Cost-effectiveness Analysis of Prophylaxis Versus On-demand Treatment for Children With Hemophilia B Without Inhibitors in China

Purpose: Hemophilia B (HB) is a hereditary bleeding disorder caused by a deficiency of coagulation factor IX (FIX), which represents 15% to 20% of all patients with hemophilia. Clinical studies have found significant benefits of prophylaxis treatment with FIX versus on-demand (OD) treatment. However, these benefits are associated with an increase in FIX consumption and a considerable increase in cost. Most Chinese children with HB receive OD treatment. Only a small proportion of patients with HB receive prophylaxis treatment in China. The patients with inhibitors could result in more complicated bleeding events, joint status, or treatment patterns. The objective of this study is to assess the cost-effectiveness of prophylaxis compared with OD treatment in children with HB without inhibitors from the Chinese health care perspective. Methods: A Markov model was used to analyze cost-effectiveness by comparing prophylaxis with OD treatment. The model uses a 17-year time horizon with a yearly cycle. Transition probabilities and utility weights were estimated using published studies. The cost data for patients with HB were collected from Beijing Children's Hospital and Capital Medical Uni-versity. One-way and probabilistic sensitivity analyses were performed to assess the robustness of the results. Findings: The model projects that prophylaxis has an incremental 1.23 quality-adjusted life-years (QALYs). The incremental cost per QALY gained for individuals with HB receiving prophylaxis was yen 155,709.80 ($23,530.36) compared with the OD group, which is under the willingness-to-pay threshold (3 times the gross domestic product per capital according to the World Health Organization rec-ommendations) in China of yen 193,932 ($29,306.37). Moreover, 1-way sensitivity analysis found that the results were sensitive to the utility of nonarticular bleeding. The lower this parameter is, the higher the probability is of the incremental cost-effectiveness ratio for prophylaxis not being cost-effective. This finding infers that when the patients have a better QALY (higher utility) at the beginning, the cost for benefit from prophylaxis treatment is lower. The results of the probabilistic sensitivity analyses indicate that the probability of prophylaxis being cost-effective is 89.3%. Implications: Although prophylaxis is a costly treatment, the results of this study indicate that it is cost-effective compared with OD treatment for children with HB in China. (Clin Ther. 2021;43:1536- 1546.) (c) 2021 Published by Elsevier Inc.