Trends in Pediatric Intestinal Failure: A Multicenter, Multinational Study.

OBJECTIVES: To assess the natural history and outcomes of children with intestinal failure in a large, multicenter, geographically diverse contemporary cohort (2010-2015) from 6 pediatric intestinal failure programs. STUDY DESIGN: Retrospective analysis of a multicenter intestinal failure cohort (n = 443). Competing-risk analysis was used to obtain cumulative incidence rates for the primary outcome (enteral autonomy, transplantation, or death). The χ2 test and Cox proportional hazard regression were used for bivariate and multivariable analyses. RESULTS: The study cohort comprised 443 patients (61.2% male). Primary etiologies included short bowel syndrome (SBS), 84.9%; dysmotility disorder, 7.2%; and mucosal enteropathy, 7.9%. Cumulative incidences for enteral autonomy, transplantation, and death at 6 years of follow-up were 53.0%, 16.7%, and 10.5%, respectively. Enteral autonomy was associated with SBS, ≥50% of small bowel length, presence of an ileocecal valve (ICV), absence of portal hypertension, and follow-up in a non-high-volume transplantation center. The composite outcome of transplantation/death was associated with persistent advanced cholestasis and hypoalbuminemia; age <1 year at diagnosis, ICV, and intact colon were protective. CONCLUSIONS: The rates of death and transplantation in children with intestinal failure have decreased; however, the number of children achieving enteral autonomy has not changed significantly, and a larger proportion of patients remain parenteral nutrition dependent. New strategies to achieve enteral autonomy are needed to improve patient outcomes.

Frailty in Children with Liver Disease: A Prospective Multicenter Study.

OBJECTIVE: To assess frailty, a measure of physiologic declines in multiple organ systems, in children with chronic liver disease using a novel pediatric frailty tool. STUDY DESIGN: We performed a prospective cross-sectional multicenter study at 17 liver transplantation (LT) centers. 71 children (5-17 years of age), 36 with compensated chronic liver disease (CCLD) and 35 with end-stage liver disease (ESLD) and listed for LT, were assessed for frailty using validated pediatric tools to assess the 5 classic Fried Frailty Criteria-slowness, weakness, exhaustion, diminished physical activity, and shrinkage. Test scores were translated to age- and sex-dependent z scores, generating a maximum frailty score of 10. RESULTS: The median frailty score of the cohort was 4 (IQR 3, 5). Subjects with ESLD had significantly higher frailty scores (median 5; IQR 4, 7) than subjects with CCLD (median 3; IQR 2, 4); (P < .0001). Area under the curve receiver operating characteristic for frailty scores to discriminate between ESLD and CCLD was 0.83 (95% CI 0.73, 0.93). Forty-six percent of children with ESLD were frail and there was no correlation between pediatric frailty scores and physician's global assessments (r = -0.24, 95% CI -0.53, 0.10). CONCLUSIONS: A novel frailty tool assessed additional dimensions of health, not captured by standard laboratory measures and identified the sickest individuals among a cohort of children with chronic liver disease. This tool may have applicability to other children with chronic disease.

A clinical prediction rule and platelet count predict esophageal varices in children.

BACKGROUND & AIMS: The validation of noninvasive tests to diagnose esophageal varices is a priority in children because repeated endoscopic evaluations are too invasive. We measured the ability of a previously developed noninvasive clinical prediction rule (CPR) to predict the presence of esophageal varices in children. METHODS: We analyzed data from 108 children, younger than age 18, who received endoscopies at 8 centers, to assess portal hypertension from chronic liver disease or portal vein obstruction. Blood test and abdominal ultrasound scan results were obtained within 4 months of endoscopy. Grading of varices identified by endoscopy was confirmed by independent blinded review. Spleen size, based on data from the ultrasound scan, was expressed as a standard deviation score relative to normal values for age. RESULTS: Of the children studied, 74 had esophageal varices (69%), including 35 with large varices (32%). The best noninvasive predictors of esophageal varices of any size were as follows: platelet:spleen size z-score ratio (area under the receiver operating characteristic curve [AUROC], 0.84; 95% confidence interval [CI] 0.75-0.93), CPR (AUROC, 0.80; 95% CI, 0.70-0.91), and platelet count (AUROC, 0.79; 95% CI, 0.69-0.90). The positive predictive values for the CPR and platelet count were 0.87 and 0.86, the negative predictive values were 0.64 and 0.63, the positive likelihood ratios were 3.06 and 2.76, and the negative likelihood ratios were 0.64 and 0.63, respectively. Based on positive and negative predictive values, the most accurate noninvasive tests were the CPR and platelet counts. CONCLUSIONS: Noninvasive tests such as CPR and platelet count can assist in triaging children for endoscopy to identify esophageal varices.