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. 2025 Jun 26;14(13):4555.
doi: 10.3390/jcm14134555.

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

Young-Shil Park  1 , Tai Ju Hwang  2 , Sang Kyu Park  3 , Eun Jin Choi  4 , Jeong A Park  5 , Hee Jo Baek  6 , Chuhl Joo Lyu  7 , Jae Hee Lee  8 , Mi Kyung Kim  9 , Ji Yoon Kim  10 , Sun Ah Lee  11 , Boram Park  12 , Da-Hye Kim  12 , Sung Beom Chung  12 , Chung-Mo Nam  13 , Yaeji Lee  14 , Ki Young Yoo  15
Affiliations

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

Young-Shil Park et al. J Clin Med. .

Abstract

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.

Keywords: annual bleeding events; factor IX; haemophilia B; prophylactic treatment; real-world evidence; treatment adherence and compliance.

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Conflict of interest statement

This study was sponsored by Pfizer. Boram Park, Da-Hye Kim, and Sung Beom Chung are employees of Pfizer. The development of this manuscript was proposed by Pfizer. However, the funders had no role in the design of the study; in the collection, analyses, or interpretation of data; in the writing of the manuscript, or in the decision to publish the results. The remaining authors declare no conflict of interest and did not receive any payment or editorial/medical writing support from Pfizer in connection with the development of this manuscript.

Figures

Figure 1
Figure 1
Correlations between annual bleeding events (ABEs) and treatment patterns. (A) Duration of treatment; (B) Dose in prophylaxis; (C) Dose in on-demand; (D) Factor consumption. Each subfigure shows a scatterplot with a least square line and its 95% confidence interval. ‘R’ represents the correlation coefficient and ‘p’ represents the p-value.
Figure 2
Figure 2
Relationships between treatment patterns and bleeding events. (A) Incidence rate ratio by treatment regimen for annual bleeding events. (B) Incidence rate ratio by the dose of factor IX for monthly bleeding events.
See this image and copyright information in PMC

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