The Efficacy of Prophylaxis for Children with Severe Hemophilia in Korea - An Experience of Single Institute / 임상소아혈액종양

ABSTRACT

BACKGROUND: While primary prophylaxis is a well-established and recommended treatment for children with severe hemophilia, there has not been Korean own data. The aim of this study is to lay the foundation for the optimal and available management of hemophilia in Korea. METHODS: We reviewed the medical records of the patients treated with regular factor VIII concentrates infusion as long term continuous treatment at Seoul Clinic of Korea Hemophilia Foundation since August, 2004, retrospectively. We analyzed the efficacy of prophylaxis with the frequency of joint bleed and clinically meaningful bleeding episodes. We also assessed the cost of primary prophylaxis, secondary prophylaxis and on-demand therapy by the factor concentrates consumption. RESULTS: The data from 65 patients were available to evaluate. Of 65 patients, 22 were treated with primary prophylaxis, and 43 with secondary prophylaxis. In the primary and secondary prophylaxis groups, there were 1.9 joint bleeds and 3.3 other bleeding episodes, 7.2 and 9.0, respectively. The mean consumption of factor concentrates per person for primary prophylaxis, secondary prophylaxis and on-demand therapy groups were 2,658.8 IU/kg/year, 2,665.1 IU/kg/year, and 1,271.9 IU/kg/year respectively. CONCLUSION: Now prophylaxis is the recommended treatment for severe hemophilia patients, we should manage our hemophilia patients with standard treatment. And the optimizing treatment regimen for each individual will be needed to establish by studies of individual pharmacokinetics and bleeding tendency.