Park Young-Shil, Hwang Tai Ju, Park Sang Kyu, Choi Eun Jin, Park Jeong A, Baek Hee Jo, Lyu Chuhl Joo, Lee Jae Hee, Kim Mi Kyung, Kim Ji Yoon, Lee Sun Ah, Park Boram, Kim Da-Hye, Chung Sung Beom, Nam Chung-Mo, Lee Yaeji, Yoo Ki Young
Department of Pediatrics, Kyung Hee University Hospital at Gangdong, Seoul 05278, Republic of Korea.
Korea Hemophilia Foundation Clinic, Gwangju 61932, Republic of Korea.
J Clin Med. 2025 Jun 26;14(13):4555. doi: 10.3390/jcm14134555.
In Haemophilia B, guideline-level factor IX (FIX) prophylaxis is recommended, but real-world dosing and adherence vary. To assess treatment patterns, adherence, FIX dosing, and their associations with bleeding events in Korean patients. 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 < 0.10 and clinical relevance were included in multivariable models. The prophylaxis group showed significantly fewer ABEs (incidence rate ratio [IRR]: 0.383, = 0.011). Each 100 IU/kg monthly dose increment reduced bleed risk (IRR: 0.692, < 0.001). Adherence showed no independent association with bleeding in adjusted models. 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.
在乙型血友病中，推荐采用指南级别的凝血因子IX（FIX）预防治疗，但实际应用中的剂量和依从性存在差异。为了评估韩国患者的治疗模式、依从性、FIX剂量及其与出血事件的关联，我们对12个中心（2022年6月至2023年5月）接受FIX治疗≥12个月的130例韩国乙型血友病患者进行了回顾性病历审查和一次性调查。共有47例患者（36.2%）接受了预防治疗（≥90 IU/kg/周，持续≥45周）；其余患者采用非预防性治疗。使用负二项回归分析年化出血事件（ABEs），并采用广义线性混合模型分析每月出血情况。多变量模型纳入了P<0.10且具有临床相关性的协变量。预防治疗组的ABEs显著减少（发病率比[IRR]：0.383，P = 0.011）。每月每增加100 IU/kg剂量可降低出血风险（IRR：0.692，P<0.001）。在调整后的模型中，依从性与出血无独立关联。乙型血友病的出血预防更多地取决于实际接受的FIX暴露量，而非治疗方案标签。研究定义的持续预防治疗仍未得到充分应用且剂量不足。个体化给药和持续的依从性监测对于缩小这一治疗差距并改善治疗效果至关重要。
