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，并使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的比较，仅检查疗效而非安全性。脊髓脊膜膨出修复随机试验是一项200例患者的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进行。该试验的主要目的是确定在妊娠18（0）至25（6）周时胎儿脊髓脊膜膨出的宫内修复是否改善了结局，如1）死亡或一年内需要心室减压分流和2）死亡或Bayley精神发育指数，与标准的产后修复相比，该提案是由乔治华盛顿大学生物统计中心作为数据和研究协调中心（DSCC）MMC修复试模。DSCC是多中心协作研究小组的重要但独立的成员，其目的是在研究设计、研究实施和统计分析方面提供专业知识和支持。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MMC修复试验的所有宣传，例如建立中心网站、邮寄医生手册、在适当的专业会议上展示试验信息以及在医学期刊和面向患者的出版物上刊登印刷广告。我们还将作为患者的中心转诊中心，以了解有关试验的更多信息，对患者资格进行初步审查，并将患者分配到胎儿外科进行最终评估。DSCC将为患者资格数据维护互联网随机化系统和基于网络的数据输入系统。我们将提供一个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将使用适当的统计技术进行中期和最终分析。我们将协助研究者准备研究结果的手稿和摘要。总之，我们将与FSU合作开展拟议的试验，目的是证明胎儿干预是否能改善脊柱裂儿童的结局并提高其生活质量。

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, clinical results 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-center unblinded randomized clinical trial of 200 patients that will be conducted at three Fetal Surgery Units (FSU), the University of California-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(0) to 25(6) 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 standard postnatal 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-center collaborative 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 including the 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 medical journals 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 Internet randomization 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 appropriate statistical techniques to conduct interim and final analyses. We will assist the investigators in preparation of manuscripts and abstracts from study results. In summary, we will participate in cooperation with the FSUs on the proposed trial with the goal of demonstrating whether fetal intervention offers improved outcome with a reasonable quality of life for spina bifida children.