Assessing factor consumption and bleeding outcomes of prophylaxis with 3 commonly prescribed factor IX products for hemophilia B: A retrospective patient medical record analysis in the United States
Year: 2025
Type: article
Study design: Observational Study
Abstract: BACKGROUND: In the United States, rIX-FP, rFIXFc, and rFIX are approved as treatment options for people with hemophilia B (PwHB). Although clinical trials have demonstrated the efficacy and safety of each product, real-world data can help to understand their use and treatment outcomes in the absence of direct head-to-head trials. OBJECTIVE: To assess real-world factor IX (FIX) consumption and bleeding outcomes for PwHB receiving rIX-FP, rFIXFc, or rFIX prophylaxis. METHODS: Retrospective, deidentified medical record information for PwHB with moderate or severe hemophilia B (FIX activity ≤5%) treated with rIX-FP, rFIXFc, or rFIX prophylaxis for at least 12 months was obtained from Hemophilia Treatment Centers in the United States between 2020 and 2023. FIX consumption was calculated using the most recently prescribed dosing frequency and dosage. Annualized bleeding rate (ABR), annualized spontaneous bleeding rate, and annualized joint bleeding rate were calculated based on the number of bleeding events over the observation period. Generalized linear models adjusting for covariates were used to test the statistical significance of the differences of consumption and ABR among the products. RESULTS: = 0.0009). CONCLUSIONS: In this retrospective study, rIX-FP prophylaxis was associated with lower FIX consumption and potentially improved protection against bleeds compared with prophylactic treatment with rFIXFc and rFIX.
Authors: Miguel Escobar, Songkai Yan, Ying Yang, Douglass Drelich, Xiang Zhang, Gbenga Kayode, Mariama Jabati, Mindy Simpson
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: green
OpenAlex ID: W4416225356