Spina bifida (SB) is the most common permanently disabling birth defect in the United States. There exists a broad spectrum in the degree to which this condition impairs each individual patient. In addition, SB involves multiple organ systems. As a result, the care of such patients is complex, requires multiple specialists and must be individualized to each patient's degree of impairment within each organ system. This has made it difficult to create evidence-based algorithms for management of spina bifida patients with mostly retrospective and incomplete data to rely on. Spina bifida can inhibit proper bladder function due to loss of normal bladder innervation. This can result in high bladder pressures, which over time can lead to hydronephrosis, vesicoureteral reflux, incontinence, urinary tract infections and renal insufficiency. The primary goal of urologic management in patients with SB is preservation of renal function. Secondary goals include prevention of urinary tract infection and achieving acceptable urinary continence. Practice patterns in the management of newborns and small children with SB vary across different institutions and regions. There are currently limited prospective data on protocols to manage these patients with clear outcomes related to renal function. The primary goal of this study is to implement and evaluate a protocol for the urologic management and follow- up of infants and young children with SB across 10 different institutions. Measures of renal function at five years of age will serve as the primary outcome for the study. Secondary outcomes will include cost- effectiveness of the protocol and frequency of urinary tract infections. Results of this prospective study will be used to compare with outcomes in renal function and urinary tract infection at other institutions involved in the recently create National Spina Bifida Patient Registry. This proposal supports the overall mission of the Centers for Disease Control and Prevention by helping protect this vulnerable population and establishing standards of care that will promote improved health among patients with SB.
Proper urologic management of infants and young children with spina bifida is critical for long-term preservation of renal function. Currently there are limted data to support the wide variety of management protocols in use across the United States. Implementation of the proposed protocol for urologic management will serve to establish data supporting an evidence-based approach to these patients.