Assessment of telemedicine versus in-person care in managing abdominal pain in children during the COVID-19 pandemic.

OBJECTIVES: The COVID-19 pandemic has led to a dramatic increase in telemedicine care delivery. This raises the question of whether the visit type affects the care provided to patients in the pediatric gastroenterology clinic. The aim of this study is to assess whether diagnostic, treatment, and outcome measures differ between telemedicine and in-person visits in patients seen in pediatric gastroenterology clinics for the chief complaint of abdominal pain. METHODS: We conducted a retrospective analysis of patients aged 0-22 who underwent their initial pediatric gastroenterology clinic visit, for abdominal pain, between March and September 2020 (n = 1769). The patients were divided into two groups: in-person or telemedicine. Clinical outcome measures were compared from the initial gastroenterology visit and followed for a total of 3 months. RESULTS: There was an increase number of images (M = 0.52 vs. 0.36, p < 0.001), labs (M = 4.87 vs. 4.05; p = 0.001), medications (M = 2.24 vs. 1.67; p < 0.001), and referrals (M = 0.70 vs. 0.54; p < 0.001) performed per visit in the in-person group. Electronic communications (3.97 vs. 5.12 p <0.003) was less frequent after in-person visits. There was no difference in number of procedures (M = 0.128 vs. 0.122, p = 0.718), emergency room visits (M = 0.037 vs. 0.017 p = 0.61), follow-up visits (M = 1.21 vs. 1.21 p = 0.922), or telephone encounters (M = 1.21 vs. 1.12 p = 0.35) between the two groups. CONCLUSION: Telemedicine utilizes less resources while having comparable outcome measurements in children with a chief complaint of abdominal pain.

Fat Soluble Vitamin Assessment and Supplementation in Cholestasis.

Malnutrition in children with chronic cholestasis is a prevalent issue and a major risk factor for adverse outcomes. Fat soluble vitamin (FSV) deficiency is an integral feature of cholestatic disease in children, often occurring within the first months of life in those with neonatal cholestasis and malnutrition. This review focuses on FSVs in cholestasis, with particular emphasis on a practical approach to surveillance and supplementation that includes approaches that account for differing local resources. The overarching strategy suggested is to incorporate recognition of FSV deficiencies in cholestatic children in order to develop practical plans for close monitoring and aggressive FSV repletion. Routine attention to FSV assessment and supplementation in cholestatic infants will reduce long periods of inadequate levels and subsequent adverse clinical sequalae.

Consensus guidelines: best practices for detection, assessment and management of suspected acute drug-induced liver injury occurring during clinical trials in adults with chronic cholestatic liver disease.

BACKGROUND: Improved knowledge of the molecular pathophysiology and immunopathogenesis of cholestatic liver diseases in recent years has led to an increased interest in developing novel therapies. Patients with cholestatic liver disease often require different approaches to assessment and management of suspected drug-induced liver injury (DILI) compared to those with healthy livers and those with parenchymal liver diseases. At present, there are no regulatory guidelines or society position papers, that systematically address best practices pertaining to detection of DILI in these patients. AIMS: To outline best practices for detection, assessment and management of suspected acute DILI during clinical trials in adults with the cholestatic liver diseases - Primary Biliary Cholangitis (PBC) and Primary Sclerosing Cholangitis (PSC). METHODS: This is one of the several papers developed by the IQ DILI Initiative, which is comprised of members from 16 pharmaceutical companies, in collaboration with DILI experts from academia and regulatory agencies. The contents are the result of an extensive literature review, as well as in-depth discussions among industry, regulatory and academic DILI experts, to achieve consensus recommendations on DILI-related issues occurring during clinical trials for cholestatic liver diseases. RESULTS: Recommended best practices are outlined pertaining to hepatic eligibility criteria, monitoring of liver tests, approach to a suspected DILI signal, and hepatic discontinuation rules. CONCLUSIONS: This paper provides a framework for the approach to detection, assessment and management of suspected acute DILI occurring during clinical trials in adults with cholestatic liver disease.

Trajectory of adherence behavior in pediatric and adolescent liver transplant recipients: The medication adherence in children who had a liver transplant cohort.

Knowledge of the longterm trajectory of nonadherence to immunosuppressants can inform decisions regarding organ allocation, adherence monitoring, and intervention efforts. The Medication Adherence in Children Who Had a Liver Transplant (MALT) prospective multisite study followed 400 pediatric and adolescent liver transplant recipients for 2 years, using the Medication Level Variability Index to monitor adherence. We hypothesized that adherence is an unstable (fluctuating) phenomenon: that patients who are adherent in year 1 may become nonadherent in year 2, and vice versa. However, we also hypothesized that a majority (more than 50%) of nonadherent patients remain nonadherent over time. We further hypothesized that the longer nonadherence lasts, the higher the likelihood of adverse events (rejection). Finally, we explored the effect of socioeconomic factors on the evolution of adherence over time. Most (59.7%) of the MALT patients who were nonadherent in year 1 remained so in year 2; 18.5% of patients who were adherent in year 1 became nonadherent in year 2. Only 4.4% of patients who were adherent in both year 1 and year 2 had a rejection, compared with 22.9% of patients who were nonadherent during 1 of the years, and 34.9% of those who were nonadherent in both years (P < 0.001), establishing a "dose-dependent" effect of adherence on transplant outcomes. Single-parent households were associated with worsening adherence. Our results suggest that good baseline adherence does not guarantee adherence later on, that nonadherence is likely to persist in the absence of interventions, and that monitoring of adherence and interventions to improve it should be expected to last for years if transplant outcomes are to be improved. Liver Transplantation 24 80-88 2018 AASLD.

Initial assessment of the infant with neonatal cholestasis-Is this biliary atresia?

INTRODUCTION: Optimizing outcome in biliary atresia (BA) requires timely diagnosis. Cholestasis is a presenting feature of BA, as well as other diagnoses (Non-BA). Identification of clinical features of neonatal cholestasis that would expedite decisions to pursue subsequent invasive testing to correctly diagnose or exclude BA would enhance outcomes. The analytical goal was to develop a predictive model for BA using data available at initial presentation. METHODS: Infants at presentation with neonatal cholestasis (direct/conjugated bilirubin >2 mg/dl [34.2 µM]) were enrolled prior to surgical exploration in a prospective observational multi-centered study (PROBE-NCT00061828). Clinical features (physical findings, laboratory results, gallbladder sonography) at enrollment were analyzed. Initially, 19 features were selected as candidate predictors. Two approaches were used to build models for diagnosis prediction: a hierarchical classification and regression decision tree (CART) and a logistic regression model using a stepwise selection strategy. RESULTS: In PROBE April 2004-February 2014, 401 infants met criteria for BA and 259 for Non-BA. Univariate analysis identified 13 features that were significantly different between BA and Non-BA. Using a CART predictive model of BA versus Non-BA (significant factors: gamma-glutamyl transpeptidase, acholic stools, weight), the receiver operating characteristic area under the curve (ROC AUC) was 0.83. Twelve percent of BA infants were misclassified as Non-BA; 17% of Non-BA infants were misclassified as BA. Stepwise logistic regression identified seven factors in a predictive model (ROC AUC 0.89). Using this model, a predicted probability of >0.8 (n = 357) yielded an 81% true positive rate for BA; <0.2 (n = 120) yielded an 11% false negative rate. CONCLUSION: Despite the relatively good accuracy of our optimized prediction models, the high precision required for differentiating BA from Non-BA was not achieved. Accurate identification of BA in infants with neonatal cholestasis requires further evaluation, and BA should not be excluded based only on presenting clinical features.

Liver transplantation for children with biliary atresia in the pediatric end-stage liver disease era: the role of insurance status.

Socioeconomic status influences health outcomes, although its impact on liver transplantation (LT) in children with biliary atresia (BA) is unknown. We hypothesized that governmental insurance [public insurance (PU)], rather than private insurance (PR), would be associated with poorer outcomes for children with BA. Children with BA who underwent first isolated LT between January 2003 and June 2011 were identified from United Network for Organ Sharing Standard Transplant Analysis and Research files. We identified 757 patients with PR and 761 patients with PU. The race/ethnicity distribution was significantly different between the groups (65% white, 12% black, and 10% Hispanic in the PR group and 33% white, 26% black, and 29% Hispanic in the PU group, P < 0.01). Wait-list mortality was higher for the PU group versus the PR group [46/1654 (2.7%) versus 29/1895 (1.5%), P < 0.01]. PR patients were older than PU patients at transplant (2.4 ± 4.5 versus 1.5 ± 3.0 years, P < 0.01). The donor types differed between the groups: 165 children (21.8%) in the PR group received living donor grafts, whereas 79 children (10.4%) in the PU group did (P < 0.01). The 1- and 5-year posttransplant patient survival rates were greater for the PR group versus the PU group (98.0% versus 94.1% at 1 year, P < 0.01; 97.8% versus 92.2% at 5 years, P < 0.01). Cox proportional hazards models revealed that the insurance type (PU), the donor type (deceased), and life support were significant risk factors for death. A separate analysis of deceased donor LT revealed that the PU group still had significantly worse patient and graft survival. In conclusion, PU coverage is an independent risk factor for significantly increased wait-list and posttransplant mortality in children with BA. Further studies are needed to unearth the reasons for these important differences in outcomes.

National and regional analysis of exceptions to the Pediatric End-Stage Liver Disease scoring system (2003-2004).

Since February 2002, the Pediatric End Stage Liver Disease (PELD) scoring system has been utilized as a means of prioritizing children for liver transplantation. The United Network for Organ Sharing database was queried to assess utilization of PELD in 2003 and 2004; 682 liver transplants were performed in pediatric recipients where the PELD score was potentially the primary determinant of liver allocation. In the majority of circumstances (53%) the actual calculated PELD score was not utilized to determine liver allocation. An exception to the PELD score was utilized in 24% of cases. An additional 29% of the children were listed as urgent (status 1) without having acute liver failure. There was considerable regional variability in the inability to utilize actual PELD scores for liver allocation to children. PELD utilization was higher in regions of the country where pediatric donor organs were more available, presumably because children have some priority for organs from pediatric donors. There were 87 deaths in children awaiting liver transplantation. The mean PELD score in children without acute liver failure or metabolic liver disease (n = 33) near the time of death was 24.2, which has a purported 3-month risk of mortality of less than 10%. In our opinion the assigned 3-month risk of mortality associated with PELD scores is understated. Three-month mortality risk is used to inter-convert the adult and pediatric scoring systems. Therefore exceptions to the scoring system are required when children compete with adults for donor organs. In conclusion, urgent reassessment of the PELD scoring system is needed to avoid morbidity and mortality in children.

Assessment of depression in medically ill children presenting to pediatric specialty clinics.

OBJECTIVE: Self-report measures of depression may be inaccurate when applied to medically ill children because of the overlap between medical and psychiatric symptoms. The American Academy of Pediatrics endorses the use of self-reports as diagnostic aids (used in patients who are suspected of having an emotional problem by their pediatricians). This approach has not been validated. We evaluated the diagnostic accuracy of the Children's Depression Inventory (CDI) in medically ill children who were referred for evaluation by their pediatricians. METHOD: The evaluation included the CDI and a best estimate diagnostic procedure that incorporated results from semistructured and other interviews. Evaluators were not aware of the CDI scores when making a diagnostic determination. RESULTS: One hundred twenty-five patients, 8-19 years old, were referred between September 2000 and May 2003. Eighty-one completed the diagnostic procedure. The CDI summary score performed well in identifying best estimate depressive disorder determinations in this cohort. A cutoff of 11 and above correctly identified 80% of cases, with a specificity of 70%. DISCUSSION: A self-report measure of depression (CDI) could be used as a diagnostic aid in medically ill children who are suspected of having an emotional problem by their pediatricians. This finding supports the "two-tier" depression screening approach endorsed by the American Academy of Pediatrics for use in pediatricians' offices.

Critical analysis of the pediatric end-stage liver disease scoring system: a single center experience.

The Pediatric End-Stage Liver Disease (PELD) scoring system is a new nationally utilized formula developed to provide a continuous numerical assessment of the risk of death in order to allocate livers to children for transplantation. A retrospective review of the clinical course of children undergoing liver transplantation at the Mount Sinai Medical Center was performed in order to assess the effectiveness of this scoring system in the first 24 months of its utilization. Forty-eight patients underwent liver transplantation with overall patient and graft survival rates of 98% and 96%, respectively. In 23 cases the PELD scoring system determined waiting time for transplantation. Of these 23 patients, 7 moved to the intensive care unit (ICU). Only 2 of 23 patients underwent transplantation with their actual PELD score. The rest required petition for exception (17) or status 1 listing (4). Significant morbidity occurred while awaiting transplantation: failure to thrive (78%), ascites (73%), hemorrhage (49%), infectious complications (39%), encephalopathy (30%), peritonitis (17%), pathologic bone fractures (13%), and hepatopulmonary syndrome (9%). In patients with PELD scores granted by exception the average score that did not yield a liver offer was 38 with an average waiting time of 55 days. At the time of transplantation actual PELD score averaged 22, while the petitioned score was 40. Based upon our center's initial experience, the current PELD scoring system is not adequate. Actual PELD scores did not lead to timely allocation of livers to children. It appears that this scoring system underestimates the near-term risk of death. Urgent reassessment is required to prevent potential morbidity and mortality in children. In conclusion the United Network for Organ Sharing policy that permits granting of exceptions has circumvented these problems with the PELD scoring system.