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Anal Botulinum Toxin in Children with Hirschsprung Disease and Functional Constipation: A Prospective Cohort study
Desiree F Baaleman1,2, Alexandra Hallagan1,3, Devin R Halleran4
1Department of Pediatrics, Division of Gastroenterology, Hepatology, and Nutrition, Nationwide Children's Hospital, Columbus, Ohio, United States.
Insights
Anal sphincter botulinum toxin injections (BTIs) effectively treat severe constipation in children with Hirschsprung disease (HD) and functional constipation (FC). BTIs offer a viable treatment option for pediatric defecation disorders.
Area of Science:
- Pediatric Gastroenterology
- Colorectal Surgery
- Pediatric Urology
Background:
- Severe defecation disorders in children, including Hirschsprung disease (HD) and functional constipation (FC), often require advanced treatment options.
- Anal sphincter botulinum toxin injections (BTIs) are an emerging therapy for managing these complex pediatric conditions.
Purpose of the Study:
- To evaluate the effectiveness and outcomes of anal sphincter BTIs in pediatric patients diagnosed with HD and FC.
- To compare the perceived effectiveness of BTIs between children with HD and FC.
Main Methods:
- A prospective cohort study involving 42 children (25 with HD, 17 with FC) treated with BTIs between July and December 2018.
- Patient-reported effectiveness, symptom assessment, and the Cleveland Clinic Constipation Score (CCCS) were recorded at baseline and at 2 weeks, 2 months, and 4 months post-injection.
Main Results:
- BTIs were perceived as effective in a majority of children with HD (76% at 2 weeks, 71% at 2 months) and FC (47% at both time points).
- Children with HD reported higher BTI effectiveness at the 2-month follow-up compared to those with FC (p=0.022).
- Self-limiting adverse effects, such as fecal incontinence and injection site pain, were reported by 44% of participants.
Conclusions:
- Anal sphincter BTIs demonstrate efficacy in treating constipation for both pediatric Hirschsprung disease and functional constipation patients.
- BTIs represent a potentially valuable therapeutic option for children experiencing severe defecation disorders.
Introduction:
Anal sphincter botulinum toxin injections (BTIs) are used in the treatment of children with severe defecation disorders, including Hirschsprung disease (HD) and functional constipation (FC). Our objective was to evaluate the outcomes of BTI in these children.
Materials And Methods:
We performed a prospective cohort study of children undergoing BTI from July 2018 to December 2018. We recorded perceived effect of the BTI, including effectiveness ranging from 0 (not at all effective) to 4 (extremely effective). In addition, we recorded symptoms and the Cleveland Clinic Constipation Score (CCCS). Data were collected at baseline and at 2 weeks, 2 months, and 4 months post-injection.
Results:
Forty-two children (HD = 25, FC = 17) were included in the study (median age 4.3 years, IQR 2.4-7.2, 52% male). Twenty-two (88%) children with HD and eight (47%) children with FC had previously undergone a BTI. BTIs were perceived effective in 16 (76%) and 12 (71%) children with HD and eight (47%) and seven (47%) children with FC at 2-week and 2-month follow-up, respectively. Effectiveness was not rated differently between groups except at the 2-month follow-up, when patients with HD rated the BTI more effective compared to those with FC (median 2 [HD] vs. median 1 [FC], p = 0.022). Over the course of the study, 17/39 (44%) children reported self-limiting adverse effects such as fecal incontinence and pain at the injection site.
Conclusion:
Anal sphincter BTIs can be effective in the treatment of constipation in both HD and FC patients.
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