Characterizing the Use of Botulinum Toxin in Patients with Hirschsprung Disease Treated at Referral Institutions for Pediatric Colorectal Surgery

Background: Botulinum toxin (BT) injection is used to treat pediatric patients with Hirschsprung disease (HD) who struggle with obstructive symptoms from hypertonic sphincters. The timing, frequency and indications of its use are not well described. We aimed to characterize the use of BT in HD patients across pediatric colorectal surgery referral centers as a step toward generation of best-practice guidelines.

Methods: A multicenter retrospective study of the use of BT in children (0-18y) with HD was performed. These patients were all evaluated at sites participating in the Pediatric Colorectal and Pelvic Learning Consortium (PCPLC) between 2017-2021. The PCPLC hosts a multi-institutional registry of patients with colorectal and pelvic disorders, providing demographic and clinical information. Sites with <10 HD patients recorded during the period were excluded. A pullthrough procedure was considered redo if prior pullthrough was performed within or outside the PCPLC. Patient characteristics including surgical history were captured and BT practice patterns were evaluated using Fisher’s exact tests, Kruskal-Wallis and other trend tests. P-values <0.05 were considered significant.

Results: A total of 494 patients with HD at 8 centers were included. 118 (23.9%) of these patients received at least one BT injection. Median age at pullthrough was 3.4 months (0.7, 8.5). The median number of days between pullthrough and first BT was 403.5 days (88.0, 903). 49 patients included had a redo pullthrough procedure. Patients who required redo pullthrough procedures were more likely to have received BT ever compared to those who only underwent one pullthrough (53.1% vs. 22.7%, <.001). Patients whose pullthrough was performed at a younger age were less likely to receive BT (p=0.021, Table 1). Hispanic patients were less likely to have received BT than non-Hispanic patients (9.6% vs. 26.3%; p=0.006). No significant difference was seen in BT use in patients with trisomy 21 or demographic factors including insurance status. Greater frequency of BT per patient was seen in patients who required redo pullthrough procedures (p <.001) and patients who underwent pullthrough at an older age (p=0.035). Percentage of HD patients receiving any BT varied significantly across sites (7.8%, 8.9%, 10%, 21.2%, 26.7%, 31%, 47.8%, 48.5%; p<.001). Use of ultrasound with injection also varied across sites (p <.001).

Conclusion: Use of botulinum toxin in patients with Hirschsprung disease varies across referral pediatric colorectal surgery centers indicating the need for consensus practice guidelines. Greater BT use was seen in patients who underwent redo surgery and in those who underwent pullthrough at an older age. Hispanic patients received less BT. These later findings warrant further investigation to evaluate how timing of surgery impacts patient outcomes and for potential disparities in care.