Cost-effectiveness analysis of prophylaxis vs. “on demand” approach in the management in children with hemophilia A in Mexico

Hemophilia is a rare bleeding disorder that is attributed to a defi ciency of coagulation factors VIII, IX and XI. This defi ciency leads to a tendency of moderate to severe bleeding. This disorder occurs mainly in males, who represent 97.32% of cases.1 Hemophilia A is caused by a factor VIII (FVIII) defi ciency.2 Globally it is estimated that the prevalence of hemophilia A in 2012 was 363,668 cases.3 In Mexico there were 3582 cases reported in 2010.l In general, it is believed that ~60% of cases of hemophilia are severe,4 i.e., when the concentration of clotting factor is <0.01 IU/ml.5,6 Hemophilia patients are at risk of bleeding in different parts of the body. The joints are the most affected and ~80% of the bleeding occurs in knees, elbows and ankles.7 As a result of the repeated joint hemorrhages, there are alterations in the tissues that make up the joint which, in turn, degenerate into chronic synovitis and destruction of the surfaces that make up the joint (hemophilic arthropathy).5 Currently, liver transplantation is the only option to cure hemophilia. On the other hand, the goal of treatment is to increase blood concentration of FVIII using replacement products in order to prevent and stop spontaneous bleeding (during the daily life of the patients) or excessive bleeding (during surgery).5 RESEARCH ARTICLE Bol Med Hosp Infant Mex 2013;70(4):290-297