文摘
摘要目的:评估subject-centered, function-related目标达到情况(t指数)经过反复abobotulinumtoxinA (aboBoNT-A)注射。我们报告更新的数据多达5个周期后,包括长期安全评估结果≤18个月。
背景:AboBoNT-A在美国被批准用于治疗小儿下肢痉挛状态(锁相环)2-17的婴儿。治疗方法和目标需要个性化的根据病人的需要。
设计/方法:这第四阶段的研究收集真实的数据aboBoNT-A临床使用的锁相环,患者年龄2-17。处方决策是由入学前/独立的研究。功能目标是确定在基线患者/父母/照顾者咨询与调查人员。不良事件报告。
结果:这个2nd临时分析包括201名患者,其中78.1% (n = 157)之前收到了肉毒杆菌毒素肉毒毒素治疗。在招生,69.2%的人年龄在2 - 9。平均时间2nd和3理查德·道金斯治疗是24.79(标准差12.38)和42.58(12.85)周,分别。总人口的累积气体t指数为51.60 (9.69)。的最后治疗评估(在这个2nd临时分析),意味着总人口的t指数(N = 201)为48.14 (8.08);BoNT-naive (n = 44) t指数为52.07(3.58)和47.52 (8.47)BoNT-non-naive (n = 157)患者2 - 9岁;t指数为46.66(8.31)和52.07(6.41),分别在10到17岁的病人。在安全人口(N = 243), 44流泪是在26日报告病人;1大多数轻度至中度,严重。肢体疼痛,肢体不适,肌肉肿胀,肌痛报道3例被认为治疗相关。研究没有报道流泪导致停药或死亡。
结论:目标达到情况结果反映了超过预期的成就(t指数略高于50.0)锁相环总人口。AboBoNT-A耐受性良好,流泪的发病率低。这些结果支持aboBoNT-A作为一种有效的治疗选择,积极为锁相环患者风险概要文件。
披露:马克•葛姆雷没有披露。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。Dabrowski博士已经收到个人薪酬在500 - 4999美元的范围为冬至担任顾问。Dabrowski博士已经收到个人薪酬在500 - 4999美元的范围为Alergan担任顾问。Dabrowski博士已经收到个人薪酬在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。Dabrowski博士已经收到个人薪酬在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梅尔兹。Dabrowski博士已经收到个人薪酬在500 - 4999美元的范围在日常的演讲人。Dabrowski博士已经收到个人补偿的范围为0 - 499美元在艾尔建的演讲人。Dabrowski博士已经收到个人薪酬在500 - 4999美元的范围在冬至的演讲人。 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. 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 on a Speakers Bureau for LSUHSC at New Orleans. 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 nothing to disclose. Pascal Maisonobe has received personal compensation for serving as an employee of ipsen pharma. Dr. Wietek has received personal compensation for serving as an employee of Ipsen.
