Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B
Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B
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DOI：
10.1182/bloodadvances.2020004085
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发表时间：
2021-07-09
期刊：
影响因子：
7.5
通讯作者：
Liesner, Raina
中科院分区：
文献类型：
--
作者：
Nolan, Beatrice;Klukowska, Anna;Liesner, Raina
PUP B-LONG 评估了重组因子 IX Fc 融合蛋白 (rFIXFc) 在先前未经治疗的 B 型血友病患者 (PUP) 中的安全性和有效性。在这项开放标签的第 3 期研究中，雄性 PUP（年龄 = 50、>= 20 和 >= 10 暴露天数 (ED) 分别暴露于 rFIXFc。一名患者（3.03%；95% 置信区间，0.08% 至15.76%）在 11 次 ED 后出现低效价抑制剂；23 名患者（70%）出现 58 例治疗相关的严重不良事件（1 名患者出现 FIX 抑制和过敏反应，导致停药）。 (>85%) 出血事件仅需要 1 次输注即可解决。在第一项报告 rFIXFc 在患有 B 型血友病的 PUP 中的结果中，rFIXFc 具有良好的耐受性，在儿科血友病人群中，rFIXFc 的不良事件情况是有效的，无论是预防还是治疗出血事件。
PUPs B-LONG evaluated the safety and efficacy of recombinant factor IX Fc fusion protein (rFIXFc) in previously untreated patients (PUPs) with hemophilia B. In this open-label, phase 3 study, male PUPs (age = 50, >= 20, and >= 10 exposure days (EDs) to rFIXFc, respectively. One patient (3.03%; 95% confidence interval, 0.08% to 15.76%) developed a low-titer inhibitor after 11 EDs; no high-titer inhibitors were detected. Twenty-three patients (70%) had 58 treatment-emergent serious adverse events; 2 were assessed as related (FIX inhibition and hypersensitivity in 1 patient, resulting in withdrawal). Median ABR was 1.24 (interquartile range, 0.00-2.49) for patients receiving prophylaxis. Most (>85%) bleeding episodes required only 1 infusion for bleed resolution. In this first study reporting results with rFIXFc in pediatric PUPs with hemophilia B, rFIXFc was well tolerated, with the adverse event profile as expected in a pediatric hemophilia population. rFIXFc was effective, both as prophylaxis and in the treatment of bleeding episodes.