(Component C) Spina bifida (SB) is the most common permanently disabling birth defect in children and affects 1 in 2,000 live births in the United States. More than 70,000 people live with SB and its long-term effects on multiple organ systems. For the urologic management of newborns with SB, preservation of the upper urinary tracts and thus avoiding permanent loss of renal function via appropriate bladder management has been a key management goal for pediatric urologists and SB healthcare providers. Newborns with SB are generally placed on a close observation protocol that includes serial imaging of the urinary tract and urodynamics, as well as treatment options such as clean intermittent catheterization (CIC) and anticholinergic medications. The rationale behind this approach is to promote detrusor muscle relaxation and development by bladder cycling and ensure bladder drainage to protect the upper urinary tract and prevent hydronephrosis. However, the outcomes of this management are poorly documented, especially in patients who are presumed at low risk for renal damage, and it is unclear if the interventions of CIC and anticholinergics are appropriate for all newborn patients when they are followed under a close observation protocol. The long-term goal of this project is to improve the clinical management of pediatric patients with SB. The objective is to implement and evaluate the best practice urologic protocol that was developed at the CDC that will lead to a standardized management protocol of efficient and effective urinary tract care in newborns and young children with SB that preserves renal function for at least the first five years of life. Our central hypothesis is that a standardzed management protocol will promote efficient use of health care resources and effective clinical management that preserves long-term renal function with minimized morbidity. The rationale for the proposed research is that standardized management protocols that have been implemented in other clinical areas such as cystic fibrosis and congenital heart conditions that have positivel influenced individual patient care as well as public health / population health. We will test our central hypothesis and accomplish the objective of this proposal by pursuing the specific aim of participating as a CDC registry site that will implement and evaluate the Urologic Management to Preserve Renal Function Protocol for Young Children with Spina Bifida. Due to the large and unique population at our institution, enrollees to the national registry will include prenatal closre patients and ethnically diverse patients including those with Hispanic backgrounds.
The purpose of this proposal is to work collaboratively with the Centers for Disease Control and Prevention and other grantee spina bifida clinics to collect longitudinal data on individuals with spina bifida. The Texas Children's Hospital/Baylor College of Medicine Spina Bifida Clinic would add a large and a racially and ethnically diverse population to the national data registry. As data is collected and analyzed, changes to clinical practice may be proposed in order to develop population-level national standards for spina bifida care management and ultimately improve the lives of people with spina bifida.