INTRODUCTION The phase 2/3 PROTECT VIII study demonstrated long-term efficacy and safety of damoctocog alfa pegol (BAY 94-9027; Jivi®), a B-domain-deleted recombinant factor VIII (FVIII), site-specifically PEGylated to improve its pharmacokinetic profile. We report a post hoc assessment of bleeding and safety outcomes in the subgroup of patients, aged 12-<18 years at enrolment. METHOD PROTECT VIII was a multicentre, open-label study of previously treated males aged 12-65 years with severe haemophilia A (FVIII <1%). Twelve patients were included in this analysis. All received damoctocog alfa pegol prophylaxis for the total time in study (median [range] time in study 4.0 [1.3-6.2] years). RESULTS Overall median (Q1; Q3) total and joint annualised bleeding rates were 1.8 (0.4; 5.1) and 0.7 (0.2; 1.8), respectively, for the entire study. During the last 6 months of treatment, eight (66.7%) and ten (83.3%) out of 12 patients experienced zero total and joint bleeds, respectively. No patient developed FVIII inhibitors. No deaths or thrombotic events were reported. CONCLUSION Efficacy and safety of damoctocog alfa pegol were confirmed in adolescent patients with haemophilia A, with data for up to 6 years supporting its use as a long-term treatment option in this group as they transition into adulthood.

中文翻译：

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Damoctocog Alfa Pegol 预防对参加 PROTECT VIII 时年龄为 12-<18 岁的 A 型血友病患者的长期疗效和安全性。


简介 2/3 期 PROTECT VIII 研究证明了 damoctocog alfa pegol（BAY 94-9027；Jivi®）的长期功效和安全性，damoctocog alfa pegol（BAY 94-9027；Jivi®）是一种 B 结构域缺失的重组因子 VIII (FVIII)，经过位点特异性聚乙二醇化以改善其药代动力学特征。我们报告了对入组时年龄为 12-<18 岁的患者亚组的出血和安全性结果的事后评估。 METHOD PROTECT VIII 是一项多中心、开放标签研究，对象为 12-65 岁患有严重血友病 A (FVIII <1%) 的既往治疗男性。该分析包括 12 名患者。所有患者在整个研究期间均接受 damoctocog alfa pegol 预防（研究中位时间[范围] 4.0 [1.3-6.2] 年）。结果 整个研究的总体中位（Q1；Q3）总出血率和关节年化出血率分别为 1.8（0.4；5.1）和 0.7（0.2；1.8）。在治疗的最后 6 个月中，12 名患者中分别有 8 名 (66.7%) 和 10 名 (83.3%) 经历了零总出血和关节出血。没有患者出现 FVIII 抑制剂。没有死亡或血栓事件的报道。结论 damoctocog alfa pegol 在青少年 A 型血友病患者中的有效性和安全性得到证实，长达 6 年的数据支持其在该群体过渡到成年时作为长期治疗选择。