Myelomeningocele Repair Randomized Trial-DSCC

自1997年以来，采用胎儿开放手术治疗180例胎儿脊髓脊膜膨出(MMC)宫内闭合症。初步 临床证据表明，这种手术减少了分流依赖型脑积水的发生率，并恢复了 小脑和脑干恢复到更正常的形态。然而，胎儿外科手术治疗MMC的临床结果是基于 与历史对照进行比较，只检查疗效而不检查安全性。 脊髓脊膜膨出修复术的随机试验是一项多中心、随机、随机的临床试验 患者将在加州大学旧金山分校的三个胎儿外科单元(FSU)进行， 费城儿童医院和范德比尔特大学医学中心。试验的主要目的是 确定妊娠18~256周胎儿脊髓脊膜膨出宫内修补术是否能改善结局 1)死亡或需要进行脑室减压术一年；2)死亡或贝利精神 发育指数，与标准产后修复相比 这项建议是由乔治华盛顿大学生物统计中心作为数据和研究 MMC维修试验协调中心(DSCC)。DSCC的宗旨是一个重要但独立的 多中心协作研究小组成员，在研究设计、研究实施方面提供专业知识和支持 并进行统计分析。我们将为研究的设计提供科学的领导，并准备最终的研究 文件包括协议、操作手册和病例报告表。DSCC将负责所有 MMC修复试验的宣传，如建立一个中心网站，邮寄医生手册，介绍 在适当的专业会议上提供试验信息，并在医学期刊和患者上投放印刷广告 以出版物为导向。我们还将作为中央转诊网站，让患者了解更多关于试验、进行 初步审查患者的资格，并将患者分配到胎儿外科单元进行最终评估。DSCC 将为患者资格数据维护一个互联网随机系统和基于网络的数据输入系统。我们会 提供全面的数据处理系统，包括中央数据录入、数据库管理和数据质量 控制力。DSCC将使用适当的统计技术进行中期和最终分析。我们将协助 研究人员准备手稿并从研究结果中摘录。 总而言之，我们将参与FSU关于拟议的试验的合作，目的是证明 胎儿干预是否能改善脊柱裂儿童的预后和合理的生活质量。

Since 1997, 180 fetuses have had in utero closure of myelomeningocele (MMC) by open fetal surgery. Preliminary clinical evidence suggests that this procedure reduces the incidence of shunt-dependent hydrocephalus and restores the cerebellum and brainstem to more normal configuration. However, clinicalresults of fetal surgery for MMC are based on comparisons with historical controls and examine only efficacy and not safety. The Myelomeningocele Repair Randomized Trial is a multi-centerunblinded randomized clinical trial of 200 patients that will be conducted at three Fetal Surgery Units (FSU), the University of Califomia-San Francisco, Children's Hospital of Philadelphia, and Vanderbilt University Medical Center. The primary objective of the trial is to determine if intrauterine repair of fetal myelomeningocele at 18¿to 256 weeks gestation improves outcome, as measured by 1) death or the need for ventricular decompressive shunting by one year of life and 2) death or Bayley Mental Development Index, as compared to standardpostnatal repair This proposal is for the George Washington University Biostatistics Center to serve as the Data and Study Coordinating Center (DSCC) for the MMC Repair Trial. The purpose of the DSCC, an important but independent member of the multi-centercollaborative study group, is to provide expertise and support in study design, study conduct and statistical analysis. We will provide scientific leadership in the design of the study and prepare the final study documents includingthe protocol, manual of operations and case report forms. The DSCC will be responsible for all publicity for the MMC Repair Trial such as establishing a central web site, mailing of physician brochures, presenting trial information at appropriate professional meetings and placing print advertisements in medicaljournals and patient oriented publications. We will also serve as the central referral site for patients to learn more about the trial, conduct preliminary review of patient eligibility and assign the patient to a Fetal Surgery Unit for final evaluation. The DSCC will maintain an Internetrandomization system and web-based data entry system for the patient eligibility data. We will provide a comprehensive data processing system including central data entry, data base management and data quality control. The DSCC will use appropnate statisticaltechniques to conductinterim and final analyses. We will assist the investigators in preparationof manuscriptsand abstracts from studyresults. In summary, we will participate in cooperation with the FSUs on the proposed trial with the goal of demonstrating whether fetal intervention offers improvedoutcome with a reasonable quality- of life for spina bifida children.