Beijing, Children's Hospital, Capital Medical University, Beijing, People's Republic of China.
Xiangya Hospital Central South University, Hunan, People's Republic of China.
Haemophilia. 2022 Nov;28(6):e199-e208. doi: 10.1111/hae.14622. Epub 2022 Jul 8.
Omfiloctocog alfa, the first China-developed recombinant factor VIII (FVIII), demonstrated efficacy and safety of prophylaxis in previously treated patients (PTPs) aged ≥12 years with severe hemophilia A in China.
To investigate efficacy, safety and pharmacokinetics (PK) of omfiloctocog alfa in pediatric PTPs with severe hemophilia A in China.
PTPs (>50 exposure days [ED] for Chinese patients aged <6 years; >150 EDs for patients aged 6-12 years) were treated with omfiloctocog alfa at 25-50 IU/kg every other day or three times per week for 24 weeks. PK was evaluated after single injection of 50 IU/kg. The primary efficacy endpoint was annualized bleeding rate (ABR).
A total of 69 patients were enrolled (<6 years, n = 35; 6-12 years, n = 34) and mean exposure to omfiloctocog alfa was 78.9 days. Mean half-life was 6.7 and 10.2 h in children < 6 years and 6-12 years, respectively. Estimated mean ABRs of all patients were 4.05 for overall bleeding episodes and 1.38 for spontaneous bleeding episodes. Of 127 bleeding episodes, the success rate was 92.1%. 39.7% patients did not experience any bleeding episodes and the mean weekly dose of FVIII was 109.1 IU/kg for these patients. 83% bleeding episodes were controlled with ≤2 injections. Adverse reactions occurred in 2.9% of the patients. One 2-year-old patient developed inhibitors after 12 EDs and it resolved with omfiloctocog alfa immune tolerance induction.
Omfiloctocog alfa was efficacious and well tolerated for the prevention and treatment of bleeding in Chinese pediatric PTPs with severe hemophilia A.
奥米伏科特奥法（Omfiloctocog alfa），中国首个自主研发的重组凝血因子 VIII（FVIII）药物，在既往接受过治疗的（PTP）患者中预防出血的疗效和安全性在中国已得到验证，这些患者年龄≥12 岁，患有严重的血友病 A。
研究奥米伏科特奥法在中国儿科 PTP 中治疗严重血友病 A 的疗效、安全性和药代动力学（PK）。
PTP 患者（中国患者<6 岁者需有≥50 个暴露日，6-12 岁者需有≥150 个暴露日）每两天或每周三次接受奥米伏科特奥法 25-50IU/kg 治疗，疗程 24 周。单次给予 50IU/kg 后进行 PK 评估。主要疗效终点是年化出血率（ABR）。
共纳入 69 例患者（<6 岁者 35 例，6-12 岁者 34 例），奥米伏科特奥法的平均暴露时间为 78.9 天。<6 岁和 6-12 岁儿童的平均半衰期分别为 6.7 和 10.2 小时。所有患者的平均 ABR 为总体出血事件 4.05，自发性出血事件 1.38。127 次出血事件中，成功率为 92.1%。39.7%的患者未发生任何出血事件，这些患者每周的 FVIII 剂量平均值为 109.1IU/kg。83%的出血事件通过≤2 次注射得到控制。2.9%的患者发生不良反应。1 例 2 岁患者在接受 12 次 ED 治疗后出现抑制剂，用奥米伏科特奥法免疫耐受诱导法得到解决。
奥米伏科特奥法对中国严重血友病 A 的儿科 PTP 患者预防和治疗出血有效且耐受良好。
