Home Treatment of Mild to Moderate Bleeding Episodes Using Recombinant Factor VIIa (Novoseven) in Haemophiliacs with Inhibitors
Home Treatment of Mild to Moderate Bleeding Episodes Using Recombinant Factor VIIa (Novoseven) in Haemophiliacs with Inhibitors
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使用重组因子 VIIa (Novoseven) 使用抑制剂在家治疗血友病患者的轻度至中度出血
DOI：
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发表时间：
1998
影响因子：
6.7
通讯作者：
Nigel S. Key
中科院分区：
文献类型：
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作者：
L. Aledort;D. Beardsley;Herbert Cooper;G. Davignon;B. Ewenstein;G. Gilchrist;J. Gill;B. Glader;W. Hoots;C. Kisker;J. Lusher;C. Rosenfield;A. Shapiro;H. Smith;E. Taft;Nigel S. Key
摘要目标。评估固定剂量的重组活化因子VII（rFVIIa; NovoSeven™）在家庭环境中治疗存在抑制剂的血友病A或B患者的轻度至中度关节、肌肉和皮肤粘膜出血发作的安全性和疗效。设计一项为期一年的多中心、开放标签、单组、III期研究。方法.患者或其护理人员接受多达3剂rFVIIa（90 μg/kg i. v.）在轻度至中度出血发作后8小时内，每隔3小时进行一次。一旦受试者认为rFVIIa在止血方面“有效”（1-3次注射后），则给予另一个（维持）剂量的rFVIIa。结果在入组的60例患者中，56例至少发生1次出血，46例完成了1年研究。根据方案定义，877例出血中的614例（70%）可评价。在平均2.2次注射后，92%（566/614）的可评价出血的止血被评定为“有效”。对于成功治疗的发作，从出血发作至首次注射给药的时间为1.1 ± 2.0 h（平均值± SD）。初步成功缓解后24小时，95%的病例报告止血得以维持。肌肉、关节和靶关节以及粘膜-皮肤出血事件的疗效相当。在对所有877例出血事件进行的意向治疗分析中，疗效结局与可评价出血事件相当，88%的治疗事件有效缓解。治疗相关不良事件发生在32例（占所有事件的3%）出血事件中，其中超过50%（18/32）的事件包括再出血/新发出血。浅表血栓性静脉炎的单次发作是遇到的唯一血栓性并发症，没有患者因不良事件退出。未检出FVII（a）抗体，未报告rFVIIa超敏反应。结论rFVIIa在家庭环境中用于治疗存在抑制剂的血友病A或B患者的轻度至中度出血事件时有效且耐受性良好。
Summary Objective. To assess the safety and efficacy of a fixed dose of recombinant activated factor VII (rFVIIa; NovoSeven™) in the home setting for mild to moderately severe joint, muscle, and mucocutaneous bleeding episodes in patients with haemophilia A or B with inhibitors. Design. Multicentre, open-label, single arm, phase III study of one year duration. Methods. Patients or their caregivers administered up to three doses of rFVIIa (90 μg/kg i.v.) at 3 h intervals within 8 h of the onset of a mild to moderate bleeding episode. Once the subject considered that rFVIIa had been “effective” with regard to haemostasis (after 1-3 injections), one further (maintenance) dose of rFVIIa was administered. Results. Of 60 patients enrolled, 56 experienced at least one bleed, and 46 completed the one year study. 614 of 877 bleeds (70%) were evaluable according to protocol definitions. Haemostasis was rated as “effective” in 92% (566/614) of evaluable bleeds after a mean of 2.2 injections. For successfully treated episodes, the time from onset of bleeding until administration of the first injection was 1.1 ± 2.0 h (mean ± SD). Twenty-four hours after initial successful response, haemostasis was reported as having been maintained in 95% of cases. Efficacy was comparable for muscle, joint and target joint, and muco-cutaneous bleeding episodes. In an intent-to-treat analysis of all 877 bleeding events, efficacy outcomes were equivalent to the evaluable bleeds, with an effective response in 88% of treated episodes. Treatment-related adverse events occurred in 32 (3% of all) bleeding episodes and consisted of re-bleeds/new bleeds in more than 50% (18/32) of these events. A single episode of superficial thrombophlebitis was the only thrombotic complication encountered, and there were no patient withdrawals due to adverse events. Development of FVII(a) antibodies could not be detected, and hypersensitivity reactions to rFVIIa were not reported. Conclusion. rFVIIa is effective and well tolerated when used in the home setting to treat mild to moderate bleeding episodes in patients with haemophilia A or B with inhibitors.
当前美国牙科协会临床实践指南的质量评估。
影响因子：
--
作者：
London,SD;Chamut,S;Fontelo,P;Iafolla,T;Dye,BA
通讯作者：
Dye,BA
人单核细胞支持因子 VIIa 激活因子 X，独立于组织因子：对血友病高剂量因子 VIIa 治疗机制的影响。
影响因子：
20.3
作者：
Hoffman,M;Monroe,DM;Roberts,HR
通讯作者：
Roberts,HR