文摘
摘要目的:这项前瞻性研究评估了纵向实现以病人为中心,function-related目标达到情况量表(气)t指数经过反复abobotulinumtoxinA (aboBoNT-A)注射≤30个月在美国(≤10周期)。
背景:针对广泛的演讲和患者需求,越来越认识到痉挛状态的治疗需要个性化,包括评估的治疗目标。
设计/方法:符合条件的患者年龄2-17锁相环是从调查人员的招募临床实践。处方决策研究是独立招生。为每个注入气体t指数评估周期和目标可以界定在每个注射访问;分数≥50反映目标的成就。不良事件(AEs)报道。
结果:210名患者的有效性人口,77.6% (n = 163)以前接受肉毒神经毒素。可用的粗大运动功能分类系统(GMFCS)水平显示,31.3%(61/195)的患者non-ambulatory (GMFCS IV和V)。意思是累积气体t指数为51.1 (SD±9.3)。总体而言,75.2%的患者达到他们的主要目标。所有周期,肌肉注射的平均数量范围从5.5(±2.9)到7.0 (±3.7);注入点的平均数量范围从8.1(±2.7)到9.9(±5.7),腓肠肌肌肉注射是最常见(85.7%)。注入指导技术被用于在周期1 - 6 > 70%的患者;电刺激是最频繁的(> 50%)。在人口安全,n = 102/242(42.1%)报告了392治疗诱发的AEs,一般轻度至中度。总共35 AEs n = 15/242例(6.2%)被认为治疗相关。
结论:总的来说,目标是实现在大多数病人或好于预期。AboBoNT-A耐受性良好,治疗相关的AEs的发病率低。这些结果证实aboBoNT-A是一种有效的治疗选择,积极为锁相环风险概要文件。
披露:马克•葛姆雷已收到个人薪酬在500 - 4999美元的范围作为顾问日常药品。马克•葛姆雷已收到个人薪酬在500 - 4999美元的范围为爱力根制药担任顾问。马克•葛姆雷已收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会日常药品。马克•葛姆雷已收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会爱力根制药。马克•葛姆雷已收到个人薪酬在500 - 4999美元的范围为服务使用者日常药品管理局。马克•葛姆雷已收到个人薪酬在500 - 4999美元的范围服务发言人艾尔建医药管理局。马克•葛姆雷的机构收到日常药品的研究支持。马克•葛姆雷的机构收到爱力根制药的研究支持。Dabrowski博士已经收到个人薪酬在500 - 4999美元的范围为梅尔兹担任顾问。Dabrowski博士已经收到个人薪酬在500 - 4999美元的范围作为日常的咨询顾问服务。 Dr. Dabrowski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Solstice. Dr. Dabrowski has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alergan. Dr. Dabrowski has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ipsen. Dr. Dabrowski has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merz. Dr. Dabrowski has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Ipsen. Dr. Dabrowski has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Allergan. Dr. Dabrowski has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Solstice. Dr. Dabrowski has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alergan. The institution of Dr. Dabrowski has received research support from Ipsen. The institution of Dr. Dabrowski has received research support from Merz. Dr. Delgado has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ipsen. Dr. Delgado has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Allergan. Dr. Delgado has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kashiv. The institution of Dr. Delgado has received research support from Ipsen. Dr. Tilton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ipsen. Dr. Tilton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for allergan. Dr. Tilton has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ipsen. Dr. Tilton has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Allergan. Dr. Tilton has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Ipsen. Dr. Tilton has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Jones Walker law firm - Defense. The institution of Asare Christian has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ipesen Biopharmaceuticals. Asare Christian has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Ipesen Biopharmaceuticals. The institution of Asare Christian has received research support from Ipesen. Sarah Helen Evans has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ipsen. Sarah Helen Evans has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Ipsen. Sarah Helen Evans has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Nicholas Zauzig. Sarah Helen Evans has received intellectual property interests from a discovery or technology relating to health care. Mrs. CALVI-GRIES has received personal compensation for serving as an employee of Atlanstat (Contractor for Ipsen). Dr. Goldberg has received personal compensation for serving as an employee of Ipsen. Dr. Goldberg has received intellectual property interests from a discovery or technology relating to health care.
