Real-world data on treatment regimen and medical use among patients in hemophilia B in Korea

Abstract

Abstract Background We aimed to investigate the real-world treatment status such as treatment regimen, bleeding events, and drug dose of patients with hemophilia B in South Korea. Methods In this retrospective chart review study, we collected the data of hemophilia B patients from the 8 university hospitals. Demographic and clinical data, treatment data such as regimen and injection number, factor IX concentrate dose, and bleeding data were reviewed. Descriptive analyses were done with annual data of 2019, 2020, and 2021, respectively as well as consecutive 3 years. Results 150 hemophilia B patients with medical records between January 1, 2019 and December 31, 2021 were collected. Among those, 72 (48.0%) was severe, 47 (31.3%) was moderate, 28 (18.7%) was mild. The results showed approximately 2 times more patients receiving prophylaxis therapy than on-demand therapy, with 66.1% patients receiving prophylaxis therapy in 2019, 64.9% in 2020, and 72.1% in 2021. Annualized bleeding rate was observed to be 2.2 (±3.1) in 2019, 1.8 (±3.0) in 2020, and 1.8 (±2.9) in 2021 among patients with prophylaxis regimens. For factor IX concentrate doses, patients with prophylaxis regimens received an average of 41.6 (±11.9) IU/Kg/Injection in 2019, 45.7 (±12.9) IU/Kg/Injection in 2020, and 60.1 (±24.0) IU/Kg/Injection in 2021. Conclusions In the real-world, prophylaxis is more prevalent than reported. Based on the insights gained from current real-world clinical evidence, it is expected that patients' unmet medical needs can be identified and physicians can consider the patient's situation and actively manage hemophilia B with more effective treatment strategies.