Olivieri Martin, Yan Songkai, Yang Ying, Tomic Radovan, Linhoff Thomas, Zhang Xiang, Drelich Douglass, Jakobs Natalie, Miesbach Wolfgang
Paediatric Thrombosis and Haemostasis Unit, Dr. Von Hauner Children's Hospital, LMU Munich, Munich, Germany.
CSL Behring, 1020 First Avenue, King of Prussia, PA, 19406, USA.
Adv Ther. 2025 Sep 18. doi: 10.1007/s12325-025-03336-y.
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.
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).
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.
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.
在德国，三种延长半衰期的凝血因子IX（FIX）产品常用于治疗B型血友病患者（PwHB）。然而，对于PwHB的治疗仍迫切需要加以区分。本研究的目的是评估在德国临床实践中，重组FIX融合蛋白（rIX-FP）与重组FIX聚乙二醇化修饰物（rFIXFc）和N9-糖蛋白（N9-GP）用于预防治疗的有效性和使用情况。
对年龄≥12岁、患有中度/重度B型血友病且接受rIX-FP、rFIXFc或N9-GP预防治疗≥12个月的PwHB进行回顾性病历审查。主要结局指标是FIX用量；次要结局指标包括年化出血率（ABR）、年化自发出血率（AsBR）和年化关节出血率（AjBR）。
该研究纳入了138例PwHB：rIX-FP组52例；rFIXFc组55例；N9-GP组31例。rIX-FP的平均FIX用量（46.9 IU/kg/周）显著低于rFIXFc（70.1 IU/kg/周，p = 0.0083），但与N9-GP（47.2 IU/kg/周，p = 0.9331）无显著差异。接受rIX-FP预防治疗的PwHB的平均出血率显著低于接受N9-GP预防治疗的患者（ABR：0.8对1.5，p = 0.0472；AsBR：0.1对0.6，p = 0.0092；AjBR：0.2对0.6，p = 0.0140）。rIX-FP和rFIXFc的出血率无显著差异。
与rFIXFc相比，rIX-FP预防治疗的FIX用量显著更低，出血率在数值上更低（但无显著差异）。与N9-GP相比，rIX-FP预防治疗的FIX用量相似，但出血率显著更低。
