Anal Botulinum Toxin in Children with Hirschsprung Disease and Functional Constipation: A Prospective Cohort study

Abstract

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.

Management of the child with refractory constipation

“…Retrospective studies describe a response rate of 70% when used to treat children with functional constipation 69 . However, in a recent prospective study of children with functional constipation or Hirschsprung disease who were treated with anal sphincter botulinum toxin injection, 47% of children with functional constipation responded compared to 76% of those with Hirschsprung disease 70 . Although the response rate is likely lower than previously thought, internal anal sphincter botulinum toxin injection remains a valuable tool in the treatment of children with a rectal evacuation disorder.…”

Hirschsprung's disease. Management

“…Botulinum toxin injection to the anal sphincter has been proven an effective therapy. [72][73][74][75]…”

“…On the contrary, if the pathology is normal and the non-relaxing IAS has normal or increased tone, we recommend the use of botulinum toxin injection to relieve obstruction. [72][73][74][75] Botulinum toxin not only has a special consideration in the management of obstructive symptoms, but it is an important tool in the management of recurrent HAEC. 72 It blocks acetyl-choline release.…”

“…A non‐relaxing internal anal sphincter (IAS) is always present even after surgery, and it can also produce obstructive symptoms, even if the pressures are normal. Botulinum toxin injection to the anal sphincter has been proven an effective therapy 72–75 …”

“…If aganglionosis or transition zone histology are present the pull‐through needs to be redone, or if the segment is very short, a trial of botulinum toxin may be indicated. On the contrary, if the pathology is normal and the non‐relaxing IAS has normal or increased tone, we recommend the use of botulinum toxin injection to relieve obstruction 72–75 …”