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.

Basic information

Original name

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

Authors

BATOROVA, Angelika, Ana BOBAN, Melen BRINZA, Toshiko LISSITCHKOV, Laszlo NEMES, Irena Zupan PRELOŽNIK, Petr SMEJKAL (203 Czech Republic, belonging to the institution), Nadezhda ZOZULYA and Jerzy WINDYGA (guarantor)

Edition

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

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30205 Hematology

Country of publisher

Romania

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

RIV identification code

RIV/00216224:14110/22:00128517

Organization unit

Faculty of Medicine

DOI

Keywords in English

hemophilia; extended half-life; prophylaxis

Tags

Tags

International impact, Reviewed
Změněno: 3/2/2023 09:02, Mgr. Tereza Miškechová

Abstract

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.