Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A
Authors: Batorova Angelika ,   1. Department of Hematology and Transfusion Medicine, National Hemophilia Center, Faculty of Medicine of Comenius University and University Hospital, Bratislava, Slovakia,   https://orcid.org/0000-0003-0567-4250, Boban Ana ,   2. Haemophilia Centre, University Hospital Centre Zagreb, School of Medicine, University of Zagreb, Zagreb, Croatia,   https://orcid.org/0000-0003-3532-2336, Brinza Melen ,   3. Centre of Hematology and Bone Marrow Transplant, European Haemophilia Comprehensive Care Centre, Fundeni Clinical Institute, Bucharest, Romania, Lissitchkov Toshiko ,   4. Expert Center for Rare Haematological Disorders, Sofia, Bulgaria, Nemes Laszlo ,   5. National Haemophilia Centre and Haemostasis Department, Medical Centre of Hungarian Defence Forces, Budapest, Hungary, Zupan Irena Preložnik ,   6. Department of Haematology, University Medical Centre Ljubljana, Faculty of Medicine, University of Ljubljana, Ljubljana, Slovenia,   https://orcid.org/0000-0001-7074-0926, Smejkal Petr ,   7. Department of Clinical Haematology, University Hospital Brno, Brno, Czech Republic,   8. Department of Laboratory Methods, Faculty of Medicine, Masaryk University, Brno, Czech Republic, Zozulya Nadezhda ,   9. National Research Center for Hematology, Moscow, Russia,   https://orcid.org/0000-0001-9933-549X, Windyga Jerzy ,   10. Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland,   Jerzy Windyga, Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland. E-mail: jwindyga@ihit.waw.pl
Published: November 2022

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.