Tertiary prophylaxis in adults: is there a rationale?

Area of Science:

• Hematology
• Clinical Medicine
• Pharmacoeconomics

Background:

• Adult haemophilia management lacks consensus on regular prophylaxis effectiveness and cost-utility.
• Prophylaxis aims to prevent bleeds and reduce morbidity but increases factor consumption and costs compared to on-demand therapy.
• Current evidence from small cohorts shows variable effects on joint health despite reduced bleeding frequency.

Purpose of the Study:

• To review literature on adult-onset prophylaxis (started >18 years) in haemophilia.
• To identify barriers to adult prophylaxis adoption.
• To highlight research gaps in optimizing prophylaxis for clinical benefit and cost-effectiveness.

Main Methods:

• Systematic literature review of studies on adult haemophilia prophylaxis.
• Analysis of clinical outcomes, factor usage, and costs.
• Discussion of patient stratification and optimal dosing strategies.

Main Results:

• Prophylaxis reduces bleeding events but has variable impact on joint status.
• Adult prophylaxis incurs significantly higher costs due to increased factor consumption.
• No clear guidelines exist for identifying patient subsets who benefit most from tertiary prophylaxis.

Conclusions:

• Evidence for the clinical and economic utility of adult haemophilia prophylaxis remains limited.
• Barriers to prophylaxis uptake in adults require further investigation.
• Future research should focus on personalized prophylaxis strategies balancing efficacy and cost.