The retrospective study of cost-effectiveness for prophylactic and on-demand treatment of the pediatric patients with hemophilia A in Iran

Hemophilia is the second most common inherited bleeding disorder after von Willebrand disease. Delayed clotting is usually observed in all main types of hemophilia caused by deficiencies in blood coagulation factors. Primary prophylaxis is considered the gold standard to prevent acute hemarthrosis and chronic arthropathy in patients with severe hemophilia A. However, some low-income countries, like Iran, have to prioritize access to preventive care to balance the financial resources and per capita access to coagulation factors. In order to compare the cost-effectiveness of on-demand and preventive treatment, we conducted a retrospective study on 55 patients with hemophilia A. We collected data from two groups of patients: those who had received routine prophylactic treatment and those who had received on-demand (episodic) treatment. The results of our study revealed a significant difference in the annualized bleeding rates (ABRs) between the two groups: ABR in the prophylactic treatment group was lower than in the on-demand treatment group (2.19 vs 7.25). In addition, we found substantial differences between the prophylactic and on-demand treatment groups in the number of hospital visits per year (9.8 vs 14.41), the annual number of infusions (107.35 vs 229.58), and in the mean cost of treatment (30.96€ vs 63€). In conclusion, prophylactic therapy seems to be more effective than on-demand treatment. This study was conducted in accordance with the principles of the Declaration of Helsinki. The study was approved by the Ethics Committee of the Ahvaz Jundishapur University of Medical Sciences, Ahvaz, Iran (IR.AJUMS.REC.1398.456). Written informed consent was obtained from the patients’ parents.