Abstract
Hemophilia A is a genetic disease in which there is a deficiency in the level of activity of the clotting factor VIII, the moderate form is defined as a blood clotting factor level from 0.01 to 0.05 IU/ml. The aim of this analysis is to estimate the cost-effectiveness of prophylactic treatment of moderate hemophilia A compared to demand treatment in Colombia. A decision tree model was developed using a life expectancy horizon and a societal perspective; annual discount rate of 3% was applied to costs and effectiveness measures. Prophylactic treatment with recombinant factor VIII (25 IU/kg 3 times per week) was compared to on demand treatment (40 IU/kg BID for 3.5 days for each bleeding episode). The base case was simulated based on a patient of 8 years old and weight of 31kg without inhibitors. Effectiveness and probabilities of adverse events were taken from the literature. Costs (direct and indirect) were taken from local tariff manuals (SOAT and SISMED). Effectiveness measures were number of cases avoided of bleeding, hemarthrosis and arthropathies. All data were validated with a clinical expert. Univariate sensitivity analysis was done. Costs are presented in 2013 USD (exchange rate US$1 = $ 1927 COP). Over the time horizon, prophylactic treatment avoids 277 episodes of bleeding and 23 of joint damage. Total expected costs with prophylactic treatment were US$ 2.7M compared to on demand treatment US$ 2.4M. The incremental cost effectiveness ratios (ICER) for prophylactic treatment were US$ 1,056/avoided bleeding and US$12,512/avoided hemarthrosis/arthropathies. Sensitivity analysis showed the robustness of the results (average ICER US$ 1,153/avoided bleeding). From the social perspective, prophylactic treatment of moderate hemophilia A, with recombinant factor VIII would be a highly cost-effectiveness intervention with strong health benefits in number of cases of bleeding and joint damage avoided.
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