Myelomeningocele Repair Randomized Trial-DSCC

自1997年以来，180例胎儿在子宫内通过开放式胎儿手术闭合脊髓脊膜膨出（MMC）。初步 临床证据表明，该手术降低了分流依赖性脑积水的发生率， 小脑和脑干恢复到更正常的形态。然而，胎儿手术治疗MMC的临床结果是基于 与历史对照进行比较，仅检查有效性而非安全性。 脊髓脊膜膨出修复随机试验是一项多中心非盲随机临床试验， 将在三个胎儿外科单位（FSU），加利福尼亚大学-旧金山弗朗西斯科， 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 确定在妊娠18 - 256周时对胎儿脊髓脊膜膨出进行宫内修复是否能改善结局， 根据1）死亡或需要心室减压分流术，根据1年寿命和2）死亡或Bayley Mental 发育指数，与标准产后修复相比 本提案由乔治华盛顿大学生物统计中心作为数据和研究中心 MMC修复试验协调中心（DSCC）。DSCC的目的是一个重要但独立的 作为多中心协作研究小组的成员，在研究设计、研究实施、 和统计分析。我们将在研究设计中提供科学领导，并准备最终研究 文件包括方案、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， 在适当的专业会议上提供试验信息，并在医学期刊和患者杂志上刊登印刷广告 定向出版物。我们还将作为患者的中心转诊站点，以了解更多有关试验的信息， 对患者资格进行初步审查，并将患者分配到胎儿手术室进行最终评估。DSCC 将为患者资格数据维护互联网化系统和基于网络的数据输入系统。我们将 提供全面的数据处理系统，包括中央数据输入、数据库管理和数据质量 控制DSCC将使用适当的检验技术进行检验和最终分析。我们将协助 研究者在研究结果的手稿和摘要的解释中。 总之，我们将参与与FSU就拟议试验的合作，目的是展示 胎儿干预是否能改善脊柱裂患儿的预后并提高其生活质量。

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.