Economic Evaluation of Immune Tolerance Induction in Children With Severe Hemophilia A and High-Responding Inhibitors: A Cost-Effectiveness Analysis of Prophylaxis With Emicizumab.

This study aimed to measure the cost-effectiveness of prophylaxis with emicizumab in PsHAhri on ITI in Brazil. A cost-effectiveness modeling analysis was used to estimate the costs per PsHAhri on ITI and the number of prevented bleedings from undertaking one intervention (prophylaxis with BpA) over another (prophylaxis with emicizumab), based on the Brazilian Ministry of Health perspective. Costs of ITI with recombinant FVIII, prophylaxis with BpA or emicizumab, and treated bleeding episodes with BpA costs were evaluated for PsHAhri who had ITI success or failure. This study was conducted with the perspective of the Brazilian Ministry of Health (payer). During ITI, prophylaxis with BpA cost US $924 666/PsHAhri/ITI, whereas prophylaxis with emicizumab cost US $488 785/PsHAhri/ITI. During ITI, there was an average of 9.32 bleeding episodes/PsHAhri/ITI when BpA were used as prophylaxis and 0.67 bleeding/PsHAhri/ITI when emicizumab was used. By univariate deterministic sensitivity analysis, emicizumab remained dominant whichever variable was modified. In this study, prophylaxis with emicizumab during ITI is a dominant option compared with prophylaxis with BpA during ITI. • The development of anti-factor VIII antibodies (inhibitor) is related to worse outcomes of treatment of people with severe hemophilia A (PsHA). • Immune tolerance induction (ITI) is the treatment of choice to eradicate inhibitors in PsHA and high-responding inhibitors (PsHAhri). The effectiveness of bypassing agents for preventing bleeding during ITI is already known. Assuming the same effectiveness of emicizumab in preventing bleeding in PsHAhri not under ITI, we evaluated the cost-effectiveness of emicizumab for PsHAhri undergoing ITI according to the Brazilian ITI protocol. • By univariate deterministic and probabilistic sensitivity analyses, emicizumab remained dominant whichever variable was modified. [ABSTRACT FROM AUTHOR]