MULTICENTER TRIAL OF FETAL MYELOMENINGOCELE REPAIR

胎儿脊髓脊膜膨出修复的多中心试验

• 批准号: 7940506
• 负责人: Diana Lee Farmer
• 金额: $ 57.73万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-03-15 至 2012-09-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7940506
• 关键词: Academic Medical Centers; Affect; Age; Age-Months; Age-Years; Biometry; Brain Stem; California; Cerebellum; Cesarean section; Cessation of life; Child; Clinical; Cognitive; Consent; Data; Diagnosis; Eligibility Determination; Enrollment; Fetus; Home environment; Hydrocephalus; Incidence; Incontinence; Individual; Infant; Infant Development; Intervention; Lesion; Life; Lower Extremity; Magnetic Resonance Imaging; Measures; Meningomyelocele; Mothers; Motor; Movement; Multicenter Trials; Neonatal; Nervous System Physiology; Neurological outcome; Open Spina Bifida; Operative Surgical Procedures; Orthopedic Procedures; Outcome; Parents; Patients; Pediatric Hospitals; Philadelphia; Pregnancy; Procedures; Psychological Tests; Quality of life; Questionnaires; Randomized; Randomized Clinical Trials; Randomized Controlled Clinical Trials; Reflex action; Risk; Safety; San Francisco; Shunt Device; Spastic; Spinal Dysraphism; Universities; Ventricular; Visit; Washington; base; cognitive function; evaluation/testing; fetal; fetus surgery; follow-up; improved; in utero; indexing; malformation; mental development; muscle strength; neonatal morbidity; postnatal; psychologic; psychosocial; repaired; reproductive; reproductive function; urinary

DESCRIPTION (provided by applicant): 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 (FSUs), the University of California-San Francisco, Children s Hospital of Philadelphia, and Vanderbilt University Medical Center, along with an independent Data and Study Coordinating Center (DSCC), the George Washington University Biostatistics Center. The primary objective of the trial is to determine if intrauterine repair of fetal myelomeningocele at 18 to 25 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. Consenting patients who satisfy eligibility criteria will be centrally randomized to either intrauterine or standard postnatal repair of the MMC. Patients assigned to the fetal surgery group will be discharged to nearby accommodation following surgery, unless unfeasible, in which case they will return to their assigned FSU at 32 weeks gestation for delivery at 37 weeks gestation by cesarean section. Patients assigned to the postnatal surgery group will return home and at 37 weeks, return to their assigned FSU for delivery by cesarean section. Magnetic resonance imaging will be conducted at enrollment, discharge or term gestation, and one year of age to determine if intrauterine repair improves the degree of the Chiari II malformation. Neonatal morbidity will be recorded as will the number of surgical procedures for conditions related to the MMC, muscle strength, ambulation status and urinary and fecal continence. Infants will make follow-up visits at twelve and thirty months corrected age for detailed neuromotor function analysis, cognitive testing and evaluation of neurodevelopmental status. In addition, the long term psychosocial and reproductive consequences in mothers will be evaluated. In summary, the proposed trial is expected to demonstrate whether fetal intervention offers improved outcome with a reasonable quality of life for spina bifida children.

描述（由申请人提供）：自 1997 年以来，已有 180 个胎儿在子宫内发育 通过开放胎儿手术闭合脊髓脊膜膨出（MMC）。初步临床 有证据表明，该手术可降低分流依赖性的发生率 脑积水并使小脑和脑干恢复正常 配置。然而，MMC 胎儿手术的临床结果基于 与历史对照进行比较，仅检查疗效而不检查安全性。 脊髓脊膜膨出修复随机试验是一项多中心、非盲法 200 名患者的随机临床试验将于 3 点进行 加州大学旧金山分校胎儿外科中心 (FSU)， 费城儿童医院和范德比尔特大学医学中心， 与独立的数据和研究协调中心 (DSCC) 一起， 乔治华盛顿大学生物统计中心。主要目标 该试验的目的是确定胎儿脊髓脊膜膨出的宫内修复是否可行 妊娠 18 至 25 周改善结局，通过 (1) 死亡或 生命满一年且 (2) 死亡时需要进行心室减压分流 或贝利心理发展指数，与标准产后修复相比。 符合资格标准且同意的患者将被集中 随机接受 MMC 宫内修复或标准产后修复。 分配到胎儿手术组的患者将被出院到附近 手术后的住宿，除非不可行，在这种情况下他们将 在妊娠 32 周时返回指定的 FSU，并在 37 周时分娩 剖腹产妊娠。分配进行产后手术的患者 小组将返回家中，并在第 37 周时返回指定的 FSU 剖腹产分娩。磁共振成像将在 入院、出院或足月妊娠，以及一岁以确定是否 宫内修复术可改善 Chiari II 畸形的程度。 将记录新生儿发病率以及外科手术的数量 用于与 MMC、肌肉力量、行走状态和 尿和大便失禁。婴儿将在十二点和十二点进行随访 三十个月的校正年龄以进行详细的神经运动功能分析， 认知测试和神经发育状态评估。此外， 对母亲的长期社会心理和生殖后果将是 评价。 总之，拟议的试验预计将证明胎儿是否 干预为患者提供了改善的结果和合理的生活质量 脊柱裂儿童。