Scholarlink

Introduction <p>The current standard of care for people with severe hemophilia B is prophylaxis with factor IX (FIX) products. This analysis assessed the effectiveness of prophylaxis for people with hemophilia B (PwHB) receiving rIX-FP or rFIXFc prophylaxis in Germany and Italy. </p> Methods <p>A retrospective, de-identified chart review included PwHB&#xa0;≥&#xa0;12&#xa0;years with severe/moderate hemophilia B from Germany or Italy, receiving prophylaxis with rIX-FP or rFIXFc for&#xa0;≥&#xa0;12&#xa0;months. The primary outcome was FIX consumption; the secondary outcomes were dosing interval, annualized bleeding rate (ABR), annualized spontaneous bleeding rate (AsBR), and annualized joint bleeding rate (AjBR). These outcomes were also explored in PwHB with pre- and post-rIX-FP switch data.</p> Results <p>Of 194 PwHB, 107 and 87 received rIX-FP and rFIXFc prophylaxis, respectively. The mean FIX consumption of rIX-FP was significantly lower compared to rFIXFc (42.4 vs. 65.2&#xa0;IU/kg/week, <i>p</i>&#xa0;=&#xa0;0.0001), with mean dosing intervals of 9.5&#xa0;days (rIX-FP) and 7.9&#xa0;days (rFIXFc). The mean bleeding rates for rIX-FP versus rFIXFc, respectively, were: ABR 0.7 versus 1.1 (<i>p</i>&#xa0;=&#xa0;0.6704), AsBR 0.1 versus 0.3 (<i>p</i>&#xa0;=&#xa0;0.3427), and AjBR 0.3 versus 0.4 (<i>p</i>&#xa0;=&#xa0;0.5296). Subgroup analyses for PwHB with severe and moderate hemophilia B separately showed similar numerical patterns when comparing these outcomes. In the 18 patients with switch data, a significant reduction in FIX consumption was observed (median 51.7 to 33.3&#xa0;IU/kg/week, <i>p</i>&#xa0;=&#xa0;0.0069), and the mean dosing interval was extended (7.2–9.5&#xa0;days). The ABR (median 1.6–0.0, <i>p</i>&#xa0;=&#xa0;0.0172; <i>n</i>&#xa0;=&#xa0;18) and AjBR (median 0.6–0.0, <i>p</i>&#xa0;=&#xa0;0.0200; <i>n</i>&#xa0;=&#xa0;14) decreased significantly, while the AsBR decreased but not significantly (median 0.2–0.0, <i>p</i>&#xa0;=&#xa0;0.1460; <i>n</i>&#xa0;=&#xa0;14).</p> Conclusion <p>rIX-FP prophylaxis was associated with reduced FIX consumption versus rFIXFc and offered equally effective or potentially improved bleed protection. Additionally, PwHB who switched to rIX-FP achieved significant decreases in FIX consumption, ABR, and AjBR compared with their prior FIX product.</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Real-World Prophylaxis Outcomes with rIX-FP and rFIXFc for Males with Hemophilia B: Pooled Analysis of Medical Chart Data from Germany and Italy

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年7月23日

查看全文
  • Johannes Oldenburg,
  • Martin Olivieri,
  • Songkai Yan,
  • Ying Yang,
  • Radovan Tomic,
  • Xiang Zhang,
  • Douglass Drelich,
  • Natalie Jakobs,
  • Mariasanta Napolitano

摘要

Introduction

The current standard of care for people with severe hemophilia B is prophylaxis with factor IX (FIX) products. This analysis assessed the effectiveness of prophylaxis for people with hemophilia B (PwHB) receiving rIX-FP or rFIXFc prophylaxis in Germany and Italy.

Methods

A retrospective, de-identified chart review included PwHB ≥ 12 years with severe/moderate hemophilia B from Germany or Italy, receiving prophylaxis with rIX-FP or rFIXFc for ≥ 12 months. The primary outcome was FIX consumption; the secondary outcomes were dosing interval, annualized bleeding rate (ABR), annualized spontaneous bleeding rate (AsBR), and annualized joint bleeding rate (AjBR). These outcomes were also explored in PwHB with pre- and post-rIX-FP switch data.

Results

Of 194 PwHB, 107 and 87 received rIX-FP and rFIXFc prophylaxis, respectively. The mean FIX consumption of rIX-FP was significantly lower compared to rFIXFc (42.4 vs. 65.2 IU/kg/week, p = 0.0001), with mean dosing intervals of 9.5 days (rIX-FP) and 7.9 days (rFIXFc). The mean bleeding rates for rIX-FP versus rFIXFc, respectively, were: ABR 0.7 versus 1.1 (p = 0.6704), AsBR 0.1 versus 0.3 (p = 0.3427), and AjBR 0.3 versus 0.4 (p = 0.5296). Subgroup analyses for PwHB with severe and moderate hemophilia B separately showed similar numerical patterns when comparing these outcomes. In the 18 patients with switch data, a significant reduction in FIX consumption was observed (median 51.7 to 33.3 IU/kg/week, p = 0.0069), and the mean dosing interval was extended (7.2–9.5 days). The ABR (median 1.6–0.0, p = 0.0172; n = 18) and AjBR (median 0.6–0.0, p = 0.0200; n = 14) decreased significantly, while the AsBR decreased but not significantly (median 0.2–0.0, p = 0.1460; n = 14).

Conclusion

rIX-FP prophylaxis was associated with reduced FIX consumption versus rFIXFc and offered equally effective or potentially improved bleed protection. Additionally, PwHB who switched to rIX-FP achieved significant decreases in FIX consumption, ABR, and AjBR compared with their prior FIX product.