Clinical Utility of Botulinum Toxin Injections in Children with Gastrointestinal Disorders

Abstract

Purpose of review: To review the clinical utility of botulinum toxin (BTX) injections in children with gastrointestinal (GI) disorders.

Recent findings: BTX is a presynaptic acetylcholine inhibitor with medical indications ranging from the treatment of muscle spasticity to gastrointestinal disorders involving sphincter dysfunction. BTX injections to the upper esophageal sphincter are used to target cricopharyngeal achalasia and retrograde cricopharyngeal dysfunction as temporary measures to decrease sphincter tone and improve swallowing, potentially eliminating the need for surgery. Lower esophageal and pyloric BTX injections have shown benefit in achalasia and gastroparesis, particularly in children unable to undergo or refractory to definitive treatments. Anorectal indications represent the largest experience, especially in children with persistent obstructive defecation symptoms after surgery for Hirschsprung's disease. BTX injection is a minimally invasive, reversible, and generally well-tolerated intervention with mild, self-limited side effects. However, its limitations include transient effects, procedural variability, and primarily off-label use, with most pediatric experience derived from small series. Prospective studies are needed to further evaluate patient selection, standardization, dosage, and long-term efficacy in pediatric gastrointestinal disorders.

Keywords: Achalasia; Botulinum toxin; Functional constipation; GI motility; Neurogastroenterology and motility; Pediatric gastroenterology.