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: 7567854
• 负责人: Diana Lee Farmer
• 金额: $ 106.77万
• 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/7567854
• 关键词: Academic Medical Centers; Affect; Age; Age-Months; Age-Years; Biometry; Brain Stem; California; Cerebellum; Cesarean section; Cessation of life; Child; Clinical; Closure; Cognitive; Condition; Consent; Data; Diagnosis; Eligibility Determination; Enrollment; Fetus; Home environment; Hydrocephalus; Image; 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; 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 Clinical Trials; 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 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, along with an independent Data and Study Coordinating Center, the George Washington University Biostatistics 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 '.o 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 infeasible, 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 followup 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 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，通过开放式胎儿手术，有180个胎儿在子宫内脊髓膨出（MMC）闭合。 初步临床证据表明，该过程减少了分流依赖性的事件 脑积水并将小脑和脑干恢复到更正常的构型。但是，临床结果 MMC胎儿手术的基于与历史控制和检查有效性的比较，而不是 安全。 脊髓脑膜静脉维修随机试验是一项多中心的未盲随机临床试验 加利福尼亚大学的200例将在三个胎儿手术单元（FSU）上进行的200例患者 弗朗西斯科，费城儿童医院和范德比尔特大学医学中心 乔治华盛顿大学生物统计学中心的独立数据和研究协调中心。这 该试验的主要目的是确定胎儿脊髓脑膜静脉内的宫内修复是否在18到256周时 妊娠改善了预后，如1）死亡或对心室减压分流的需求 生命年份和2）死亡或贝利心理发展指数，如'.o标准后修复 满足资格标准的同意患者将被集中随机与宫内或 MMC的标准产后修复。分配给胎儿手术组的患者将被出院 手术后的住宿，除非不可行，在这种情况下，他们将返回其分配的FSU，在32€ 剖宫产妊娠37周的妊娠周期。分配到产后手术的患者 小组将返回家园，并在37周回家，返回其分配的FSU剖腹产。磁的 共振成像将在入学，出院或妊娠期进行，并年龄一岁 确定宫内修复是否改善了Chiari II畸形的程度。新生儿发病率将是 记录为与MMC，肌肉强度相关的条件的手术程序的数量 行走状态和尿和粪便持续性。婴儿将在十二零三十个月进行后续访问 详细的神经运动功能分析，认知测试和神经发育评估的校正年龄 地位。此外，将评估长期的社会心理和再现后果。 总而言之，拟议的试验将能够证明胎儿干预是否提供 脊柱裂儿童的生活质量合理，改善了结果。