Bethanechol in Tracheomalacia: Two Case Series and a Review of the Literature

Objective: To determine the impact of bethanechol on respiratory outcomes in children with tracheomalacia, we present 2 independent retrospective studies conducted at different institutions with similar conclusions. These studies evaluated the objective clinical outcomes and subjective parental opinion of bethanechol. Methods: Retrospective studies were performed at 2 pediatric institutions. The first was a chart review study of children treated with bethanechol after bronchoscopic diagnosis of moderate-to-severe tracheomalacia by Santiago et al. at Cohen's Children's Hospital. The number of hospitalizations per year, number of days on oral steroids, and frequency of cough were compared before and after treatment. These data were presented in abstract form at the American Thoracic Society International Conference. The second study was a retrospective survey administered to parents of 22 children with bronchoscopy-diagnosed tracheomalacia treated with bethanechol by Piccione et al. at the Children's Hospital of Philadelphia. From this group, parents of 12 patients participated in the survey that asked about perceived changes in symptoms of tracheomalacia and perceived side effects of bethanechol. These data were presented in abstract form at the American Thoracic Society International Conference. Results: In the chart review study, 6-12 months after initiation of bethanechol, trends toward fewer hospitalizations and decreased frequency of cough were observed. The number of days of oral steroids per year was unchanged. In the telephone survey study, among parents surveyed, >50% reported perceived efficacy of bethanechol and overall improvement in their child's quality of life after initiation of therapy. Parents also reported decreased frequency of emergency department visits and cough. No significant side effects were reported. Conclusions: Based on parental feedback and chart abstraction, bethanechol may be a safe and effective medical therapy for young children with tracheomalacia. Although these studies were limited by their retrospective nature and small sample sizes, they represent the first cohort studies supporting earlier proof of concept studies. Given the paucity of medical therapies for tracheomalacia, a trial of bethanechol should be considered for symptomatic children. Further research is needed to confirm these findings and help determine which children are most likely to benefit from this therapy.