Prophylaxis in adults with haemophilia

Area of Science:

• Hematology
• Internal Medicine
• Pharmacology

Background:

• Prophylaxis in adults with haemophilia (PWH) is controversial, with unclear benefits versus costs, especially given existing arthropathy.
• The objectives of secondary prophylaxis in adults differ from primary prophylaxis in children.
• Decisions regarding continuing primary prophylaxis into adulthood are uncertain.

Purpose of the Study:

• To review the literature on prophylactic treatment in adult patients with haemophilia A.
• To evaluate the efficacy and indications for prophylaxis in adults.
• To explore the discontinuation of prophylaxis in this population.

Main Methods:

• Literature review of studies on prophylactic treatment in adult haemophilia A patients.
• Analysis of studies examining the discontinuation of prophylaxis.
• Synthesis of findings regarding clinical benefits and cost-effectiveness.

Main Results:

• Studies suggest clinical benefit from prophylaxis in adulthood, including fewer bleeding episodes, less pain, and improved quality of life compared to on-demand treatment.
• Optimized prophylaxis, such as tailored intermediate- or low-dose regimens, may improve clinical outcomes.
• The cost-effectiveness argument for adult prophylaxis is more complex than for children, but indirect benefits may counterbalance costs.

Conclusions:

• Prophylaxis in adults with haemophilia A demonstrates clinical benefits, supporting its use.
• Tailoring prophylaxis regimens based on individual bleeding frequency may enhance outcomes.
• While direct cost-effectiveness is challenging, indirect benefits like increased workdays and reduced healthcare utilization support prophylaxis.