Center for Thrombosis and Hemorrhagic Diseases, Humanitas Clinical and Research Center-IRCCS, Rozzano, Milan, Italy.
University of Minnesota Medical Center, Minneapolis, Minnesota, United States.
Thromb Haemost. 2021 Aug;121(8):1079-1086. doi: 10.1055/a-1333-5536. Epub 2020 Dec 9.
BAY 94-9027 (damoctocog alfa pegol, Jivi) is an extended-half-life recombinant factor VIII (rFVIII) shown to be well-tolerated and efficacious in bleeding prevention in previously treated patients with severe hemophilia A. During the PROTECT VIII study, prophylaxis patients received BAY 94-9027 at intervals determined based on their bleeding phenotype, observed during a 10-week run-in treatment period with twice-weekly dosing. Those with ≤ 1 spontaneous joint or muscle bleed were randomized to either 45 to 60 IU/kg every 5 days or 60 IU/kg every 7 days; patients could increase dosing frequency to every 5 days or twice weekly in the case of bleeds. Those enrolled after the randomization arms were full, and those with ≥ 2 bleeds in the run-in period, received 30 to 40 IU/kg twice weekly. Patients completing the main study could receive open-label BAY 94-9027 in the extension phase. Dosing regimen, total, and joint annualized bleeding rates were analyzed over three periods: prestudy, main study, and extension. A total of 80 patients who were on prophylaxis treatment prior to and during the study and had prior bleed data available were evaluated in this post hoc analysis of PROTECT VIII. Most patients (> 80%) required fewer infusions with BAY 94-9027 prophylaxis versus their previous standard-half-life (SHL) rFVIII product. Lower bleeding and joint bleeding rates were observed over time from the prestudy to the extension study period in all treatment regimens. Compared with SHL FVIII, BAY 94-9027 prophylaxis allows patients to reduce infusion frequency with maintained or improved protection from bleeds.
BAY 94-9027（达莫肝素奥法妥珠单抗，Jivi）是一种延长半衰期的重组凝血因子 VIII（rFVIII），在先前接受过治疗的重度 A 型血友病患者中，预防出血时显示出良好的耐受性和疗效。在 PROTECT VIII 研究中，预防治疗患者根据其在为期 10 周的导入治疗期内每两周一次给药时观察到的出血表型，以确定的间隔接受 BAY 94-9027 治疗。那些自发性关节或肌肉出血≤1 次的患者随机分为每 5 天接受 45 至 60IU/kg 或每 7 天接受 60IU/kg；如果出现出血，患者可以增加至每 5 天或每两周两次的给药频率。在随机分组手臂全部入组后，以及在导入期内出现≥2 次出血的患者，接受每周两次 30 至 40IU/kg 的治疗。完成主要研究的患者可以在扩展阶段接受开放标签的 BAY 94-9027 治疗。在三个时期（研究前、主要研究和扩展）分析了总剂量和关节年化出血率：研究前、主要研究和扩展。在 PROTECT VIII 的这项事后分析中，评估了 80 名在研究前和研究期间接受预防治疗且有先前出血数据的先前接受过预防治疗的患者。与他们以前的标准半衰期（SHL）rFVIII 产品相比，大多数患者（>80%）使用 BAY 94-9027 预防治疗需要的输注次数更少。在所有治疗方案中，从研究前到扩展研究期间，出血和关节出血率随时间推移而降低。与 SHL FVIII 相比，BAY 94-9027 预防治疗可让患者减少输注频率，同时保持或改善对出血的保护。
