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.
| Farmer, Diana L; Thom, Elizabeth A; Brock 3rd, John W et al. (2018) The Management of Myelomeningocele Study: full cohort 30-month pediatric outcomes. Am J Obstet Gynecol 218:256.e1-256.e13 |
| Antiel, Ryan M; Adzick, N Scott; Thom, Elizabeth A et al. (2016) Impact on family and parental stress of prenatal vs postnatal repair of myelomeningocele. Am J Obstet Gynecol 215:522.e1-6 |
| Johnson, Mark P; Bennett, Kelly A; Rand, Larry et al. (2016) The Management of Myelomeningocele Study: obstetrical outcomes and risk factors for obstetrical complications following prenatal surgery. Am J Obstet Gynecol 215:778.e1-778.e9 |
| Tulipan, Noel; Wellons 3rd, John C; Thom, Elizabeth A et al. (2015) Prenatal surgery for myelomeningocele and the need for cerebrospinal fluid shunt placement. J Neurosurg Pediatr 16:613-20 |
| Brock 3rd, John W; Carr, Michael C; Adzick, N Scott et al. (2015) Bladder Function After Fetal Surgery for Myelomeningocele. Pediatrics 136:e906-13 |
| Adzick, N Scott; Thom, Elizabeth A; Spong, Catherine Y et al. (2011) A randomized trial of prenatal versus postnatal repair of myelomeningocele. N Engl J Med 364:993-1004 |
