Botulinum Toxin Improves Quality of Life and Clinical Outcomes in Pediatric Defecation Disorders.

Objective: To elucidate the effect of internal anal sphincter (IAS) botox as an adjunct to standard treatment for disordered defecation (DD), defined by the inability to effectively evacuate stool from the rectum resulting in constipation, on patient/caregiver quality of life (QoL) metrics in association with corresponding clinical outcomes.

Methods: Consecutive children undergoing IAS botox for DD completed surveys at baseline, two weeks, and three months post-procedure. Time points included assessment of clinical symptoms (Rome IV Questionnaire), QoL (Pediatric Quality of Life Inventory [PedsQL]), and caregiver well-being/family functioning (PedsQL-Family Impact Module [PedsQL-FIM]).

Results: Of 60 participants, the median age was 7 years (IQR 4-10), with 34 (56.7%) males and 32 (53.3%) first-time Botox recipients. The median onset of positive effect after Botox was 6 days (IQR 3-14), while the median loss of effect was 70 days (IQR 39-83). Compared to baseline, by 3 months there was a lower chance of meeting Rome IV criteria for functional constipation in first-time recipients (RR 0.73, 95% CI: 0.58-0.91; p = 0.005). This remained significant after adjusting for any bowel regimen change during the 3-month period following Botox (OR 0.33, 95% CI: 0.13-0.74; p = 0.012). Regarding QoL, there was significant improvement in total PedsQL score at both 2-weeks (11.79 point improvement, 95% CI: 6.36-17.22; p < 0.0001) and 3 months (13.97 point improvement, 95% CI: 8.47-19.47; p < 0.0001) from baseline for first-time recipients, while improvement was only observed at 2 weeks for prior recipients (6.67 point improvement, 95% CI: 0.65-12.69; p = 0.030). First-time recipients demonstrated significant improvements in total PedsQL-FIM score at both 2 weeks (9.33 point improvement, 95% CI: 3.77-14.89; p = 0.001) and 3 months (11.57 point improvement, 95% CI: 5.94-17.20; p < 0.0001) from baseline.

Conclusion: Our findings establish far-reaching benefits of IAS botox primarily for first-time recipients, both clinically and psychosocially, suggesting that appropriate incorporation into the treatment paradigm may globally enhance outcomes in children with DD.