Yang Renchi, Zhao Yongqiang, Wang Xuefeng, Sun Jing, Wu Runhui, Jin Chenghao, Jin Jie, Wu Depei, Rendo Pablo, Sun Feifei, Rupon Jeremy, Huard Francois, Korth-Bradley Joan M, Xu Lihong, Luo Binyu, Liu Yingxue Cathy
Thrombosis and Hemostasis Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, People's Republic of China.
Department of Hematology, Peking Union Medical College Hospital, Beijing, People's Republic of China.
J Blood Med. 2020 Nov 25;11:439-448. doi: 10.2147/JBM.S241605. eCollection 2020.
Moroctocog alfa albumin-free cell culture (AF-CC) increases plasma levels of factor VIII (FVIII) activity and, in China, is indicated for the control and prevention of bleeding episodes in patients with hemophilia A. This study aimed to evaluate the efficacy, safety, and recovery data of moroctocog alfa (AF-CC) in patients with hemophilia participating in two open-label studies, both conducted in China.
The authorization study (clinicaltrials.gov identifier NCT00868530) enrolled patients aged ≥6 years, previously treated with ≥1 exposure day of FVIII replacement therapy. The real-world study (clinicaltrials.gov identifier NCT02492984) enrolled patients of any age who were previously untreated or requiring surgical prophylaxis. In both studies, on-demand treatment was administered over 6 months. Key assessments included response to treatment, FVIII inhibitor development, and recovery.
In the authorization study (N = 53; mean age, 23.2 years; severe hemophilia, 23%), response was excellent/good for 90% of infusions at 24 hours. Seven patients developed inhibitors. Mean (SD) FVIII recovery at the initial and final visits was 1.77 (0.50) and 1.67 (0.45) (IU/dL)/(IU/kg), respectively. In the real-world study (N = 85; mean age, 9.5 years; severe hemophilia, 58%), response was rated as excellent or good for most (87%) on-demand infusions and for all surgical prophylaxis patients (n = 14). Seven patients developed FVIII inhibitors. Mean (SD) FVIII recovery at the initial and final visits was 1.71 (0.50) and 1.68 (0.31) (IU/dL)/(IU/kg), respectively. No new safety signals were observed in either study.
On-demand treatment and surgical prophylaxis with moroctocog alfa (AF-CC) is safe and effective for both previously treated and previously untreated Chinese patients with hemophilia A.
重组人凝血因子VIII无白蛋白细胞培养物（AF-CC）可提高血浆中凝血因子VIII（FVIII）的活性水平，在中国，其被用于控制和预防A型血友病患者的出血发作。本研究旨在评估在中国进行的两项开放性研究中，重组人凝血因子VIII（AF-CC）在血友病患者中的疗效、安全性及恢复数据。
授权研究（clinicaltrials.gov标识符NCT00868530）纳入年龄≥6岁、既往接受过≥1个暴露日FVIII替代治疗的患者。真实世界研究（clinicaltrials.gov标识符NCT02492984）纳入任何年龄、既往未接受治疗或需要手术预防的患者。在两项研究中，按需治疗均持续6个月。主要评估指标包括治疗反应、FVIII抑制剂的产生及恢复情况。
在授权研究中（N = 53；平均年龄23.2岁；重度血友病患者占23%），24小时时90%的输注反应为优/良。7例患者产生了抑制剂。首次和末次访视时FVIII的平均（标准差）回收率分别为1.77（0.50）和1.67（0.45）（IU/dL）/（IU/kg）。在真实世界研究中（N = 85；平均年龄9.5岁；重度血友病患者占58%），大多数（87%）按需输注以及所有手术预防患者（n = 14）的反应被评为优或良。7例患者产生了FVIII抑制剂。首次和末次访视时FVIII的平均（标准差）回收率分别为1.71（0.50）和1.68（0.31）（IU/dL）/（IU/kg）。两项研究均未观察到新的安全信号。
对于既往接受过治疗和未接受过治疗的中国A型血友病患者，重组人凝血因子VIII（AF-CC）按需治疗及手术预防均安全有效。
