文摘
摘要目的:目的:评价疗效、安全性和耐受性的皮下免疫球蛋白(SCIg)治疗重症肌无力(MG)患者丙种球蛋白作为常规clinicalcare的一部分。
背景:背景:丙种球蛋白已被证实能改善量化毫克(评分)得分相比安慰剂相比,在另一项研究中有效血浆置换。在日常保健,SCIg可能比丙种球蛋白更容易管理。
设计/方法:方法:多中心北美开放标签前瞻性研究者发起的研究有两部分:丙种球蛋白筛选阶段(ISP;周10−−1)紧随其后的实验处理阶段(ETP;周0到12)。我们假设65%以上的患者进入ETP评分会稳定在第12周(主要结果)。我们招募了23个患者在ISP和22进入ETP。12/22(54.5%)的女性,18(78%)白色,与平均年龄17±51.4年。我们有完整的ETP评分数据19/22;一个主题退出ISP由于恶化条件和两个主题周4前退出(针不喜欢)。每个协议主要统计分析是进行n = 22对象使用一个片面的z检验比例在5%的显著性水平。敏感性分析进行了使用一个队列(n = 22科目使用“最坏”归罪场景以及事后分析。
结果:以初级分析,发现:19/22 (86.4%;95%置信区间:0.72—-1.00)治疗“成功”(p = 0.018)。灵敏度分析使用“最坏”归罪导致17/22(77.3%,0.60 - -0.95)声明为治疗成功(p = 0.114)。事后的分析主要结果证实治疗成功的17/20 (85%,0.69 - -1.00)(p = 0.0304)。在二级结果没有区别措施虽然MG复合在第12周的更好。人口SCIg是安全,耐受性良好。
结论:结论:大多数MG患者做好丙种球蛋白保持疾病稳定一旦转变SCIg 12周。
披露:Dimachkie博士已经收到个人赔偿咨询、担任科学顾问委员会说,或其他活动Alnylam、Audentes, Biomarin,催化剂,CSL-Behring, Genzyme, Mallinckrodt,动量,诺华,NuFactor, Octapharma, RMS医疗、赛诺菲安万特,夏尔和作秀的成分。Dimachkie博士已经收到Alexion研究支持,Alnylam、法庭之友,Biomarin,百时美施贵宝,催化剂,CSL-Behring, FDA /门诊部当,葛兰素史克公司基因泰克,Grifols, MDA, NIH,诺华公司Genzyme, Octapharma Viromed和蓝玉UCB生物制药。成Bril博士已经收到个人赔偿咨询、担任科学顾问委员会说,与中超贝林或其他活动,联合,Alnylam、Alexion, Grifols, Octapharma,夏尔,辉瑞和Bionevia。成Bril博士已经收到了来自中超贝林研究支持,联合,Alnylam、Alexion, Grifols, Octapharma,夏尔,Bionevia。莱文博士已经收到个人赔偿咨询、担任科学顾问委员会说,与玩家或其他活动,Alexion, Nufactor,外交官,夏尔,科林斯的参考实验室。莱文博士持有股票或股票期权在科林斯的参考实验室。莱文博士已经收到Alnylam制药和Malinckrodt研究支持。Trivedi博士已经收到个人赔偿咨询、担任科学顾问委员会说,与赛诺菲安万特或其他活动。Trivedi博士已经收到赛诺菲的研究支持,CSL贝林。Silvestri博士已经收到个人赔偿咨询、担任科学顾问委员会说,与Alexion制药和OptionCare或其他活动。 Dr. Phadnis has nothing to disclose. Dr. Saperstein has nothing to disclose. Dr. Nations has received research support from Catalyst Pharmaceutical. Dr. Katzberg has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with CSL Behring, Amazentis, Flexpharma, Momenta, Octapharma, Terumo, Grifols, and Sanofi Genzyme. Dr. Katzberg has received research support from CSL Behring, Octapharma and Grifols. Dr. Wolfe has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Grifols, Shire, and Alexion. Dr. Wolfe has received research support from ArgenX, Ra, and Immunovant. Dr. Herbelin has nothing to disclose. Dr. Higgs has nothing to disclose. Dr. Heim has nothing to disclose. Dr. McVey has nothing to disclose. Dr. Rico has nothing to disclose. Dr. Statland has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Acceleron Pharma, Inc., Expansion Therapeutics, , Fulcrum Therapeutics, Genea Biocells, and Strongbridge Biopharma. Dr. Statland has received research support from FSH Society, Muscular Dystrophy Association, and the National Institutes of Health. Dr. Barohn has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with NuFACTOR Specialty Pharmacy. Dr. Barohn has received research support from Alexion, BioMarin, Cytokinetics, Eli Lilly, Ionis, Neuraltus, Novartis, PTC Therapeutics, Sanofi/Genzyme, Sarepta, Teva. Dr. Barohn has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with NuFACTOR Specialty Pharmacy. Dr. Barohn has received research support from Alexion, BioMarin, Cytokinetics, Eli Lilly, Ionis, Neuraltus, Novartis, PTC Therapeutics, Sanofi/Genzyme, Sarepta, Teva. Dr. Pasnoor has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with TerumoBCT, Alexion Pharmaceuticals and Momenta Pharmaceuticals.
