连接凝血因子 IX 和白蛋白的重组融合蛋白 (rIX-FP) 对既往未接受过治疗的 B 型血友病患者的安全性和有效性。

Richard Lemons, Michael Wang, Julie Curtin, Lynda Mae Lepatan, Christoph Male, Flora Peyvandi, Mario von Depka Prondzinski, Rongrong Wang, William McKeand, Wilfried Seifert, Johannes Oldenburg
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引用次数: 0

摘要

导言:凝血因子 IX (FIX) 与白蛋白的重组融合蛋白(rIX-FP)已被证明是一种有效、耐受性良好的治疗方法,适用于之前接受过因子替代疗法的重度血友病 B 患者。本研究调查了 rIX-FP 在既往未接受过治疗的患者 (PUP) 中的安全性和有效性。方法 以前未接受过 FIX 替代产品治疗的中度/重度 B 型血友病患者(FIX ≤2%)每周接受一次 rIX-FP(25-75 IU/kg)预防性治疗或按需治疗,暴露天数≥50 天 (ED)。主要结果是出现 FIX 抑制剂的患者人数,以及服用 50 IU/kg 剂量 rIX-FP 后的平均恢复增量 (IR)。次要结果包括不良事件(AE)发生率和年出血率(ABR)。结果 共有 12 名中位数年龄为 0 岁(0-11 岁)的 PUP 接受了 rIX-FP 治疗,中位数为 50 次 ED(6/12 次预防;6/12 次按需再预防)。总体而言,11/12 名患者未出现 FIX 抑制剂;一名 11 岁患者在接受 8 次 ED 治疗后出现了 FIX 抑制剂,并最终停药。IR的平均值(标准差)为1.2(0.4,n = 8)(IU/dL)/(IU/kg)。在记录的 137 例治疗突发 AE 中,有 5 例归因于 rIX-FP。在预防方案中,ABR 的中位数为 1.0(范围为 0-3.9,n = 12)。研究期间未发生血栓栓塞事件或死亡。结论 本研究提供的数据支持了 rIX-FP 在需要按需或预防性治疗中度/重度 B 型血友病的 PUP 中的安全性和有效性,这与之前治疗患者的结果一致。总体而言,1/12 的患者出现了 FIX 抑制剂。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Safety and Efficacy of Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) in Previously Untreated Patients with Hemophilia B.

Introduction  Recombinant fusion protein linking coagulation factor IX (FIX) with albumin (rIX-FP) has been shown to be an effective, well-tolerated treatment for patients with severe hemophilia B who had previously received factor replacement therapy. This study investigated the safety and efficacy of rIX-FP in previously untreated patients (PUPs). Methods  Patients with moderately severe/severe hemophilia B (≤2% FIX) previously untreated with FIX replacement products received rIX-FP (25-75 IU/kg) prophylaxis weekly or on-demand treatment over ≥50 exposure days (EDs). Primary outcomes were the number of patients who developed FIX inhibitors and mean incremental recovery (IR) following a 50 IU/kg dose of rIX-FP. Secondary outcomes included incidence of adverse events (AEs) and annualized bleeding rates (ABRs). Results  In total, 12 PUPs with a median age of 0 years (range, 0-11 years) were treated with rIX-FP for a median of 50 EDs (6/12 prophylaxis; 6/12 on-demand then prophylaxis). Overall, 11/12 patients did not develop FIX inhibitors; one 11-year-old patient developed an inhibitor against FIX after 8 EDs and was ultimately withdrawn. Mean (standard deviation) IR was 1.2 (0.4, n  = 8) (IU/dL)/(IU/kg). Of the 137 treatment-emergent AEs recorded, five were attributed to rIX-FP. On the prophylaxis regimen, median ABR was 1.0 (range, 0-3.9, n  = 12). No thromboembolic events or deaths occurred during the study. Conclusion  This study provides data to support the safety and efficacy of rIX-FP in PUPs requiring on-demand or prophylactic treatment for moderately severe/severe hemophilia B, consistent with results in previously treated patients. Overall, 1/12 patients developed an inhibitor against FIX.

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