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

Scientific paper - Original scientific paper

• Author: Angelika Batorova
• Author: Ana Boban
• Author: Melen Brinza
• Author: Toshiko Lissitchkov
• Author: Laszlo Nemes
• Author: Irena Preložnik Zupan
• Author: Petr Smejkal
• Author: Nadezhda Zozulya
• Author: Jerzy Windyga
• Author's institution: University of Zagreb; School of Medicine; (Department of Internal Medicine)
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Clinical Medical Sciences; Internal Medicine

Abstract (english)

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.

• Keywords (english): Europe; Expert Testimony; Hemophilia A / drug therapy; Hemophilia A / prevention & control; Hemorrhage / prevention & control; Humans
• Language: english
• Publication type: Scientific paper - Original scientific paper
• Publication status: Published
• Peer review: Peer review
• Publication version: Published version
• Journal title: Journal of Medicine and Life
• Numbering: vol. 15, no. 4, pp. 570-578
• p-ISSN: 1844-122X
• e-ISSN: 1844-3117
• DOI: https://doi.org/10.25122/jml-2022-0103
• URN:NBN: urn:nbn:hr:105:402005
• Publication: 2022-04
• Document URL: https://medandlife.org/wp-content/uploads/JMedLife-15-570.pdf
• Type of resource: Text
• Access conditions: Open access
• Created on: 2022-12-12 09:14:20