肌萎缩性脊髓侧索硬化症(ALS) Biorepository可行性研究(P5.094)

文摘

目的:确定的可行性发展中国家ALS biorepository与注册表。背景:加强国家ALS登记处,有毒物质与疾病登记处联合机构(有毒物质)协调试点研究找到最好的方法来收集和储存未来ALS相关研究的生物样本。设计/方法:本研究包括两个样本采集部分:1)家庭收集的血液,尿液,头发,指甲,两次大约分开6个月;和2)后期收集的大脑、脊髓,CSF,骨骼,肌肉,皮肤;参与者可以做两个。这些组件的组合允许全面评价与装配相关国家的挑战,基于biorepository。合格的参与者必须参加全国ALS注册表和同意联系关于研究项目。结果:所有50个州三百三十九人同意捐赠生物标本和330提供标本至少一次。大约18 (percnt)的参与者没有完成第二个画主要是由于死亡和疾病。参与者的年龄范围从31 - 87年和60 (percnt)是男性。 Fifty-four percent of participants lived 50 or more miles from an ALS specialty/referral center. Thirty people consented to postmortem tissue donation from 18 states. Fifteen donations have been completed. The first 11 cases all showed neuropathologically-confirmed ALS with TDP-43 inclusions with a mean age of 66 years (range: 43-76), 55[percnt] female, and mean brain weight of 1260g. The average length of time from study consent to death was eight months. Discussion/Conclusions: Creating a geographically diverse biorepository has unique challenges. However, based on expert input, ATSDR determined that it is feasible to incorporate a biorepository into the National ALS Registry. These specimens will be a valuable resource for researchers who will be able to request specimens for ALS studies. Study Supported by: ATSDR contract #200-2011-40830

披露:凯博士已经收到个人补偿活动与格林咨询公司作为一个员工。瓦格纳女士已经收到个人补偿活动与格林咨询公司作为一个员工。斯坦博士没有披露。梅塔博士没有披露。