Background: In research on pediatric chronic intestinal failure, heterogeneity in reported definitions and outcomes exists. This leads to a risk of reporting bias and impossibility of evidence synthesis. Also, reported outcomes should be relevant to both healthcare providers and patients and their parents. Therefore, the aim of this study is to create a core outcome set (COS) to be used in studies on pediatric chronic intestinal failure.Methods: Candidate outcomes were selected from a recent systematic review. A three‐round Delphi study among key stakeholders and a consensus meeting with an expert panel were undertaken to achieve consensus on the COS.Results: Seventy‐two stakeholders (79%) completed all three rounds of the Delphi process. Ninety‐eight outcomes were assessed, and five new outcomes were added after the first round. Ten outcomes were included in the final COS: weaning from parenteral nutrition, growth, mortality, central line–related infection, central line longevity, sepsis not related to central line infection, central line–related thrombosis, intestinal failure–associated liver disease, (serious) adverse events, and health‐related quality of life.Conclusion: This pediatric chronic intestinal failure COS consists of 10 outcomes important for all key stakeholders. Usage of this set in future research should minimize outcome heterogeneity and enhance the value of evidence synthesis. This will lead to better management in this field of rare gastrointestinal conditions.

Pediatric chronic intestinal failure (PIF) is a rare and heterogeneous condition characterized by the inability of the patient’s intestine to adequately absorb the required fluids and/or nutrients for growth and homeostasis. As a result, patients will become dependent on home parenteral nutrition (HPN). A MEDLINE search was performed in May 2024 with keywords “intestinal failure”, “parenteral nutrition” and “pediatric”. Different underlying conditions which may result in PIF include short bowel syndrome, intestinal neuromuscular motility disorders and congenital enteropathies. Most common complications associated with HPN are catheter-related bloodstream infections, catheter-related thrombosis, intestinal failure-associated liver disease, small intestinal bacterial overgrowth, metabolic bone disease and renal impairment. Treatment for children with PIF has markedly improved with a great reduction in morbidity and mortality. Centralization of care in specialist centers and international collaboration between centers is paramount to further improve care for this vulnerable patient group. A recently promising medical therapy has become available for children with short bowel syndrome which includes glucagon-like peptide 2, a naturally occurring hormone which is known to delay gastric emptying and induce epithelial proliferation. Despite advances in curative and supportive treatment, further research is necessary to improve nutritional, pharmacological and surgical care and prevention of complications associated with parenteral nutrition use.

Life expectancy of children with chronic intestinal failure (CIF) on home parenteral nutrition has greatly improved. Children are now able to grow into adulthood which requires transfer from pediatric to adult health care. A guideline for structured transition is lacking and the demand for a more standardized care for this patient group is necessary. Therefore, we investigated the perceptions of health care professionals from various disciplines working in this specific field, concerning effective interventions regarding transition to adult health care. To create a standardized protocol which provides practical guidance for health care professionals in order to bridge the gap between pediatric and adult health care and to facilitate successful transition of children with chronic intestinal failure. A survey consisting of 20 interventions for transition was sent out to members of the Intestinal Failure working group of European Reference Network for Rare Inherited Congenital (gastrointestinal and digestive) Anomalies (ERNICA) and the Network of Intestinal Failure and Intestinal Transplant in Europe (NITE) group - European Society for Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN) healthcare professionals in 48 medical centers in various countries. Next to 20 interventions, an open-ended question to fill in any other suggestion with respect to most effective intervention was included. Interventions scoring higher than 80% by the participants were included in the protocol. Interventions scoring between 50% and 80% and other own suggestions were discussed during a consensus meeting and included when consensus, defined as unanimous agreement, was reached. Interventions scoring as effective by < 50% of participants were excluded directly. A total of 80 healthcare professionals from 33 medical centers (participation rate 69%) participated. The protocol consisted of modifiable components expected to be targets of interventions. The most important key outcomes of the survey were: 1) assessment of patient's transition readiness and provision of knowledge to the patient by the pediatric team, 2) involvement of parents in the transition process, and 3) collaboration between the pediatric and adult chronic intestinal failure team. In addition it is advised that the transition process should start 1-2 years before transfer. A nurse specialist working in both services should form a bridge. All interventions must be tailor-made and based on the maturity of the patient. This study provides a protocol describing transition of children with chronic intestinal failure from pediatric to adult care. This international protocol will serve as practical guidance for pediatric chronic intestinal failure which will provide a more structured, optimal transition process. It is advised to use this protocol as a formal checklist that can be placed in the patient's chart to review and track the transition process by CIF team members. Future research investigating transition readiness of CIF patients is needed.

Background: Children with chronic IF require long‐term home parenteral nutrition (HPN), administered through a central venous catheter. Catheter‐related bloodstream infection (CRBSI) with Staphylococcus aureus is known to be a serious infection with a high mortality rate and risk of complications. A standardized protocol on the management of S aureus CRBSIs in children receiving HPN is lacking. The aim of this study is to evaluate the effectiveness and safety of the current management in an HPN expertise center in the Netherlands.Methods: We performed a retrospective descriptive cohort study between 2013 and 2022 on children 0–18 years of age with chronic IF requiring long‐term HPN. Our primary outcomes were the incidence of S aureus CRBSI per 1000 catheter days, catheter salvage attempt rate, and successful catheter salvage rate. Our secondary outcomes included complications and mortality.Results: A total of 74 patients (39 male; 53%) were included, covering 327.8 catheter years. Twenty‐eight patients (38%) had a total of 52 S aureus CRBSIs, with an incidence rate of 0.4 per 1000 catheter days. The catheter salvage attempt rate was 44% (23/52). The successful catheter salvage rate was 100%. No relapse occurred, and no removal was needed after catheter salvage. All complications that occurred were already present at admission before the decision to remove the catheter or not. No patients died because of an S aureus CRBSI.Conclusion: Catheter salvage in S aureus CRBSIs in children receiving HPN can be attempted after careful consideration by a multidisciplinary team in an HPN expertise center.

Objectives: To describe incidence, clinical course, diagnostic and therapeutic management and long‐term follow‐up of paediatric intestinal pseudo‐obstruction (PIPO) in the Netherlands between 2000 and 2020.Methods: Multicenter, national, retrospective, observational study including patients aged <18 years diagnosed with PIPO and treated between 2000 and 2020 in Dutch academic medical centres. Outcomes included demographics, incidence, symptoms, diagnostic‐ and treatment methods used during follow‐up, number of hospital admissions and mortality.Results: Between 2000 and 2020, 43 children (median age 120 months, range 13–301, 54% female) were diagnosed with PIPO in the Netherlands. Mean incidence was 0.008/100,000/years (range 0/100 000–0.029/100 000). Twenty‐six patients developed PIPO in the neonatal period. Initial symptoms were vomiting (n = 21/35, 60%) and abdominal distension (n = 14/35, 40%). Diagnostic strategies included imaging, manometry, histopathology, metabolic‐ and genetic screening, endoscopy and exploratory surgery. Treatment was divided in nutritional support, pharmacotherapy, colonic irrigation and surgical interventions, of which nutrition and surgery were the cornerstones for care. During the observed study period, the median number of hospital admissions was 22.5 (range 1–176) with a median of 157.5 days (range 3–840) during 20‐year follow‐up. Two patients (6%) died: one from sepsis and one due to a severe underlying neurological disease. Heterogeneity in diagnostic‐ en treatment methods existed between patients.Conclusions: PIPO is a rare, long‐lasting complex disease requiring a high number of diagnostic and therapeutic interventions and hospital admissions. However, mortality rate is relatively low. Based on our results, we recommend centralization and standardization of care for this complex rare disease.

Catheter-related thrombosis (CRT) is a serious complication associated with home parenteral nutrition (HPN) in children with chronic intestinal failure (CIF). Guidelines on pediatric HPN state that there is insufficient evidence to advocate the prophylactic use of CRT. Aim is to evaluate the efficacy and safety of prophylactic anticoagulation in preventing CRT in children on HPN. We performed a prospective, international, multicenter study across three expertise centers. Children aged 0-18 years on HPN were included and divided into two groups: 1) primary/secondary prophylaxis- and 2) non-prophylaxis group. Participants were followed for 24 months and screened for CRT using ultrasonography annually. Primary outcomes included total incidence of CRT per 1000 catheter days, and association between prophylactic anticoagulation and CRT. Propensity score stratification and logistic regression were used to evaluate the association between prophylactic anticoagulation and CRT, adjusting for significant covariates differing between groups. Balance was visually assessed before and after stratification, and statistical significance (p < 0.05) was determined. Secondary outcomes included incidence of catheter-related bloodstream infections (CRBSI) and bleeding events per 1000 catheter days. A total of 115 children, mean age of 6,9 years (SD 4,6), were included. Fifty-seven patients were receiving prophylactic anticoagulation (50 %). The overall incidence of CRT was 0.17 per 1000 catheter days in 13 patients (11 %), with no significant difference between the prophylaxis (n = 6) and non-prophylaxis group (n = 7) (odds ratio 0.64, 95 % CI: 0.12-3.4, p = 0.60). Incidence rate of CRBSIs was 0.34/1000 catheter days (n = 25). Patients with CRT during follow up were significantly more likely to have a CRBSI (54 %) compared to those without (14 %) (p = 0.010). Two minor bleeding events were reported in the prophylaxis group, resulting in an incidence of 0,03/1000 catheter days. Our study shows that prophylactic anticoagulation does not significantly reduce the incidence of CRT in children on HPN. Our results do underline the clinical importance of optimal catheter care and infection prevention as CRBSIs were more likely to occur in patients with CRT, and emphasize the need for larger multicenter trials to establish evidence-based recommendations for the use of prophylactic anticoagulation in children on HPN.