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.