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: 7568028
• 负责人: JOHN W. BROCK
• 金额: $ 0.65万
• 依托单位: VANDERBILT UNIVERSITY
• 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/7568028
• 关键词: Academic Medical Centers; Affect; Age; Age-Months; Age-Years; Brain Stem; California; Cerebellum; Cesarean section; Cessation of life; Child; Clinical; Clinical Trials; Closure; Cognitive; Condition; Consent; Data; Diagnosis; Enrollment; Fetus; Home environment; Hydrocephalus; Incontinence; Infant; Infant Development; Intervention; Lesion; Life; Lower Extremity; Magnetic Resonance Imaging; Measures; Meningomyelocele; Mothers; Motor; Movement; Multicenter Trials; Neonatal; Nervous System Physiology; Neurological outcome; Numbers; Open Spina Bifida; Operative Surgical Procedures; Orthopedic Procedures; Outcome; Parents; Patients; Pediatric Hospitals; Philadelphia; Pregnancy; Procedures; Psychological Tests; Quality of life; Questionnaires; Randomized; Randomized Controlled Clinical Trials; Reflex action; Risk; Safety; San Francisco; Shunt Device; Spastic; Spinal Dysraphism; Standards of Weights and Measures; Universities; Ventricular; Visit; Washington; Week; base; cognitive function; evaluation/testing; fetal; fetus surgery; improved; in utero; indexing; malformation; mental development; muscle strength; neonatal morbidity; postnatal; psychologic; psychosocial; repaired; reproductive; reproductive function; urinary

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 incidenceof shunt-dependent hydrocephalus and restores the cerebellum and brainstem to more normalconfiguration. However, clinical results of fetal surgery for MMC are based on comparisons with historical controlsand examineonly efficacy and not safety. The Myelomeningocele Repair RandomizedTrial is a multi-center unblindedrandomized clinical trial of 200 patients that will be conducted at three Fetal SurgeryUnits (FSU), 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, the George Washington UniversityBiostatisticsCenter. The primary objective of the trial is to determine if inrrauterine 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 postnatalrepair Consenting patients who satisfy eligibilitycriteria will be centrallyrandomizedto either intrauterineor standard postnatal repair of the MMC. Patientsassigned to the fetal surgery group will be discharged to nearby accommodation following surgery, unless infeasible, in which case they will return to their assigned FSU at 32^ weeks gestation for delivery at 3?0 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 deliveryby 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, musclestrength,ambulation status and urinary and fecal continence. Infants will make followup visits at twelve and thirty monthscorrected age for detailed neurbmotor 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 will be able to demonstrate whether fetal intervention offers improved outcome with a reasonable quality of life for spina bifida children.

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， 小脑和脑干恢复到正常形态。然而，MMC胎儿手术的临床结果是基于 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 这将在三个胎儿外科单位（FSU），加州大学旧金山分校弗朗西斯科，儿童医院 和范德比尔特大学医学中心，沿着独立的数据和研究协调中心 中心，乔治华盛顿大学生物制药中心。试验的主要目的是确定 妊娠18至25周时胎儿脊髓脊膜膨出的子宫内修复可改善结局，通过1）死亡来衡量 或需要心室减压分流术的1年生命和2）死亡或贝利精神发育指数， 与标准产后修复相比， 符合致死性标准的患者将被集中随机分配到子宫内或标准 MMC的产后修复。被分配到胎儿手术组的病人将被安排到附近的病房 手术后，除非不可行，在这种情况下，他们将在妊娠32周时返回分配的FSU， 三点送货？妊娠0周剖宫产。分配到产后手术组的患者将回家 37周时，回到指定的FSU进行剖宫产。磁共振成像将是 在入组、出院或足月妊娠时以及一岁时进行，以确定宫内修复术是否改善了 基亚里II畸形的程度。新生儿发病率将被记录， 与MMC、肌肉力量、行走状态以及大小便失禁相关的疾病。婴儿会使 在12个月和30个月的矫正年龄进行随访，进行详细的神经运动功能分析，认知测试， 评估神经发育状况。此外，长期的社会心理和生殖后果， 母亲将接受评估。 总之，拟议的试验将能够证明胎儿干预是否提供了改善的结果， 脊柱裂儿童的合理生活质量。