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Background: Achievement of enteral autonomy (EA) is the ultimate treatment goal in pediatric intestinal failure (IF). We aimed to assess predictors of EA in pediatric short bowel syndrome (SBS) and explore the impact of residual small bowel (SB) and large bowel (LB) length on EA. Methods: A retrospective cohort study was performed on infants aged <12 months (n = 367, six centers) with SBS referred between 2010 and 2015. The cohort was stratified based on the achievement of EA. Statistical testing was completed using t‐test, chi‐square, Cox proportional hazards regression model, and Kaplan‐Meier analysis. Results: EA was achieved in 229 patients. In the multivariable analysis, the percentage of residual LB (hazard ratio [HR] = 1.02; 95% CI = 1.01–1.02) and SB (HR = 1.01; 95% CI = 1.01–1.02) length, presence of the ileocecal valve (HR = 2.02; 95% CI=1.41–2.88), and not coming from a high‐volume transplantation center (HR = 2.42; 95% CI = 1.68–3.49) were positively associated with EA, whereas a negative association was seen with the presence of stoma at the time when shortest remnant was documented (HR = 0.72; 95% CI = 0.52–1.00). EA achievement was significantly different between the anatomical subgroups (log‐rank test P < 0.001) with an EA rate of 80.4% in infants with ≥50% SB and LB (median time 209 days); 62.5% with ≥50% SB and <50% LB (397 days); 58.3% with <50% SB and ≥50% LB (1192 days), and 25.9% with <50% SB and LB. Necrotizing enterocolitis (NEC) was not associated with a better achievement of EA (NEC vs other etiologies: log‐rank test P = 0.33). Conclusions: Overall, 62% of infants with IF secondary to SBS achieved EA over a mean time of follow‐up of 2.3 years. A colon length of >50% can compensate for the loss of small bowel (<50%) and account for similar EA rates as those in children with residual SB > 50%. [ABSTRACT FROM AUTHOR]

Aim: To explore the perceptions and practices of Australasian paediatric gastroenterologists in diagnosing coeliac disease (CD) before and during the COVID‐19 pandemic. Methods: Paediatric gastroenterologists in Australasia were invited via email to complete an anonymous online questionnaire over a 2‐week period in 2021. Results: The questionnaire was completed by 39 respondents: 33 from Australia and six from New Zealand (NZ) equating to a 66% response rate. Thirty‐four (87%) of the 39 respondents reported they currently practised non‐biopsy diagnosis of CD in eligible children, while the rest diagnosed CD using biopsy confirmation only. All NZ respondents practised non‐biopsy CD diagnosis. A majority of responders (76%) who practised non‐biopsy CD diagnosis followed the 2020 European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) guidelines. Twenty‐two (56%) respondents reported that they started using a non‐biopsy CD diagnosis protocol before the pandemic and did not change their practice during the pandemic, 10 (26%) started diagnosing non‐biopsy CD during the pandemic, 5 (13%) stated their practices of CD were not impacted by the pandemic and 2 (5%) did not respond on whether the pandemic changed their practice. Conclusion: The majority of Australasian gastroenterologist respondents reported they routinely utilised the 2020 ESPGHAN diagnostic criteria in eligible children; half of them started prior to the pandemic and another quarter started this approach due to the pandemic. A minority of practitioners routinely rely only on biopsy confirmation to diagnose CD. [ABSTRACT FROM AUTHOR]

This letter to the editor discusses the incidence rates of pediatric-onset inflammatory bowel disease (IBD) in Māori and Pasifika children in Auckland, New Zealand. The study found that out of 178 children diagnosed with IBD between 2010 and 2020, only two (1.1%) were Māori and none were Pasifika, despite these groups representing a significant portion of the population. The incidence rates of IBD in Māori children were found to be low compared to other ethnicities, consistent with existing literature. However, the study acknowledges limitations in accurately identifying ethnicity and calls for further research to study the incidence and risk factors for IBD in Māori and Pasifika children. [Extracted from the article]

Despite regression of the pituitary lesion, the patient continued to require growth hormone therapy. Hypoglycaemia and deficiencies in growth in the presence of infantile haemangiomata should prompt early pituitary function testing and neuroimaging. In patients with five or more cutaneous lesions, abdominal ultrasonography to identify hepatic lesions is recommended. However, Kasabach-Merritt phenomenon is associated with Kaposiform haemangioendotheliomas which are histologically distinct and locally aggressive vascular tumours that progressively proliferate without expected involution, unlike IH.2 If lesions are found in the liver, consumptive hypothyroidism can occur due to type III iodothyronine deiodinase activity. [Extracted from the article]

Aim: Eosinophilic oesophagitis (EoE) is a rare, chronic, relapsing immune/antigen‐mediated disease characterised by symptoms of oesophageal dysfunction, with a paucity of data among New Zealand (NZ) children. This 3‐year prospective study aimed to characterise EoE diagnosed nationally and to describe initial treatment strategies adopted. Methods: Information on new diagnoses of paediatric EoE was obtained via the New Zealand Paediatric Surveillance Unit, through monthly questionnaires. Results: From February 2014 to January 2017, 73 new cases (74% male) of EoE were reported, including 74% NZ European, 10% Asian, 7% Māori, 5% Middle‐Eastern and 3% Pacific peoples. Median age of symptom onset was 4 years; dysphagia (48%) was the most common, followed by vomiting/regurgitation (40%), food impaction (19%) and epigastric pain (16%). A co‐morbid history of other allergic conditions was present in 62% of patients, and 41% had a first degree relative with atopy. Seventy‐nine percent of patients had abnormal endoscopic findings, most commonly linear furrows and white plaques; none had strictures. Median eosinophil count per high‐powered field was 40 and 50 in the mid and distal oesophagus, respectively. Fifty‐four percent of patients were initially managed with dietary manipulation alone (four required elemental feeds, five nasogastric tubes). Fifty‐four percent of patients were treated with swallowed corticosteroids and 7% with prednisone. One patient was also treated with a leukotriene receptor antagonist. Conclusion: This first prospective study on paediatric patients with EoE in NZ finds similar demographics and disease characteristics as in other populations despite our unique ethnic population. Long‐term prospective observational data should significantly improve our knowledge of this rare condition. [ABSTRACT FROM AUTHOR]

In this retrospective cohort study, we describe the anthropometric and clinical outcomes of EEN as induction therapy for pCD in NZ. Although 56.3% of our patients achieved clinical remission, these rates are lower than European pCD cohorts where remissions rates are 84-86.6%.4,5 Further prospective study is warranted to understand the reasons behind this difference. Paediatric-onset Crohn's disease (pCD) is rising in incidence in Australasia.1 Exclusive enteral nutrition (EEN) is first-line therapy for induction of remission in pCD.2 Despite its routine use, there is little published on the outcomes of EEN in New Zealand (NZ). [Extracted from the article]

Background and Aims: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the G lobal AL agille A lliance (GALA) study., Approach and Results: Maralixibat trials comprise 84 patients with ALGS with up to 6 years of treatment. GALA contains retrospective data from 1438 participants. GALA was filtered to align with key maralixibat eligibility criteria, yielding 469 participants. Serum bile acids could not be included in the GALA filtering criteria as these are not routinely performed in clinical practice. Index time was determined through maximum likelihood estimation in an effort to align the disease severity between the two cohorts with the initiation of maralixibat. Event-free survival, defined as the time to first event of manifestations of portal hypertension (variceal bleeding, ascites requiring therapy), surgical biliary diversion, liver transplant, or death, was analyzed by Cox proportional hazards methods. Sensitivity analyses and adjustments for covariates were applied. Age, total bilirubin, gamma-glutamyl transferase, and alanine aminotransferase were balanced between groups with no statistical differences. Event-free survival in the maralixibat cohort was significantly better than the GALA cohort (HR, 0.305; 95% CI, 0.189-0.491; p <0.0001). Multiple sensitivity and subgroup analyses (including serum bile acid availability) showed similar findings., Conclusions: This study demonstrates a novel application of a robust statistical method to evaluate outcomes in long-term intervention studies where placebo comparisons are not feasible, providing wide application for rare diseases. This comparison with real-world natural history data suggests that maralixibat improves event-free survival in patients with ALGS., (Copyright © 2023 The Author(s). Published by Wolters Kluwer Health, Inc.)

Background and Aims: Alagille syndrome (ALGS) is a multisystem disorder, characterized by cholestasis. Existing outcome data are largely derived from tertiary centers, and real-world data are lacking. This study aimed to elucidate the natural history of liver disease in a contemporary, international cohort of children with ALGS., Approach and Results: This was a multicenter retrospective study of children with a clinically and/or genetically confirmed ALGS diagnosis, born between January 1997 and August 2019. Native liver survival (NLS) and event-free survival rates were assessed. Cox models were constructed to identify early biochemical predictors of clinically evident portal hypertension (CEPH) and NLS. In total, 1433 children (57% male) from 67 centers in 29 countries were included. The 10 and 18-year NLS rates were 54.4% and 40.3%. By 10 and 18 years, 51.5% and 66.0% of children with ALGS experienced ≥1 adverse liver-related event (CEPH, transplant, or death). Children (>6 and ≤12 months) with median total bilirubin (TB) levels between ≥5.0 and <10.0 mg/dl had a 4.1-fold (95% confidence interval [CI], 1.6-10.8), and those ≥10.0 mg/dl had an 8.0-fold (95% CI, 3.4-18.4) increased risk of developing CEPH compared with those <5.0 mg/dl. Median TB levels between ≥5.0 and <10.0 mg/dl and >10.0 mg/dl were associated with a 4.8 (95% CI, 2.4-9.7) and 15.6 (95% CI, 8.7-28.2) increased risk of transplantation relative to <5.0 mg/dl. Median TB <5.0 mg/dl were associated with higher NLS rates relative to ≥5.0 mg/dl, with 79% reaching adulthood with native liver ( p < 0.001)., Conclusions: In this large international cohort of ALGS, only 40.3% of children reach adulthood with their native liver. A TB <5.0 mg/dl between 6 and 12 months of age is associated with better hepatic outcomes. These thresholds provide clinicians with an objective tool to assist with clinical decision-making and in the evaluation of therapies., (Copyright © 2023 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of American Association for the Study of Liver Diseases.)