Background Vedolizumab is increasingly used off-label to treat children and adolescents with inflammatory bowel disease (IBD). In the absence of rigorous clinical trial experience, multicenter observational data are important to establish expectations for efficacy and safety. We examined one-year outcomes following vedolizumab therapy in a large multicenter paediatric IBD cohort. Methods We performed a retrospective study of 159 paediatric patients (4-17 years old) with IBD (78, Crohn disease [CD]; 81, ulcerative colitis/IBD-unspecified [UC/IBD-U]) treated with vedolizumab for 1-year at 8 paediatric medical centers in the United States. Demographics, clinical outcomes, laboratory data, and vedolizumab dosing were recorded. The primary outcome was corticosteroid free clinical remission at 1-year. Other measured outcomes were clinical remission at 12-weeks and/or 24 weeks, laboratory outcomes at 1 year, and endoscopy/histology results at 1 year. Results Among the 159 patients (mean age, 14.5±2.4 y; 86% anti-TNF experienced), 68/159 (43%) achieved corticosteroid free clinical remission at 1 year (CD 35/78, 45%; UC/IBD-U 33/81, 40%). Vedolizumab therapy failed and was discontinued in 33/159 (21%) patients prior to 1-year (CD, 18/78, 23%; UC/IBD-U, 15/81, 19%). While Week 12 clinical remission was not predictive of 1-year clinical remission in either CD or UC/IBD-U, week 24 clinical remission was predictive of 1-year clinical remission only in CD patients. No infusion reactions or serious side effects were noted. Conclusion Vedolizumab was safe and effective in this paediatric population with approximately 43% achieving corticosteroid free clinical remission at 1-year. Similar efficacy was noted in both CD and UC.

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Background and objectivesPosterior urethral valve is the most common cause of bladder outlet obstruction in infants. We aimed to describe the rate and timing of kidney-related and survival outcomes for children diagnosed with posterior urethral valves in United States children’s hospitals using the Pediatric Health Information System database.Design, setting, participants, & measurementsThis retrospective cohort study included children hospitalized between January 1, 1992 and December 31, 2006, who were in their first year of life, had a diagnosis of congenital urethral stenosis, and underwent endoscopic valve ablation or urinary drainage intervention, or died. Records were searched up to December 31, 2018 for kidney-related mortality, placement of a dialysis catheter, and kidney transplantation. Cox regression analysis was used to identify risk factors, and Kaplan–Meier survival analysis used to determine time-to-event probability. Subgroup survival analysis was performed with outcomes stratified by the strongest identified risk factor.ResultsIncluded were 685 children hospitalized at a median age of 7 (interquartile range, 1–37) days. Thirty four children (5%) died, over half during their initial hospitalization. Pulmonary hypoplasia was the strongest risk factor for death (hazard ratio, 7.5; 95% confidence interval [95% CI], 3.3 to 17.0). Ten-year survival probability was 94%. Fifty-nine children (9%) underwent one or more dialysis catheter placements. Children with kidney dysplasia had over four-fold risk of dialysis catheter placement (hazard ratio, 4.6; 95% CI, 2.6 to 8.1). Thirty-six (7%) children underwent kidney transplant at a median age of 3 (interquartile range, 2–8) years. Kidney dysplasia had a nine-fold higher risk of kidney transplant (hazard ratio, 9.5; 95% CI, 4.1 to 22.2).ConclusionsPatients in this multicenter cohort with posterior urethral valves had a 5% risk of death, and were most likely to die during their initial hospitalization. Risk of death was higher with a diagnosis of pulmonary hypoplasia. Kidney dysplasia was associated with a higher risk of need for dialysis/transplant.PodcastThis article contains a podcast at https://www.asn-online.org/media/podcast/CJASN/2019_10_03_CJN04350419.mp3

Background The increasing incidence of pediatric inflammatory bowel disease (IBD), along with more extensive and severe disease in children, raises concern for related health care expenditures. Aim The aim of this study was to quantify and characterize costs of pediatric IBD in the year following diagnosis. Methods We identified all patients diagnosed with IBD at Connecticut Children’s Medical Center in 2016 with a minimum of 1 year follow-up. Clinical and demographic factors were recorded at diagnosis. We examined paid service and professional costs related to outpatient medications and infusions, outpatient procedures and radiology imaging, inpatient services, and outpatient visits. Actual dollar reimbursements were from private and public payers. Data is reported as mean ± SD and median (IQR). Results First-year cost data were collected on 67 patients (43 Crohn’s disease [CD], 24 ulcerative colitis [UC], mean age 13 years [SD 3.22]) revealing a mean cost of $45,753 (SD $37,938), with $43,095 (SD $30,828) for CD and $50,516 (SD $48,557) for UC. Severe CD (n = 11) had a mean cost of $71,176 (SD $43,817) and severe UC (n = 5) $134,178 (SD $40,920). Patients receiving infusion therapy had a mean cost of $59,376 (SD $38,724) compared with $27,903 (SD $28,795) for those not receiving infusions. Overall cost distribution showed 37% from infusion costs, 25% hospital costs, 18% outpatient procedures, 10% outpatient oral medications, 7% outpatient imaging, and 3% outpatient visits. Conclusions Infusion therapy is a key driver of first-year costs for children newly diagnosed with IBD. Understanding cost distribution in relation to disease presentation can be helpful to anticipate future related costs.

This pilot project was designed to (1) implement a mindfulness-based wellness curriculum for otolaryngology residents, (2) determine the impact of a mindfulness-based curriculum on resident mood, and (3) examine the use of mindfulness among otolaryngology residents. Otolaryngology residents participated in a 6-week course guided by the Headspace mindfulness mobile application. Resident use of mindfulness was measured by the validated Mindful Attention Awareness Scale (MAAS). Changes in mood before and after each session were assessed using the validated Positive and Negative Affect Schedule (PNAS). Residents reported a statistically significant decrease in postsession negative affect scores ( P < .001). A moderate positive correlation was noted between mindfulness scores and presession positive mood (Pearson r = 0.597, P < .001). This pilot study supports the feasibility and impact of including mindfulness training as part of a resident wellness curriculum.

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Background: Ileocolonoscopy (IC) detects mucosal inflammation and magnetic resonance enterography (MRE) detects transmural inflammation in Crohn disease (CD). We aimed to evaluate the relationship between the simplified magnetic resonance index of activity (MARIAs) and measures of inflammation by IC in children with newly diagnosed CD. Methods: Retrospective review of 140 patients 6-18 years of age with CD who had baseline IC and MRE within 5 weeks of diagnosis. MARIAs was calculated for each intestinal segment (terminal ileum [TI], ascending colon, transverse colon, descending colon, sigmoid colon, rectum), defined asSensitivity and specificity were derived using receiver operating characteristic (ROC) curves to compare MARIAs to IC findings. Results: Using IC as the reference standard, the cutoff MARIAs !1 identified TI segments with active inflammation with 84% sensitivity, 73% specificity, 85% positive predictive value (PPV), 70% negative predictive value (NPV), and area under the curve (AUC) 0.782 (95% confidence interval [CI] 0.689-0.876). The cutoff MARIAs !2 identified TI segments with severe lesions with 87% sensitivity, 76% specificity, 87% PPV, 76% NPV, and AUC 0.814 (95% CI 0.712-0.916). There was poor sensitivity for all colonic segments. Conclusion: The MARIAs is feasible and accurate in reflecting disease activity in the TI, but not in the colon, in children with newly diagnosed CD. Although the MARIAs may be useful for monitoring TI disease activity over time, full assessment continues to require both IC and MRE.

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