Safety and Efficacy of Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) in Previously Untreated Patients with Hemophilia B.

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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Abstract

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not develop FIX inhibitors; one 11-year-old patient developed an inhibitor against FIX after 8 EDs and was ultimately withdrawn.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.

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。
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。
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