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，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，该手术可降低分流依赖性 脑积水和恢复小脑和脑干更正常的配置。然而，临床结果 的MMC胎儿手术是基于与历史对照的比较，仅检查疗效， 安全为代价的 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， 200名患者将在三个胎儿外科单位（FSU）进行，加利福尼亚大学旧金山分校 弗朗西斯科，费城儿童医院和范德比尔特大学医学中心，沿着一个 独立的数据和研究协调中心，乔治华盛顿大学生物统计中心。的 试验的主要目的是确定18 - 256周胎儿脊髓脊膜膨出的宫内修复 妊娠可改善预后，如1）死亡或需要心室减压分流， 2）死亡或Bayley精神发育指数，与标准产后修复相比 符合资格标准的患者将被集中随机分配至子宫内或 MMC的标准产后修复。分配到胎儿手术组的患者将出院到附近的 手术后的住宿，除非不可行，在这种情况下，他们将返回他们分配的FSU在32？ 孕37周剖宫产分娩。分配接受产后手术的患者 组将返回家中，并在37周时返回分配的FSU进行剖宫产分娩。磁 将在入组、出院或足月妊娠时以及1岁时进行共振成像， 确定宫内修补术是否改善了基亚里II畸形的程度。新生儿发病率将 记录与MMC、肌肉力量， 无菌状态和尿和粪便的细菌。婴儿将在12个月和30个月时进行随访 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 status.此外，还将对母亲的长期心理社会和生殖后果进行评估。 总之，拟议的试验将能够证明胎儿干预是否提供 改善脊柱裂儿童的预后，提高其生活质量。