Department of Hemostasis Disorders and Internal Medicine, Institute of Haematology and Transfusion Medicine, Warsaw, Poland.
Institute of Blood Pathology and Transfusion Medicine, Lviv, Ukraine.
Clin Appl Thromb Hemost. 2020 Jan-Dec;26:1076029620950836. doi: 10.1177/1076029620950836.
This phase 3, prospective, open-label, multicenter, continuation study (NCT01286779) investigated the use of a recombinant factor IX (FIX), nonacog gamma (BAX 326, RIXUBIS) in patients with severe or moderately severe hemophilia B. The study population included 85 patients transitioning from a phase 1/3 pivotal study (NCT01174446), a pediatric study (NCT01488994), and 30 newly recruited patients, naïve to nonacog gamma. Patients received nonacog gamma as prophylaxis treatment (standard, modified or PK-tailored) or on-demand, as determined by the investigator. Treatment was assessed for safety, immunogenicity, hemostatic efficacy and consumption. In this study, after ≥100 exposure days, nonacog gamma resulted in no treatment-related serious adverse events, and no patients developed inhibitory antibodies to FIX. Nonacog gamma was efficacious at controlling bleeding episodes, with an 89.1% overall hemostatic efficacy rating of excellent or good, and 56% of bleeds resolved with one infusion. The annualized bleeding rate was considerably lower during prophylactic treatment (median ABR of 1.3 in 108 patients) than during on-demand treatment (median ABR of 16.5 in 13 patients). These results show that in previously treated patients and nonacog gamma-naïve patients, long-term use of nonacog gamma had acceptable safety and tolerability, and was efficacious as a prophylactic treatment for the management of bleeding episodes.NCT01286779, EudraCT: 2010-022726-33.
这项 3 期、前瞻性、开放标签、多中心、延续研究（NCT01286779）调查了重组凝血因子 IX（FIX）、非活化凝血因子 IX（FIX）（BAX 326，RIXUBIS）在重度或中度重度乙型血友病患者中的应用。研究人群包括 85 名从 1 期/3 期关键研究（NCT01174446）、儿科研究（NCT01488994）和 30 名新招募的、对非活化凝血因子 IX 无经验的患者中过渡而来的患者。根据研究者的决定，患者接受非活化凝血因子 IX 作为预防治疗（标准、改良或 PK 定制）或按需治疗。治疗安全性、免疫原性、止血疗效和消耗情况进行了评估。在这项研究中，≥100 个暴露日后，非活化凝血因子 IX 没有导致与治疗相关的严重不良事件，也没有患者产生针对 FIX 的抑制性抗体。非活化凝血因子 IX 能有效控制出血发作，总体止血疗效评分为优秀或良好的比例为 89.1%，56%的出血发作通过一次输注得到解决。在预防治疗期间（108 名患者的年出血率中位数 ABR 为 1.3），年出血率明显低于按需治疗期间（13 名患者的 ABR 中位数为 16.5）。这些结果表明，在先前接受治疗的患者和非活化凝血因子 IX 无经验的患者中，长期使用非活化凝血因子 IX 具有可接受的安全性和耐受性，并且作为预防治疗管理出血发作是有效的。NCT01286779，EudraCT：2010-022726-33。
