A case-based update on current options for the management of neurogenic bladder (or neurogenic lower urinary tract dysfunction [NLUTD]) in children was presented at the Plenary Session at the AUA annual meeting on Sunday, May 5, 2024, in San Antonio, Texas. The authors have worked collaboratively to advance care for NLUTD through the CDC’s Urologic Management to Preserve Initial Renal Function in Infants and Young Children with Spina Bifida (UMPIRE) protocol. This iterative study, initiated in 2015, involves 10 centers in the US that manage NLUTD due to the myelomeningocele form of spina bifida in children from birth to 10 years in a standard manner with allowance for deviations as dictated by individual patient factors.¹ Pediatric NLUTD is not unique to spina bifida, but much of the research and literature on the topic involves spina bifida since it is the most common cause of pediatric NLUTD.

Management of NLUTD remains one of the more vexing challenges to pediatric urologists. While exciting changes are occurring in the field, the goals of care remain the same: (1) preservation of renal function, (2) minimization of urinary tract infections, (3) progression toward urinary continence, if important to the child and caregivers, (4) empowerment to gain independence in bladder management, and (5) avoidance of significant disease- and treatment-related complications. Addressing bowel management and sexual health are important as well but are beyond the focus of this discussion.

Two approaches exist in the management of pediatric NLUTD: reactive and proactive. Reactive management is driven by demonstrable clinical changes and is, therefore, less invasive and costly. The proactive approach is defined by early and regular assessment of renal and bladder anatomy and function with videourodynamics, sonography, and laboratory testing. Based on the results, there is a low threshold to initiate clean intermittent catheterization and medical therapy to optimize bladder emptying, minimize bladder storage pressures, and protect the kidneys. Although superiority of the proactive approach is unproven in terms of long-term renal outcomes, it has been increasingly adopted to reduce morbidity.² It is hoped that the continued analysis of accumulating data from UMPIRE study sites can provide greater clarity to this debate.

Annual screening with renal sonography is relatively inexpensive, readily available, and can provide excellent anatomic detail in most without radiation exposure (Figure). Serum creatinine-based estimated glomerular filtration rate has traditionally been used to quantify renal function, but many with NLUTD have decreased muscle mass due to lower extremity disability and, thus, produce less creatinine. To prevent potential overestimation of estimated glomerular filtration rate, use of cystatin C-based formulas is gaining popularity, and comparative studies are ongoing.³ Data from the UMPIRE study have shown that most newborns with myelomeningocele start life with normal kidneys, as less than 4% had more than minimal hydronephrosis, less than 8% had defects noted on nuclear dimercaptosuccinic acid renal scan, and only 15% had vesicoureteral reflux.⁴ Bladders are screened for potential hostility with urodynamic evaluation, and less than 15% of the initial 157 infants participating in the UMPIRE study had high risk bladders.⁵ It should be acknowledged that urodynamic evaluation in infants and small children are notoriously challenging to perform and interpret, even in experienced hands.⁶

When unfavorable bladder characteristics are noted and/or continence becomes a concern, medical therapy with or without clean intermittent catheterization is indicated. Oxybutynin has been the mainstay of such therapy for nearly 50 years, but side effects are common, including dry mouth, constipation, flushing, and, more rarely, mental changes. These side effects can limit efficacy and usage, whether given in immediate- or extended-release oral form, transdermal patch, or intravesical instillation. Other antimuscarinic medications have been introduced more recently; side effects can be similar, and some are not approved for use in children. Newer agents achieving similar effects through stimulation of the β-3 adrenergic receptor in bladder muscle cells are garnering attention because their side effects profile is much more favorable. Mirabegron has been approved for use in children, and vibegron is currently being studied in this age group. The next line of treatment in children for whom these medications are not effective and/or tolerated is intravesical injection of botulinumtoxin A.⁷ The primary downsides in children are that most require cystoscopy under general anesthesia for injection and repeat injections are typically required every 6 to 12 months.

When these measures fail, lower urinary tract reconstruction (LUTR) may be needed to achieve bladder storage pressure less than 40 cm H₂O, reduce the frequency of urinary tract infections, and/or reduce vesicoureteral reflux and/or hydronephrosis. If continence is not a goal, then cutaneous vesicostomy or pouched ileovesicostomy offer simpler forms of diversion than ileal conduits, which were commonly used in the mid-20th century for NLUTD. Augmentation cystoplasty can also achieve those goals, as well as continence, and may be combined with continent catheterizable channels, bladder neck procedures for continence when indicated, and/or procedures to allow antegrade enemas to manage neurogenic bowel. LUTR is not without a significant risk of complications; Szymanski et al⁸ found incidences of additional surgeries, bladder stone surgeries, and bladder perforation in 46%, 33%, and 9%, respectively, in a modern series. The number of augmentation cystoplasties may have decreased in the US over the last 15 to 25 years as use of botulinumtoxin A has increased.⁹ Simultaneously, efforts to reduce the perioperative morbidity of LUTR have included the use of robotic-assisted laparoscopy and adoption of enhanced recovery regimens. The PURSUE study group is prospectively investigating the benefits of the latter with measures such as avoiding or reducing the use of preoperative bowel preps, nasogastric tubes, surgical drains, intraoperative intravenous fluid, and opiates while promoting early feeding and multimodal pain control.¹⁰

Few disorders in pediatric urology receive more attention than NLUTD. Despite its relative rarity, inadequate treatment can lead to decreased quality of life, renal failure, and even death. Fortunately, a combination of collaborative research efforts, newer pharmacologic agents, and efforts to reduce short- and long-term surgical morbidity portend a brighter future for affected children, their caregivers, and the health care community.