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Introduction <p>In Germany, three extended half-life factor&#xa0;IX (FIX) products are commonly used to treat people with haemophilia&#xa0;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.</p> Methods <p>A retrospective chart review was performed for PwHB aged ≥ 12&#xa0;years with moderate/severe haemophilia&#xa0;B, who received prophylaxis with rIX-FP, rFIXFc or N9-GP for ≥ 12&#xa0;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).</p> Results <p>The study included 138 PwHB: rIX-FP, <i>n</i> = 52; rFIXFc, <i>n</i> = 55; and N9-GP, <i>n</i> = 31. Mean FIX consumption with rIX-FP (46.9&#xa0;IU/kg/week) was significantly lower than that of rFIXFc (70.1&#xa0;IU/kg/week, <i>p</i> = 0.0083) but not significantly different from N9-GP (47.2&#xa0;IU/kg/week, <i>p</i> = 0.9331). PwHB receiving rIX-FP prophylaxis had significantly lower mean bleeding rates than those receiving N9-GP (ABR: 0.8 vs. 1.5, <i>p</i> = 0.0472; AsBR: 0.1 vs. 0.6, <i>p</i> = 0.0092; and AjBR: 0.2 vs. 0.6, <i>p</i> = 0.0140). Bleeding rates for rIX-FP and rFIXFc did not differ significantly.</p> Conclusion <p>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.</p>

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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

  • 期刊论文

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  • 发表日期：

    2025年9月18日

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

摘要

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.