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Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis
P S Smith1, S M Teutsch, P A Shaffer
1Department of Pediatrics, Brown University, Providence 02903, USA.
The Journal of Pediatrics
|September 1, 1996
Summary
Prophylactic care for severe hemophilia A significantly reduces bleeding events but incurs substantial costs. Cost-effectiveness improves with longer treatment duration and lower concentrate prices.
Area of Science:
- Hematology
- Health Economics
- Pediatric Care
Background:
- Severe hemophilia A requires lifelong management to prevent bleeding complications.
- Current treatment paradigms include episodic care for bleeding events and prophylactic infusions.
- The cost-effectiveness of these approaches requires detailed analysis.
Purpose of the Study:
- To evaluate the incremental cost-effectiveness of prophylactic versus episodic care in boys with severe hemophilia A.
- To compare the economic impact of different prophylactic infusion durations.
Main Methods:
- A comparative analysis using charge data from 11 U.S. hemophilia treatment centers.
- Data collected from 70 boys on episodic care and 27 boys on prophylactic care over two years.
- A cost-effectiveness model developed for hypothetical patients from ages 3 to 50 years.
Main Results:
- Prophylactic care resulted in significantly fewer bleeding events (median 3 vs 31 annually) but higher concentrate use.
- Factor VIII concentrate constituted over 93% of costs for both care types.
- Prophylaxis from ages 3-20 cost $1100 per bleeding event prevented; from 3-50 years, $1380.
- Total prophylactic costs could match episodic costs if concentrate prices were halved.
Conclusions:
- Prophylactic care substantially decreases bleeding frequency and is expected to prevent joint damage.
- Despite significant costs, prophylaxis offers clinical benefits in managing severe hemophilia A.
- Economic viability of prophylaxis is sensitive to Factor VIII concentrate pricing.
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