文摘
摘要目的:分析的有效性和安全性丙种球蛋白IgPro10 (CSL贝林)2 CIDP的试验。
背景:IgPro10(最近在美国批准CIDP)进行的一项小型研究(表面上),随后在较大的路径研究。
设计/方法:表面上是一个潜在的、非盲、单臂研究28 CIDP患者(n = 13 IVIG-pretreated;n = 15未经处理的)调查的有效性和安全性IgPro10感应(2 g / kg)和维持治疗(1克/公斤每3周21周)。这个方案也被用于207 IVIG-pretreated患者在10 - 13周pre-randomization阶段的路径研究(随机化前皮下免疫球蛋白维持治疗或安慰剂)。这两项研究调查了1点降低炎性病变造成和治疗(INCAT)残疾调整分数作为响应参数,和评价的变化意味着握力和医学研究委员会(MRC)得分。治疗诱发不良事件(AEs)进行评估。
结果:反应率为76.9%(95%可信区间[CI]: 49.7 - -91.8)的表面上(IVIG-pretreated患者)在21周,72.9%(95%置信区间:66.5—-78.5)路径在13周;首先INCAT响应的平均时间为3.0和3.7周,分别。中位数(Q1;第三季度)改善措施结果(基线持续观察)的预处理的病人和路径,分别是:INCAT,−2.0 (−3.0;−1.0)和1.0−−2.0;0.0)分;握力,5.0(−9.0;22.0)和9.4 (1.3,18.8)kPa;和MRC得分,5.0(3.0,10.0)和3.0(0.0,6.0)点。首席安全人口(n = 28), 108 AEs发生在22例(78.6%)患者(0.417 /注入);284年100名(48.3%)患者AEs(0.175 /注入)据报道在道路安全人口(n = 207)。头痛是最常见的AE。有因果联系严重AEs表面上和路径发生在2和7例,分别。
结论:类似的功效IgPro10 CIDP的结果中观察到的和路径pre-randomization时期。IgPro10耐受性良好,临床有意义的改善CIDP患者的运动能力。
研究支持:CSL贝林
披露:Mielke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GmbH或其他活动。Van Schaik博士已经收到了个人在一篇社论中补偿能力Cochrane神经肌肉疾病组。Van Schaik博士已经收到了来自中超贝林研究支持;巴克斯特。分类帐博士已经收到个人赔偿咨询、担任科学顾问委员会说,与Baxalta或其他活动;CSL贝林;Kedrion;局部反馈;诺华;的联合; Terumo-BCT; Pfizer. Dr. Leger has received personal compensation in an editorial capacity for Masson-Elsevier; Wiley Blackwell; Wolters Kluwer. Dr. Leger has received research support from CSL Behring; LFB; Novartis. Dr. Bril has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Octapharma, Grifols, CSL, UCB. Dr. van Geloven has received research support from CSL Behring. Dr. Hartung has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biogen Idec, GeNeuro, Sanofi Genzyme, Merck, Novartis Pharmaceuticals, Octapharma, Opexa Therapeutics, Teva Pharmaceuticals, MedImmune, Bayer HealthCare, Forward Pharma, and Roche. Dr. Lewis has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with CSL Behring; Axelacare; Pharnext; Biotest; Kedrion; Nufactor; Optioncare; Grifols. Dr. Lewis has received research support from Neutralis; Cytokinetics. Dr. Sobue has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Mitsubishi Tanabe Pharma Co; Shionogi Co Ltd; Bristol Myers Squibb; Sumitomo Dainippon Pharma Co Ltd; Novartis Pharma KK; Bayer Yakuhin Ltd; Pfizer Japan Inc; Boehringer Ingelheim Japan Inc; Kissei Pharmaceutical Co Ltd; Janssen Pharmaceutical KK; Teijin. Dr. Lawo has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with CSL Behring GmbH. Dr. Durn has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with CSL Behring. Dr. De Bleecker has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Pfizer;Sanofi-Genzyme; CSL Behring. Dr. Cornblath has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Acetylon Pharmaceuticals Inc; Alcobra Pharma; Alnylam Pharmaceuticals; Annexon Biosciences; Akros Pharma; Biotest; Boehringer Ingelheim; Cigna Health Management Inc; CSL Behring; DP Clinical Inc; GLAXOSMITHKLINE Plc; Grifols S.A.; Hansa Medical Inc; Karos. Dr. Cornblath has received royalty, license fees, or contractual rights payments from Acetylon Pharmaceuticals Inc; AstraZeneca Pharmaceuticals LP; Calithera Biosciences; Genentech Inc; Neurocrine Biosciences; Merrimack Pharmaceuticals Inc; Seattle Genetics, Inc; Shire Development, LLC. Dr. Sommer has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Alnylam; Baxalta; CSL Behring; Genzyme; Grifols; Kedrion; Pfizer; UCB. Dr. Sommer has received research support from Kedrion. Dr. Robberecht has received research support from CSL Behring. Dr. Saarela has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with CSL Behring; Sanofi Genzyme. Dr. Kamienowski has nothing to disclose. Dr. Stelmasiak has nothing to disclose. Dr. Tackenberg has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Bayer Healthcare, Biogen, CSL Behring, GRIFOLS, Merck Serono, Novartis, Octapharma, Roche, Sanofi Genzyme, TEVA und UCB Pharma. Dr. Tackenberg has received personal compensation in an editorial capacity for Frontiers Neurology. Dr. Tackenberg has received research support from Biogen, Novartis, Merck. Dr. Merkies has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Talecris. Dr. Merkies has received personal compensation in an editorial capacity for Journal of Peripheral Nervous System.
