, Irena Zupan - Smejkal, Petr - Zozulya, Nadezhda - Windyga, Jerzy
PY  - 2022
TI  - Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A
JF  - Journal of Medicine and Life
VL  - 15
IS  - 4
SP  - 570-578
EP  - 570-578
PB  - Carol Davila University Press
SN  - 1844122X
KW  - hemophilia
KW  - extended half-life
KW  - prophylaxis
UR  - https://medandlife.org/all-issues/2022/issue-4-2022/original-article-issue-4-2022/expert-opinion-on-current-and-future-prophylaxis-therapies-aimed-at-improving-protection-for-people-with-hemophilia-a/
N2  - 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.
ER  -

BATOROVA, Angelika, Ana BOBAN, Melen BRINZA, Toshiko LISSITCHKOV, Laszlo NEMES, Irena Zupan PRELOŽNIK, Petr SMEJKAL, Nadezhda ZOZULYA a Jerzy WINDYGA. Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A. \textit{Journal of Medicine and Life}. Bucharest: Carol Davila University Press, 2022, roč.~15, č.~4, s.~570-578. ISSN~1844-122X. Dostupné z: https://dx.doi.org/10.25122/jml-2022-0103.

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