Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Milan, Italy.
Haemophilia. 2021 May;27(3):434-444. doi: 10.1111/hae.14294. Epub 2021 Mar 16.
BAY 94-9027 (damoctocog alfa pegol; an extended half-life PEGylated recombinant factor VIII [FVIII]) demonstrated efficacy and safety in previously treated paediatric patients (PTPs) aged <12 years with severe haemophilia A in the PROTECT VIII Kids study (NCT01775618).
To evaluate the long-term safety of BAY 94-9027 in PTPs aged <12 years at enrolment.
In the PROTECT VIII Kids study, boys <12 years with severe haemophilia A were enrolled in two age cohorts (6-<12 years and <6 years) and treated prophylactically twice weekly, every 5 days or every 7 days, with BAY 94-9027 for ≥50 exposure days (EDs). Patients who had completed ≥50 EDs and ≥6 months in the main study or 12-week safety expansion study were eligible to participate in the extension. Primary safety variable was frequency of inhibitor development; main efficacy variable was annualised bleeding rate (ABR).
Of 73 PTPs from the main/expansion studies, 59 (81%) entered the extension phase for a median (range) duration of 5.0 (0.4-5.9) years. Overall, 39 patients completed ≥5 years of treatment. No patients developed FVIII inhibitors/anti-PEG antibodies, and two patients aged <6 years discontinued. Median ABR for total bleeds was 1.5 (<6 years) and 1.9 (6-<12 years). Total ABR improved in the extension vs. the main study. In the last 12 months of treatment, median spontaneous ABR was 0.0 in both age groups.
BAY 94-9027 showed long-term safety and efficacy for the prevention and treatment of bleeds in younger and older paediatric patients with severe haemophilia A.
BAY 94-9027（达莫肝素奥扎戈非洛；一种延长半衰期的聚乙二醇化重组因子 VIII [FVIII]）在以前接受过治疗的儿科患者（PTP）中表现出疗效和安全性，这些患者年龄<12 岁，患有严重的血友病 A，PROTECT VIII Kids 研究（NCT01775618）。
评估 BAY 94-9027 在<12 岁的 PTP 中的长期安全性。
在 PROTECT VIII Kids 研究中，年龄<12 岁的严重血友病 A 男孩被分为两个年龄组（6-<12 岁和<6 岁），并每周两次预防性使用 BAY 94-9027 治疗，每 5 天或每 7 天一次，至少使用 50 个暴露日（ED）。在主要研究或 12 周安全性扩展研究中完成≥50 个 ED 且≥6 个月的患者有资格参加扩展研究。主要安全性变量是抑制剂发展的频率；主要疗效变量是年化出血率（ABR）。
在主要/扩展研究的 73 名 PTP 中，59 名（81%）进入扩展阶段，中位（范围）持续时间为 5.0（0.4-5.9）年。总体而言，有 39 名患者完成了≥5 年的治疗。没有患者出现 FVIII 抑制剂/抗聚乙二醇抗体，有两名<6 岁的患者停药。总出血的中位数 ABR 为 1.5（<6 岁）和 1.9（6-<12 岁）。在扩展阶段，总 ABR 有所改善。在治疗的最后 12 个月，两个年龄组的自发性 ABR 中位数均为 0.0。
BAY 94-9027 对预防和治疗严重血友病 A 的年轻和年长儿童患者的出血具有长期安全性和疗效。
