Impact of switching prophylaxis treatment from factor VIII to emicizumab in hemophilia A patients without inhibitors. (31st December 2023)

• Record Type: Journal Article
• Authors: Escobar, Miguel; Agrawal, Neha; Chatterjee, Sagnik; Bhattacharya, Swastik; Caicedo, Jorge; Bullano, Michael; Schultz, Bob G.
• Is Part Of: Journal of medical economics. Volume 26:Number 1(2023)
• Page Start: 574
• Page End: 580
• Publication Date: 2023-12-31
• Subjects: Hemophilia -- non-inhibitor -- real-world -- FVIII -- emicizumab -- comparative; I11 -- I1 -- I -- I00; Medical care -- Cost control -- Periodicals; Medical economics -- Periodicals; 362.10941
• DOI: 10.1080/13696998.2023.2196922
• Languages: English
• ISSNs: 1369-6998

Abstract

Abstract: Background: Factor VIII (FVIII) replacement and emicizumab are effective at preventing bleeds in patients with hemophilia A (HA). Though benefits of emicizumab among inhibitor patients with HA (PwHA) are well established, more real-world evidence among non-inhibitor patients is needed. Methods: Using a United States healthcare claims database, we compared billed annualized bleed rates (ABRb ) and the total cost of care (TCC) before and after switching from FVIII prophylaxis to emicizumab among non-inhibitor male PwHA. Bayesian inferences were used to assess the difference in ABRb and TCC per patient per year (PPPY) pre- versus post-prophylaxis switch. Results: We included 101 non-inhibitor male PwHA aged between 3 and 63 years old who switched from FVIII prophylaxis to emicizumab prophylaxis in 2018 or 2019. The ABRb increased from 0.52 to 0.62 ( p = 0.83) after switch. The posterior probability of the mean ABRb increasing after the switch was 75.54%. The TCC PPPY increased from $517, 143 to $627, 005 ( p < 0.0001) after switch and the posterior probability of mean costs increasing after the switch was 99.80%. Conclusions: Personalization of care through the identification of the most appropriate therapy for each patient can optimize clinical and economic outcomes. Future real-world evidence research could help establish the value of prophylactic options in targeted populations such as the non-inhibitor male PwHA.