文摘
摘要目的:调查的有效性、安全性和耐受性每周SCIG IgPro20超过24周的慢性炎性脱髓鞘多神经病(CIDP)。
背景:皮下IgPro20 (Hizentra®, CSL贝林)是有效的在CIDP的安全性为0.2克/公斤和0.4克/公斤剂量每周(路径研究)。我们进行了48周多中心、非盲研究未来的扩展路径调查长期治疗。
设计/方法:患者开始0.4克/公斤每周和切换到0.2克/公斤每周24周后。在恶化,0.4克/公斤是重新启动。在后面研究修正案患者开始每周0.2克/公斤与剂量增加疾病复发定义为至少1点恶化的调整INCAT总分与基线相比。
结果:八十二例患者登记172路径。六十二例最初收到0.4克/公斤,20科目每周0.2克/公斤。复发发生在47.9%的0.2克/公斤,9.7%的受试者0.4克/公斤。复发率不同从4.8到0.4克/公斤组中13.6%和40.0 - 50.0% 0.2克/公斤组,根据以前的治疗路径。剂量减少后从0.4克/公斤到0.2克/公斤,52%的病人恶化(32%的人完成了路径没有复发剂量)其中89%改善后的重启0.4克/公斤剂量。百分之七十六的受试者(62/82)有180个AEs。绝大多数(93%)的AEs在两个治疗组是轻度或中度没有相关的严重不良事件。
结论:IgPro20被证明是有效的CIDP的长期维持治疗。受试者持续皮下IgPro20 0.4克/公斤每周从路径很少复发。大部分受试者完成了路径研究0.4克/公斤可以down-titrated 0.2克/公斤没有进一步恶化。在道路安全结果相似。
披露:Van Schaik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,巴克斯特。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荷兰的研究支持组织科研、荷兰王子比阿特丽克斯溺爱。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。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。范博士Geloven已经收到了来自中超贝林研究支持。哈博士已经收到个人赔偿咨询、担任科学顾问委员会说,与拜耳医疗或其他活动,生原体,Geneuro, Genzyme,落实的默克公司Receptos Celgene公司,诺华,罗氏,CSL贝林。刘易斯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,Axelacare, Pharnext,生物监测,Kedrion, NuFactor Optioncare, Grifols。刘易斯博士研究支持Neuraltus和Cytokinetics接收。 Dr. Sobue has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Mitsubishi Tanabe Pharma Co; Takeda Pharmaceutical Company Limited. Dr. Lawo has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with CSL Behring GmbH. Dr. Praus 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. Cornblath has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Acetylon Pharmaceuticals Inc., Alnylam Pharmaceuticals, Annexon Biosciences, Akros Pharma, argenx BVBA, Biotest Pharmaceuticals, Inc., Boehringer Ingelheim, Cigna Health Management, Inc., CSL Behring, DP Clinical, Inc., Grifols S.A., Hansa Medical Inc., Karos Pharmaceuticals, Inc., Merrimack Pharmaceuticals, Inc., Neurocrine Biosciences, Novartis Corp., Octapharma AG, Pharnext SAS, Seattle Genetics, Inc., Sun Pharmaceuticals, Syntimmune. Data Safety Monitoring Board: Pfizer Inc., Ionis Pharmaceuticals, Axovant Sciences LTD., Ampio Pharmaceuticals, PledPharma, Momenta Pharma, Sanofi, GBS CIDP Foundation International. Dr. Cornblath has received personal compensation in an editorial capacity for JPNS. Dr. Cornblath has received compensation The Peripheral Nerve Society. Dr. Cornblath has received royalty, license fees, or contractual rights payments from Acetylon Pharmaceuticals Inc., Akros Pharma, AstraZeneca Pharmaceuticals, LP, Calithera Biosciences, Genentech Inc, Karos Pharma, Neurocrine Biosciences, Merrimack Pharmaceuticals, Inc. Seattle Genetics, Inc., Shire Development, LLC. 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 the Peripheral Nerve Society.
