比较血友病B的延长半衰期因子IX （rIX-FP vs. rFIXFc和N9-GP）预防的实际结果：来自德国的医疗图表数据分析

IF 4 3区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL

Advances in Therapy Pub Date : 2025-09-18 DOI:10.1007/s12325-025-03336-y

Martin Olivieri, Songkai Yan, Ying Yang, Radovan Tomic, Thomas Linhoff, Xiang Zhang, Douglass Drelich, Natalie Jakobs, Wolfgang Miesbach

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引用次数: 0

摘要

在德国，三种延长半衰期因子IX （FIX）产品通常用于治疗B型血友病（PwHB）患者。然而，仍然迫切需要区分PwHB的治疗方法。本研究的目的是在德国临床实践中评估rIX-FP与rFIXFc和N9-GP在预防方面的有效性和利用率。方法：回顾性分析年龄≥12岁的PwHB合并中重度B型血友病患者，接受rIX-FP、rFIXFc或N9-GP预防治疗≥12个月。主要终点是FIX的消耗；次要结局包括年化出血率（ABR）、年化自发出血率（AsBR）和年化关节出血率（AjBR）。结果：纳入PwHB: rIX-FP 138例，n = 52；rFIXFc, n = 55；N9-GP, n = 31。rIX-FP组的FIX平均消耗量（46.9 IU/kg/周）显著低于rFIXFc组（70.1 IU/kg/周，p = 0.0083），但与N9-GP组（47.2 IU/kg/周，p = 0.9331）差异不显著。接受rIX-FP预防的PwHB患者的平均出血率显著低于接受N9-GP的患者（ABR: 0.8 vs. 1.5, p = 0.0472; AsBR: 0.1 vs. 0.6, p = 0.0092; AjBR: 0.2 vs. 0.6, p = 0.0140）。rIX-FP和rFIXFc的出血率无显著差异。结论：与rFIXFc相比，rIX-FP预防与显著降低FIX消耗和数值（但不显著）降低出血率相关。与N9-GP相比，rIX-FP预防与相似的FIX消耗和显着降低出血率相关。

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Comparing Real-World Outcomes of Prophylaxis with Extended Half-life Factor IX (rIX-FP vs. rFIXFc and N9-GP) for Haemophilia B: An Analysis of Medical Chart Data from Germany.

Introduction: In Germany, three extended half-life factor IX (FIX) products are commonly used to treat people with haemophilia B (PwHB). However, there remains a critical need to differentiate treatments for PwHB. The aim of this study was to assess the effectiveness and utilisation of rIX-FP compared with rFIXFc and N9-GP for prophylaxis in clinical practice in Germany.

Methods: A retrospective chart review was performed for PwHB aged ≥ 12 years with moderate/severe haemophilia B, who received prophylaxis with rIX-FP, rFIXFc or N9-GP for ≥ 12 months. The primary outcome was FIX consumption; secondary outcomes included annualised bleeding rate (ABR), annualised spontaneous bleeding rate (AsBR) and annualised joint bleeding rate (AjBR).

Results: The study included 138 PwHB: rIX-FP, n = 52; rFIXFc, n = 55; and N9-GP, n = 31. Mean FIX consumption with rIX-FP (46.9 IU/kg/week) was significantly lower than that of rFIXFc (70.1 IU/kg/week, p = 0.0083) but not significantly different from N9-GP (47.2 IU/kg/week, p = 0.9331). PwHB receiving rIX-FP prophylaxis had significantly lower mean bleeding rates than those receiving N9-GP (ABR: 0.8 vs. 1.5, p = 0.0472; AsBR: 0.1 vs. 0.6, p = 0.0092; and AjBR: 0.2 vs. 0.6, p = 0.0140). Bleeding rates for rIX-FP and rFIXFc did not differ significantly.

Conclusion: rIX-FP prophylaxis was associated with significantly lower FIX consumption and numerically (but not significantly) lower bleeding rates compared with rFIXFc. Compared to N9-GP, prophylaxis with rIX-FP was associated with similar FIX consumption and significantly lower bleeding rates.

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来源期刊

Advances in Therapy 医学-药学

CiteScore

7.20

自引率

2.60%

发文量

353

审稿时长

6-12 weeks

期刊介绍： Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.