Expanding the Toolbox With Non-Factor Therapies.

Area of Science:

• Hematology
• Pharmacology
• Internal Medicine

Background:

• Prophylaxis is standard care for people with haemophilia (PwH), preventing bleeds.
• Traditional prophylaxis requires frequent intravenous infusions, posing adherence challenges.
• Limitations include rapid drug clearance and impact on efficacy.

Purpose of the Study:

• To review advancements in haemophilia prophylaxis.
• To discuss the evolution from standard half-life to extended half-life and non-factor therapies.
• To highlight benefits and limitations of new prophylactic treatments.

Main Methods:

• Review of current literature on haemophilia prophylaxis.
• Analysis of standard half-life, extended half-life, and non-factor therapies.
• Discussion of clinical implications and patient outcomes.

Main Results:

• Extended half-life products offer greater flexibility and fewer infusions.
• Emicizumab provides subcutaneous prophylaxis for haemophilia A with inhibitors, but breakthrough bleeds can occur.
• Emerging non-factor therapies promise expanded prophylactic options for various haemophilia types.

Conclusions:

• Therapeutic options for haemophilia prophylaxis have significantly evolved.
• Newer agents improve adherence, reduce infusion frequency, and offer new administration routes.
• Continued innovation aims to provide better protection and personalized care for all PwH.