The Evolving Landscape of Onabotulinumtoxin A Use in Pediatric Lower Urinary Tract Dysfunction

• Published in: Current treatment options in pediatrics 2024-12, Vol.10 (4), p.213-222
• Main Authors: Ha, Darren, Harris, Kelly T., Rove, Kyle O.
• Format: Article
• Language: English
• Subjects: Anticholinergics; Biofeedback; Bladder; Clinical trials; Compliance; Constipation; Critical Care Medicine; Family Medicine; FDA approval; General Practice; Intensive; Intubation; Maternal and Child Health; Medicine; Medicine & Public Health; Patients; Pediatric Urology (A Buchanan and G Campbell; Pediatrics; Section Editors; Success; Topical Collection on Pediatric Urology; Urinary incontinence; Urinary retention; Urinary tract infections; Urogenital system; Urology
• ISSN: 2198-6088; 2198-6088
• DOI: 10.1007/s40746-024-00314-4

Purpose of Review Despite receiving United States Food and Drug Administration (FDA) approval for use as a treatment in pediatric neurogenic detrusor overactivity in 2021, onabotulinumtoxin A (BTX), has been used off-label for decades and has demonstrated safety and efficacy in the management of various types of lower urinary tract dysfunction in children, namely, neurogenic detrusor overactivity, overactive bladder, and dysfunctional voiding. However, data on BTX use in pediatric urology rely heavily on smaller studies with varying definitions of treatment success, leading to some difficulty with result interpretation and challenges with synthesis of pediatric-specific recommendation for BTX use. Recent Findings BTX has shown success when used in managing pediatric lower urinary tract dysfunction with minimal adverse events. These treatments reduce urinary incontinence, improve quality of life, and delay invasive bladder augmentation procedures. In light of these promising results, dosing and durability vary widely. Additionally, there is some discordance between subjective symptomatic improvement and objective urodynamics results. Summary Onabotulinumtoxin A is a feasible and effective treatment option for pediatric lower urinary tract dysfunction, particularly in those who are refractory to conservative measures. A limitation is the likely need for retreatment, which can be a barrier in the overall trajectory of chronic disease management. Large prospective studies are needed, particularly to evaluate the use of BTX for overactive bladder and dysfunctional voiding.