A Multicenter Study of Recombinant Factor VI11 (Recombinate): Safety, Efficacy, and Inhibitor Risk in Previously Untreated Patients With Hemophilia A
A Multicenter Study of Recombinant Factor VI11 (Recombinate): Safety, Efficacy, and Inhibitor Risk in Previously Untreated Patients With Hemophilia A
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重组因子 VI11（重组）的多中心研究：对既往未经治疗的 A 型血友病患者的安全性、有效性和抑制剂风险
DOI：
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发表时间：
2000
期刊：
影响因子：
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通讯作者：
M. Lee
中科院分区：
文献类型：
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作者：
B. Bray;Gomperts;S. Courter;R.;Gruppo;E. Gordon;M. Manco‐Johnson;Shapiro;É.;Scheibel;G. White;M. Lee
1990 年 7 月，重组研究小组启动了重组因子 VIII (r-FVIII) 的前瞻性、开放性研究，以评估其安全性和有效性，并描述未经治疗的 A 型血友病患者 (PUPS) 抑制剂发展的自然史。所有研究对象均患有严重的 FVIII 缺乏症（基线 WIII 水平为正常值的 52%），并且在研究开始前没有血液制品暴露史。第一次r-FVIII输注后，每3个月筛查一次血浆中的抑制剂，并且每6个月至少测定一次输注后30分钟和24小时时r-FVIII的血浆回收率。截至 1993 年 5 月，最初参加试验的 79 名患者中有 73 名继续参加。接受过至少一次 r-FVIII 输注的 71 名受试者的 r-FVIII 暴露天数中位数为 11。总共进行了 1,785 次输注，以治疗 810 起出血事件。百分之九十二的出血事件对一两次输注有预期的反应。二、非复发性、急性不良反应的发生符合HE研究设计。这是 r-FVIII（重组）的开放标记研究。入组后，患者根据需要接受急性出血发作或预防出血的治疗。记录每次新出血事件的出血位置、出血类型和症状出现时间。每次输注 r-FVIII 时都会监测生命体征、其他不良体征或症状以及治疗的临床反应的显着变化。针对特定出血并发症的 r-FVIII 给药时间表和剂量由每个参与的血友病治疗中心单独确定，并遵循治疗或预防血友病 A 相关出血的普遍接受的指南。
In July 1990, the Recombinate Study Group initiated a prospective, open-labeled investigation of recombinant factor Vlll (r-FVIII) to assess its safety and efficacy and to character- ize the natural history of inhibitor development in previously untreated patients (PUPS) with hemophilia A. All study subjects have severe FVlll deficiency (baseline Wlll level 52% of normal) and no history of blood product exposure before study entry. Following the first r-FVIII infusion, plasma was screened for inhibitors once every 3 months, and plasma recovery of r-FVIII at 30 minutes and 24 hours postinfusion was assayed at least once every 6 months. As of May 1993, 73 of 79 patients originally enrolled in the trial continue to participate. The median number of r-FVIII exposure-days for the 71 subjects who have received at least one r-FVIII infusion is 11. A total of 1,785 infusions have been administered to treat 810 bleeding events. Ninety-two percent of bleeding events responded as anticipated to one or two infusions. Two, nonrecurring, acute adverse reactions occurred coinci-HE Study design. This is an open-labeled study of r-FVIII (Recom- binate). Following enrollment, patients were treated on demand for acute bleeding episodes or for the prevention of bleeding. The loca-tion, type of hemorrhage, and the time of onset of symptoms were recorded for each new bleeding event. Each infusion of r-FVIII was monitored for significant changes in vital signs, other adverse signs or symptoms, and clinical response to treatment. The schedule and dose of r-FVIII administration for a given bleeding complication were individually determined at each participating hemophilia treatment center and followed generally accepted guidelines for the treatment or prevention of hemophilia A-related bleeding.