Antegrade Continence Enema Alone for the Management of Functional Constipation and Segmental Colonic Dysmotility (ACE-FC): A Pediatric Colorectal and Pelvic Learning Consortium Study.

PURPOSE: The purpose of the study was to determine if antegrade continence enema (ACE) alone is an effective treatment for patients with severe functional constipation and segmental colonic dysmotility. METHODS: A retrospective study of patients with functional constipation and segmental colonic dysmotility who underwent ACE as their initial means of management. Data was collected from six participating sites in the Pediatric Colorectal and Pelvic Learning Consortium. Patients who had a colonic resection at the same time as an ACE or previously were excluded from analysis. Only patients who were 21 years old or younger and had at least 1-year follow-up after ACE were included. All patients had segmental colonic dysmotility documented by colonic manometry. Patient characteristics including preoperative colonic and anorectal manometry were summarized, and associations with colonic resection following ACE were evaluated using Fisher's exact test and Wilcoxon rank-sum test. p-Values of less than 0.05 were considered significant. Statistical analyses and summaries were performed using SAS version 9.4 (SAS Institute Inc., Cary, North Carolina, United States). RESULTS: A total of 104 patients from 6 institutions were included in the study with an even gender distribution (males n = 50, 48.1%) and a median age of 9.6 years (interquartile range 7.4, 12.8). At 1-year follow-up, 96 patients (92%) were successfully managed with ACE alone and 8 patients (7%) underwent subsequent colonic resection for persistent symptoms. Behavioral disorder, type of bowel management, and the need for botulinum toxin administered to the anal sphincters was not associated with the need for subsequent colonic resection. On anorectal manometry, lack of pelvic floor dyssynergia was significantly associated with the need for subsequent colonic resection; 3/8, 37.5% without pelvic dyssynergia versus 1/8, 12.5% (p = 0.023) with pelvic dyssynergia underwent subsequent colonic resection. CONCLUSION: In patients with severe functional constipation and documented segmental colonic dysmotility, ACE alone is an effective treatment modality at 1-year follow-up. Patients without pelvic floor dyssynergia on anorectal manometry are more likely to receive colonic resection after ACE. The vast majority of such patients can avoid a colonic resection.

Multi-Institutional Review From the Pediatric Colorectal and Pelvic Learning Consortium of Minor Spinal Cord Dysraphism in the Setting of Anorectal Malformations: Diagnosis, Treatment, and Outcomes.

BACKGROUND/RATIONALE: Anorectal malformations (ARM) are associated with congenital anomalies of the spine, but the impact of a minor spinal cord dysraphism (mSCD) on fecal continence in the setting of ARM remains unclear. MATERIALS/METHODS: A retrospective review was performed utilizing data from the Pediatric Colorectal and Pelvic Learning Consortium (PCPLC) registry. The patient cohort was reviewed for ARM type, mSCD screening/incidence/neurosurgical intervention and age-based BMP utilization. RESULTS: 987 patients with ARM were categorized into mild (38%), moderate (32%) or complex (19%). 694 (70%) had normal spinal (NS) status. 271 (27.5%) patients had mSCD. MRI alone (49%) was the most common screening test for mSCD. US screening had a positive predictive value of 86.3% and a negative predictive value of 67.1%. Surgical intervention rates for mSCD ranged between 13% and 77% at a median age of 0.6-5.2 years. 726 (73.6%) patients were prescribed BMP (74.4% NS, 77.5% mSCD). Laxatives were most utilized BMP in all groups <5yo. ≥5yo, enema utilization increased with ARM complexity independent of spine status (with or without neurosurgical intervention). Neurosurgical intervention did not affect BMP utilization at any age or with any ARM when mSCD was identified. CONCLUSIONS: MSCD influence on bowel function in the setting ARM remains unclear. No significant impact of mSCD was noted on ARM patient bowel management program utilization. Variability exists within PCPLC site with screening and intervention for mSCD in patients with ARM. Future studies with standardized care may be needed to elucidate the true impact of mSCD on long term patient outcomes in ARM patients. TYPE OF STUDY: Retrospective Comparative Study. LEVEL OF EVIDENCE: III.

Fecal continence disparities in patients with idiopathic constipation treated at referral institutions for pediatric colorectal surgery.

PURPOSE: Fecal continence is a concern for many patients with idiopathic constipation and can significantly impact quality of life. It is unknown whether racial, ethnic, and socioeconomic disparities are seen in fecal continence within the idiopathic constipation population. We aimed to evaluate fecal continence and associated demographic characteristics in children with idiopathic constipation referred for surgical evaluation. METHODS: A multicenter retrospective study of children with idiopathic constipation was performed at sites participating in the Pediatric Colorectal and Pelvic Learning Consortium (PCPLC). All patients >3y of age with idiopathic constipation diagnosis were included. The primary outcome was fecal continence, categorized as complete (no accidents ever), daytime (no accidents during the day), partial (occasional incontinence day/night), and none (incontinent). We evaluated for associations between fecal continence and race, sex, age, insurance status, and other patient-level factors, employing Kruskal-Wallis and trend tests. RESULTS: 458 patients with idiopathic constipation from 12 sites were included. The median age of diagnosis was 4.1 years. Only 25% of patients referred for surgical evaluation were completely continent. Age at the visit was significantly associated with fecal continence level (p = 0.002). In addition, patients with public and mixed public and private insurance had lower levels of continence (p<0.001). Patients with developmental delay were also more likely to have lower continence levels (p = 0.009) while diagnoses such as anxiety, ADD/ADHD, autism, depression, obsessive-compulsive disorder were not associated. Approximately 30% of patients had an ACE operation (antegrade continence enema) at a median age of 9.2 years at operation. Black patients were significantly less likely to undergo ACE operation (p = 0.016) when compared to white patients. CONCLUSION: We observed data that suggest differences in fecal incontinence rates based on payor status. Further investigation is needed to characterize these potential areas of disparate care. LEVEL OF EVIDENCE: Level III.

The presence of a neurodiverse disorder is associated with increased use of antegrade enema therapy in children with severe constipation: A study from the Pediatric Colorectal and Pelvic Learning Consortium (PCPLC).

INTRODUCTION: Children with severe constipation and a neurodiverse disorder (Autism and/or developmental delay) represent a challenging bowel management group. Treatment outcomes with laxative or enema therapy remain limited and are often complicated by patient/caregiver compliance. We hypothesized that children with neurodiverse disorders and severe constipation would benefit from a bowel management program (BMP) that includes early use of antegrade enemas. MATERIALS AND METHODS: Children requiring BMP in the Pediatric Colorectal and Pelvic Learning Consortium (PCPLC) registry with diagnosis of constipation and/or constipation with pseudo-incontinence were reviewed. Those with Hirschsprung disease and/or Anorectal Malformation were excluded. BMP needs in patients with a neurodiverse diagnosis were compared to those without to evaluate differences in BMP's. RESULTS: 372 patients requiring a BMP were identified. 95 patients (58 autism spectrum, 54 developmental delay) were neurodiverse, and 277 patients were not. Neurodiverse patients had a higher prevalence of enema therapy 62.1% (59/95) vs. 54.9% (152/273) and use of antegrade enema therapy 33.7% (32/95) vs. 21.2% (58/273), p = 0.126. Neurodiverse patients were older 37.9% (36/95) > 12 years vs. 23.1% (63/273), p = 0.001 and 47.6% (10/21) were changed from laxative to enema therapy over time. 80% (8/10) of those changed from laxatives to enemas used antegrade therapy. 67.3% (35/52) of neurodiverse patients followed over time were on enema therapy at the most recent visit with 80% (28/35) requiring antegrade therapy. CONCLUSION: A large portion of patients with a neurodiverse disorder who fail laxative therapy use antegrade enemas to achieve effective bowel management. Early consideration of an antegrade conduit may simplify treatment in this group of children. LEVEL OF EVIDENCE: III.

Use of expedited post-operative protocol for children undergoing appendicostomy reduces length of hospitalization.

BACKGROUND: An appendicostomy (ACE) is a surgical option for antegrade enemas in children with severe constipation and/or fecal incontinence who have failed medical management.  In 2019, we initiated an expedited post-operative protocol and sought to examine our short-term outcomes compared with our historical cohort. METHODS: A retrospective review was performed of all children undergoing ACE between 2017 and 2020. Children were excluded if they underwent an associated procedure (e.g. colon resection). Patients were divided into two cohorts: historical cohort (2017-2018, Group A) and the expedited protocol (2019 to present, Group B). The primary outcome was length of stay. RESULTS: 30 patients met inclusion (Group A = 16, Group B = 14). The most common indications for ACE were constipation (50%) and constipation or fecal incontinence associated with anorectal malformation (43%). Group B experienced a decreased length of stay (1 vs 3 days, P = 0.001) without differences in 30-day surgical site infection (7.1% vs 18.8%, p = 0.61) or unplanned visit (15.4% vs 18.8%, p = 1.0). Group B had a higher prevalence of MiniACE® button placed through the appendix vs. Malone (42.8% vs 12.5%, p = 0.10). CONCLUSIONS: Our expedited post-op protocol decreased length of stay without other significant adverse clinical sequelae. LEVEL OF EVIDENCE: Retrospective Comparative Study, Level III.

Multi-institutional review of bowel management strategies in children with anorectal malformations.

BACKGROUND/RATIONALE: To describe current bowel management program (BMP) strategies in anorectal malformation (ARM) patients based on patient-level predictors using data from a multi-institutional consortium. MATERIALS/METHODS: Patient bowel function and BMP were reviewed from Pediatric Colorectal and Pelvic Learning Consortium (PCPLC) data. The PCPLC is comprised of multidisciplinary specialists researching colorectal and pelvic disorders. Seven US institutions submitted de-identified clinical data on ARM patients into a centralized patient registry. RESULTS: The primary ARM of 624 patients was categorized into Mild (45.2%), Moderate (40.4%) or Complex (14.2%) anomaly classifications. Patient-specific BMP were examined based on age and on the presence of spinal cord/sacral anomalies. 418 (67%) enrolled patients were prescribed BMP (<5 yo 56.4%; ≥5-<12 yo 86.7%; ≥12 81.5%). Constipation was the primary chief complaint (80.2%). Forty percent of patients on a BMP were toilet trained and approximately one-half (48.5%) reported daytime stool accidents. Secondary surgical interventions for antegrade continence enemas (ACE) were examined; 14.5% of patients employed ACE strategies and utilization increased with age and varied based on anatomic anomalies. CONCLUSIONS: This is the first report on BMP strategies for patients with ARM from the Pediatric Colorectal and Pelvic Learning Consortium. Individual patient characteristics are explored for their impact on bowel management strategy utilization. LEVEL OF EVIDENCE: IV.

Intermediate and long-term outcomes of a bowel management program for children with severe constipation or fecal incontinence.

PURPOSE: We sought to examine the long-term clinical success rates of a bowel management program (BMP) for children with severe constipation or fecal incontinence. METHODS: A single center review was conducted of children (≤18 years) enrolled in a BMP and followed in a colorectal specialty clinic (2011-2017). All patients who completed an initial week of the BMP were included. Patients enrolled in a BMP after 2018 were excluded. Success was defined as no accidents and <2 stool smears per week. RESULTS: A total of 285 patients were reviewed. BMP was initiated at a median age of 7 years (9 months-17 years). Primary diagnoses included functional constipation (112), anorectal malformation (ARM) (104), Hirschsprung Disease (HD) (41), rectal prolapse (14), spina bifida (6), fecal incontinence (3) and other (5; 4 sacral coccygeal teratomas and a GSW to the buttocks). Initial bowel regimen included large volume enema in 54% and high dose stimulant laxative in 46%. The initial Bowel Management Week (BMW) was successful in 233 (87% of adherent patients) patients with 17 (6%) non-adherent. One hundred twenty-two patients had follow-up at 12 months (72% success amongst adherent patients, 7% of patient non-adherent) and 98 patients had follow-up at 24 months (78% success amongst adherent patients, 10% of patients non-adherent). 21/154 (14%) patients started on enemas were later successfully transitioned to laxatives and 13/132 (10%) patients started on laxatives subsequently required enemas in order to stay clean. Clinic phone contact occurred outside of scheduled visits for adjustment to the BMP in 44% of patients. 33% of patients had surgery to aid bowel management (antegrade colonic enema (ACE) = 81, resection + ACE = 13, diverting stoma = 4). Median follow up was 2.5 years (5 weeks-7 years). CONCLUSION: Children who follow a structured BMP with readily available personnel to provide outpatient assistance can experience successful treatment of severe constipation or fecal incontinence long-term. A multi-institutional collaboration is necessary to identify factors which predict failure of a BMP and non-adherence. TYPE OF STUDY: Single-center retrospective chart review. LEVEL OF EVIDENCE: 3.

Colonic diverticulosis in an infant: A clue to total colonic Hirschsprung disease.

Total colonic aganglionosis is a rare phenotype of Hirschsprung disease (HD). While the diagnosis is generally established within the neonatal period there are reports of delayed presentation. In this case, we describe a 9-month old girl with no previous medical or surgical history who presented with a small bowel obstruction. A contrast enema performed as part of the work up demonstrated right sided colonic diverticula with no other anatomical abnormalities. Surgical intervention was required to manage the obstruction and colonic biopsies were consistent with HD. This case highlights the association of the unusual finding of colonic diverticula with total colonic HD in infants.

Characteristics of the Contrast Enema Do Not Predict an Effective Bowel Management Regimen for Patients with Constipation or Fecal Incontinence.

BACKGROUND: A bowel management program using large volume enemas may be required for children with anorectal malformations (ARM), Hirschsprung's disease (HD), severe medically refractive idiopathic constipation (IC), and other conditions. A pretreatment contrast enema is often obtained. We sought to determine if the contrast enema findings could predict a final enema regimen. METHODS: A retrospective review was performed at a tertiary care children's hospital from 2011 to 2014 to identify patients treated with enemas in our bowel management program. Patient characteristics, contrast enema findings (including volume to completely fill the colon), and final enema regimen were collected. RESULTS: Eighty-three patients were identified (37 ARM, 7 HD, 34 IC, and 5 other). Age ranged from 10 months to 24 years, and weight ranged from 6.21 kg to 95.6 kg at the time bowel management was initiated. Linear regression showed contrast enema volume was of limited value in predicting effective therapeutic saline enema volume (R(2 )= 0.21). The addition of diagnosis, colon dilation, and contrast retention on plain x-ray the day after the contrast enema moderately improved the predictive ability of the contrast enema (R(2 )= 0.35). Median final effective enema volume was 22 mL/kg (range: 5 - 48 mL/kg). CONCLUSIONS: We were unable to demonstrate a correlation with contrast enema findings and the effective enema volume. However, no patient required a daily enema volume greater than 48 mL/kg to stay clean.

Enterocolitis and bowel function in children with Hirschsprung disease and trisomy 21.

BACKGROUND/PURPOSE: The purpose of this study was to study the effect of trisomy 21 (T21) on enterocolitis rates and bowel function among children with Hirschsprung disease (HD). METHODS: A retrospective cohort study of patients with HD treated at our tertiary children's hospital (2000-2015) and a cohort of patients with HD treated in our pediatric colorectal center (CRC) (2011-2015) were performed. RESULTS: 26/207 (13%) patients with HD had T21. 70 (41%) with HD alone were diagnosed with enterocolitis episodes compared to 9 (38%) with HD+T21 (p=0.71). 55/207 patients were managed in the CRC. 11/55 patients (20%) had HD+T21. 25 (58%) with HD had one or more enterocolitis episodes compared to 4 (36%) with HD+T21 (p=0.20). Number of hospitalizations for enterocolitis was similar between all groups. Toilet training was assessed in 32 CRC patients (25 HD, 7 HD+T21). One child with HD+T21 was toilet trained by age 4years versus 12 with HD (p=0.20). Laxative or enema therapy was required for constipation management in 57% HD versus 64% HD+T21. CONCLUSION: Enterocolitis rates in children with HD+T21 did not differ from rates in children with HD alone. The majority of patients with CRC follow-up had constipation requiring laxative or enema therapy, which demonstrates the need for consistent postoperative follow-up. LEVEL OF EVIDENCE: Retrospective Study - Level II.

Effectiveness of an organized bowel management program in the management of severe chronic constipation in children.

BACKGROUND: Chronic constipation is a common problem in children. The cause of constipation is often idiopathic, when no anatomic or physiologic etiology can be identified. In severe cases, low dose laxatives, stool softeners and small volume enemas are ineffective. The purpose of this study was to assess the effectiveness of a structured bowel management program in these children. METHODS: We retrospectively reviewed children with chronic constipation without a history of anorectal malformation, Hirschsprung's disease or other anatomical lesions seen in our pediatric colorectal center. Our bowel management program consists of an intensive week where treatment is assessed and tailored based on clinical response and daily radiographs. Once a successful treatment plan is established, children are followed longitudinally. The number of patients requiring hospital admission during the year prior to and year after initiation of bowel management was compared using Fisher's exact test. RESULTS: Forty-four children with refractory constipation have been followed in our colorectal center for greater than a year. Fifty percent had at least one hospitalization the year prior to treatment for obstructive symptoms. Children were treated with either high-dose laxatives starting at 2mg/kg of senna or enemas starting at 20ml/kg of normal saline. Treatment regimens were adjusted based on response to therapy. The admission rate one-year after enrollment was 9% including both adherent and nonadherent patients. This represents an 82% reduction in hospital admissions (p<0.001). CONCLUSIONS: Implementation of a structured bowel management program similar to that used for children with anorectal malformations, is effective and reduces hospital admissions in children with severe chronic constipation.

Effect of decreased parenteral soybean lipid emulsion on hepatic function in infants at risk for parenteral nutrition-associated liver disease: a pilot study.

PURPOSE: We performed a pilot trial to compare reduced dose versus standard soybean lipid emulsion in neonates at risk for parenteral nutrition-associated liver disease. METHODS: A prospective randomized controlled trial was performed (2009-2011) enrolling surgical patients ≥ 26 weeks' gestation anticipated to require >50% of daily caloric intake from parenteral nutrition (PN) for at least 4 weeks. Randomization occurred into either reduced (1.0 g/kg/day) or standard (3g/kg/day) groups. Co-primary outcomes for interpretation of the results were conjugated bilirubin and total bile acids. Additional outcomes included ALT, AST, GGT, alkaline phosphatase, growth, and essential fatty acid levels. Outcomes were compared between treatment groups using Wilcoxon rank sums tests. RESULTS: Twenty-eight patients (47% enrollment rate) were included in the study with an average treatment duration of 5.4 weeks. Groups had similar PN calories and protein intake throughout the study. Total increase from baseline was smaller in the reduced vs. standard group for conjugated bilirubin (p=0.04) and total bile acids (p=0.02). Weight z-score increased more in the standard group, and no patient experienced essential fatty acid deficiency. CONCLUSION: Markers of cholestasis rose at a slower rate using reduced lipid doses. This pilot study demonstrates feasibility and need for a larger study evaluating the effects of reduced lipids in patients at risk for developing parenteral nutrition-associated liver disease.

Elimination of soybean lipid emulsion in parenteral nutrition and supplementation with enteral fish oil improve cholestasis in infants with short bowel syndrome.

BACKGROUND: Parenteral nutrition-associated liver disease (PNALD) is a potentially fatal complication for children with intestinal failure. Fish oil-based lipid emulsions have shown promise for the treatment of PNALD but are not readily available. Six cases are presented in which cholestasis resolved after soybean lipid emulsion (SLE) was removed from parenteral nutrition (PN) and enteral fish oil was given. METHODS: A retrospective review at a tertiary children's hospital (July 2003 to August 2008) identified 6 infants with intestinal failure requiring PN for >6 months who developed severe hepatic dysfunction that was managed by eliminating SLE and providing enteral fish oil. RESULTS: Twenty-three infants with short bowel syndrome requiring prolonged PN developed cholestasis. SLE was removed in 6 of these patients, and 4 of the 6 received enteral fish oil. Standard PN included 2-3 g/kg/d SLE with total PN calories ranging from 57 to 81 kcal/kg/d at the time of SLE removal. Hyperbilirubinemia resolved after elimination of SLE within 1.8-5.4 months. Total PN calories required to maintain growth generally did not change. CONCLUSIONS: Temporary elimination of SLE and supplementation with enteral fish oil improved cholestasis in PN-dependent infants. Further trials are needed to evaluate this management strategy.