APSA Outcomes & Clinical Trials Center

The APSA Outcomes and Clinical Trials Center was established in July 2000, and is located within at the American College of Surgeons, Headquarters, in Chicago IL. The goal of this center is to conduct and promote research efforts in pediatric surgery, and to define evidence-based guidelines through outcomes assessment. The APSA Outcomes Center is a nationally centralized data and communication hub for a network of participating study centers, APSA members, and volunteers across the United States. This network of participation can be utilized quickly to achieve adequate sample size for meaningful analysis.

The Center's capabilities include project coordination, database design/management, data analysis, and data dissemination. The APSA Outcomes Web Site has been developed for general information dissemination as well as secured data submission. We strongly encourage ideas and potential research projects to utilize the center.

This six center prospective observational database is designed to gather data on practice patterns, natural history of disease, and surgical outcomes for neonatal surgical congenital anomalies. It is planned that data generated will provide preliminary data for the design and conduct of future trials. It is also anticipated that this six-center group will be able to form the core of much larger consortium that will be competitive for sustained funding. Entities currently targeted for study include, esophageal atresia, abdominal enterocolitis, and others. Data collection will occur locally and be managed at a centralized data center in Boston. The project is funded by the Elizabeth Glaser Pediatric Research Network.

Infants with gastroschisis are at significant risk for morbidity and mortality largely related to inadequate capacity of the abdominal cavity. Current methods of treatment are associated with significant morbidity, prolonged hospitalization, and high costs. This study attempts to evaluate routine use of the Spring-loaded Silo (SLS) in a prospective, randomized fashion to treat neonates with gastroschisis. The primary outcome variable is length of time on the ventilator. Secondary outcome variables are length of time to return of bowel function, bowel ischemia as measured by serum lactate and intraabdominal pressure, and hospital cost.

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Research Coordinator: Aimee Pastor

A Federal grant from the Maternal and Child Health Bureau and Emergency Medical Services for Children was awarded to APSA in 1999. The objectives of this grant are to develop clinical guidelines for the management of specific subsets of pediatric trauma patients. Dr. Steven Stylianos, Chair of the APSA Trauma Committee, has led this work and Dr. Karen Guice is the Project Director. Following are the study profiles:

Isolated Liver or Spleen Injuries: A retrospective analysis of 856 children treated at 32 centers was conducted. Guidelines based on severity of injury by CT grade were proposed and implemented for ICU stay, hospital stay, imaging, and physical activity restriction. The guidelines are in print in the Journal of Pediatric Surgery Stylianos S, and the APSA Trauma Committee. Evidence-based guidelines for resource utilization in children with isolated spleen or liver injury. J Pediatr Surg 2000;35:164-169.

Analysis of the prospective application of guidelines in 312 patients is in the process of dissemination. This information was presented at the APSA Annual Meeting, in Naples, May 20-23rd, 2001 and at the Pediatric Critical Care Colloquium on Pediatric Trauma, in San Diego, September 14-15th, 2001. This article is published in the Journal of Pediatric Surgery Steven Stylianos and the APSA Liver/Spleen Trauma Study Group. Compliance With Evidence-Based Guidelines in Children with Isolated Spleen or Liver Injury: A Prospective Study. J Pedicatr Surg 2002;37;3:453-456.

Pelvic Fracture: A retrospective evaluation of 266 patients from 19 centers revealed inadequate follow-up data and long-term outcomes information to offer clinical guidelines for children. This was confirmed by similar limitations in published data. This information was presented at the American Academy of Pediatrics, in San Francisco, Oct 20th, 2001. View Abstract.

A prospective study was developed to understand functional outcomes and quality of life in pediatric patients after pelvic fracture. This study opened February 2001. 12 centers are IRB approved and open for enrollment. Data will be analyzed for clinical practices that correlate with best functional and quality of life outcomes, and appropriate discharge recommendations will be proposed.

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Nosocomial Pneumonia: This study to examine the incidence, risk factors, and treatment of nosocomial and ventilator acquired pneumonia in pediatric trauma patients was postponed after a retrospective review of existing data revealed a low incidence of pneumonia in the pediatric trauma patient.

Nutritional Trial: A randomized clinical trial of glutamine supplementation and enteral feeding in critically injured children was proposed. An expert panel convened to review literature and existing data. This study was postponed after a retrospective review of existing data revealed a low incidence of bacteraemia and pneumonia in the pediatric trauma patient.

Burns: This multi-center study is designed to identify current burn wound management strategies in children 14 years of age and younger with less than or equal to 20% total body surface area, partial thickness, scald burns. The study opened for patient enrollment in October 2003. Twenty-nine centers located throughout the United States, Canada, and South Africa have registered to participate in the study.

Project Coordinator: Dr. Karen Guice

An R01 funded prospective randomized clinical trial to examine the question of peritoneal drainage vs. laparotomy in the management of small premature infants with necrotizing enterocolitis. The primary outcome variables are operative mortality, i.e. death within 30 days of intervention and failure of primary peritoneal drainage (PPD). The secondary outcome variables include, length of time to complete enteral nutrition, short bowel syndrome defined as the need for chronic parenteral nutrition (greater than 3 months), episodes of fungal sepsis, length of hospital stay, and hospital costs.

Patient enrollment is ongoing at 13 participating centers. 62 patients have been randomized. The first data analysis will take place once 69 patients have been enrolled.

Research Coordinator: Bonnie Lang Silverman, PhD

A prospective observational study of outcomes after the Nuss and Ravich methods of correction of pectus excavatum collects information regarding the anatomy, physiology, psychology, and treatment of pectus. Anatomic defects are assessed by CT scan; physiologic data are collected by pulmonary function tests; psychologic effects are assessed by body image survey, and treatment, performed by the surgeon's best method, is evaluated by monitoring of operative complications and postoperative results. Patients are tested both pre and post- op. Randomization is not appropriate since patients usually come with preconceived ideas of which surgery is preferable and surgeons should be free to do the procedure they believe they do best. A major focus of the study is to document outcomes of repair regardless of method used.

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Research Coordinator: Karen Mitchell

This study attempts to determine if prenatal corticosteroids improves oxygenation and reduces time to ventilator independence in fetuses diagnosed with CDH. The primary outcome variable is time to ventilator independence and oxygenation index at 3 hours. Secondary outcome variables include mortality, birth weight, head circumference, length at birth, oxygenation index of 25 or greater, ECMO use, FiO2 and ventilator settings at 30 days, need for oxygen at discharge, treatment with a gastrostomy tube at 30 days or before discharge, treatment with postnatal steroids, and length of hospital stay. 15 centers from 4 countries have committed to participate; there are 7 active centers and 2 more are submitting for IRB approval. There have been 25 eligible patients with 15 enrolled; 9 of them have completed the protocol. This study is ongoing and open for enrollment.

Principal Investigator: Kevin Lally, MD

A 5-year, NIH sponsored, randomized, controlled multi-center trial comparing patients who undergo fetal repair to those who undergo standard postnatal care for open neural tube defects. The aim of this study is to evaluate the safety and efficacy of surgical repair of fetal MMC before 26 weeks gestation. There are two primary outcomes, a reduction in meeting objectively defined criteria for shunting hydrocephalus and an assessment of neurodevelopment at 12 and 30 months. There are several secondary outcomes which look at a variety of maternal and fetal/neonatal issues.

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Program Manager : Catherine Shaer, MD

A prospective randomized, controlled trial comparing two groups, patients who undergo endoscopic fetal tracheal occlusion between 24 and 28 weeks' gestation, and patients who deliver at or near term and receive standard tertiary postnatal care. This single center NIH sponsored randomized clinical trial for the treatment of severe congenital diaphragmatic hernia completed enrollment in the fall of 2001. The primary outcome variable,survival at 90 days, has been determined and the results are being compiled. The secondary outcome measures comprise long-term follow-up issues: growth and development, neurologic sequelae, pulmonary function and recurrent hospitalizations. The children are assessed annually at age 1 and 2 years. That information continues to be gathered at this time, as the youngest children are just 1 year of age.

For more information regarding this study, contact: fetus@surgery.ucsf.edu

MOMS Project: Fetal Surgery Trial for Spina Bifida Dr. Scott Adzick, CHOP Dr. Joe Brunner, Vanderbilt Dr. Michael Harrison, UCSF

NICHD has sponsored a 5-year multi-center, randomized, controlled trial of 200 patients, which will compare the safety and efficacy of fetal surgical repair and traditional postnatal repair of open neural tube defects. Patients carrying a fetus diagnosed with spina bifida will be eligible for the prenatal surgery, which will be performed between the 19th and 25th weeks of pregnancy. The study has two primary outcomes: death or the need for ventricular decompressive shunting by one year of life and a composite outcome of two measures, the Bayley Scale of Infant Development and distal somatosensory function and motor sensory assessment of level of lesion as compared to anatomic level. There are many secondary outcomes which look at maternal, neonatal and infant issues such as maternal death, maternal reproductive functioning at 30 months post delivery, neonatal death or stillbirth, prematurity and complications related to it, and appearance of the Chiari II, motor function and total days of hospitalization of the infant through 30 months of age.

Program Manager: Catherine Shaer, MD