Hematology Department, Haemophilia and Thrombosis Center, San Bortolo Hospital, Vicenza, Italy.
Department of Hematology/Oncology, Cleveland Clinic, Cleveland, OH, USA.
Haemophilia. 2020 May;26(3):450-458. doi: 10.1111/hae.13980. Epub 2020 Apr 15.
Turoctocog alfa pegol is a glycoPEGylated recombinant factor VIII (FVIII) with an extended half-life developed for prophylaxis, treatment of bleeds and perioperative management in patients with haemophilia A.
Evaluate the efficacy and safety of turoctocog alfa pegol treatment for major and minor surgeries in the pathfinder 3 and 5 phase III trials.
Adults/adolescents aged ≥12 years with severe haemophilia A (FVIII <1%) received perioperative turoctocog alfa pegol treatment planned to achieve FVIII activity levels >80% during major surgery (pathfinder 3). The primary end point was haemostatic efficacy during surgery; secondary end points were blood loss, haemostatic effect postsurgery, consumption, transfusions, safety and health economics. Children (0-11 years) undergoing minor surgeries received 20-75 IU/kg turoctocog alfa pegol at Investigator's discretion (pathfinder 5).
pathfinder 3 included 35 patients undergoing 49 major surgeries. Haemostasis was successful in 47/49 (95.9%) surgeries; two had moderate haemostatic responses. Median (mean) blood loss during major surgery was 75 (322.6) mL. Four bleeds were reported postsurgery; three were successfully treated with turoctocog alfa pegol (one was not evaluated). On the day of surgery, overall mean (median) dose was 75.5 (74.5) IU/kg and mean (median) number of doses was 1.7 (2.0). Five procedures required 11 transfusions on the day of surgery or days 1-6. No safety concerns or inhibitors were identified. Forty-five minor surgeries in 23 children were performed without complications.
Turoctocog alfa pegol was effective for perioperative haemostatic management of major and minor surgeries in patients across age groups with severe haemophilia A.
特立帕肽聚乙二醇是一种延长半衰期的重组因子 VIII（FVIII），专为血友病 A 患者的预防、出血治疗和围手术期管理而开发。
评估特立帕肽聚乙二醇在探索者 3 期和 5 期 III 期临床试验中用于大、小手术的疗效和安全性。
年龄≥12 岁的重度血友病 A（FVIII<1%）患者接受围手术期特立帕肽聚乙二醇治疗，计划在大手术期间使 FVIII 活性水平>80%（探索者 3 期）。主要终点是手术期间的止血效果；次要终点是手术期间和手术后的失血量、止血效果、消耗、输血、安全性和卫生经济学。接受小手术的儿童（0-11 岁）由研究者酌情给予 20-75IU/kg 特立帕肽聚乙二醇（探索者 5 期）。
探索者 3 期包括 35 名患者进行的 49 例大手术。47/49（95.9%）手术止血成功；2 例出现中度止血反应。大手术期间的中位（平均）失血量为 75（322.6）mL。术后报告了 4 例出血；3 例用特立帕肽聚乙二醇成功治疗（1 例未评估）。手术当天，总体平均（中位数）剂量为 75.5（74.5）IU/kg，平均（中位数）剂量数为 1.7（2.0）。5 例手术当天或第 1-6 天需要 11 次输血。未发现安全性问题或抑制剂。23 名儿童的 45 例小手术均无并发症。
特立帕肽聚乙二醇可有效用于各年龄段重度血友病 A 患者的大、小手术围手术期止血管理。
