Characteristics and Treatment Patterns of Patients with Haemophilia B Receiving Recombinant Coagulation Factor IX

초록

Introduction: In Haemophilia B, guideline-level factor IX (FIX) prophylaxis is recommended, but real-world dosing and adherence vary. Aim: To assess treatment patterns, adherence, FIX dosing, and their associations with bleeding events in Korean patients. Methods: We conducted a retrospective chart review and one-time survey of 130 Korean patients with haemophilia B treated with FIX for >= 12 months at 12 centers (June 2022-May 2023). A total of forty-seven patients (36.2%) received prophylaxis (>= 90 IU/kg/week for >= 45 weeks); the remainder were managed non-prophylactically. Annualized bleeding events (ABEs) were analyzed using negative binomial regression, and monthly bleeds with a generalized linear mixed model. Covariates with p < 0.10 and clinical relevance were included in multivariable models. Results: The prophylaxis group showed significantly fewer ABEs (incidence rate ratio [IRR]: 0.383, p = 0.011). Each 100 IU/kg monthly dose increment reduced bleed risk (IRR: 0.692, p < 0.001). Adherence showed no independent association with bleeding in adjusted models. Conclusions: Bleed prevention in haemophilia B is driven more by delivered FIX exposure than by regimen label. Study-defined sustained prophylaxis remains underused and under-dosed. Individualized dosing and continuous adherence monitoring are essential to close this treatment gap and improve outcomes.

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