Utilization of Segmental Grafts Is Associated With Higher Transplant Rates in Pediatric Patients.

INTRODUCTION: In July 2017, a policy to increase the use of segmental grafts (SGs) was implemented at our institution. The aim was to compare changes in waitlist activity after implementation of this policy. METHODS: A single-center, retrospective study. Pediatric patients on the liver waiting list between January 2015 and December 2019 were screened. Patients were classified as receiving a liver transplant (LT) before (Period 1) or after (Period 2) policy changes. Primary end points were transplant rates and time to transplant. RESULTS: Sixty five first LT performed on 65 patients were included. Twenty nine LT were performed during Period 1 and 36 during Period 2. More than half (55%) of LT in Period 2 were SG, compared to 10.3% in Period 1 (P < 0.001). Forty nine and 56 pediatric candidates on the waiting list accounted for 38.78 and 24.48 person-years during Period 1 and Period 2, respectively. Transplant rates per 100 person-years on the waiting list increased from 85.09 during Period 1 to 187.87 in Period 2 (Rate ratio: 2.20; P < 0.001). Median time to receive a LT decreased from 229 d in Period 1 to 75 d during Period 2 (P = 0.013). One-year patient survival rates were 96.6% in Period 1 and 95.7% in Period 2. One-year graft survival rates were 89.7% and 88% in Period 1 and Period 2, respectively. CONCLUSIONS: A policy to increase the use of SG was associated with significantly higher transplant rates and lower waiting times. Implementation of this policy can be done successfully with no observed negative impact on patient and graft survival.

Fractionated Bilirubin Among 252 892 Utah Newborns with and Without Biliary Atresia: A 15-year Historical Birth Cohort Study.

OBJECTIVES: To determine whether neonatal conjugated or direct bilirubin levels were elevated in infants with biliary atresia (BA) and to estimate the number of newborns who would have positive screens in the nursery necessitating repeat testing after discharge. STUDY DESIGN: We used administrative data from a large integrated healthcare network in Utah to identify newborns who had a fractionated bilirubin recorded during birth admission from 2005 through 2019. Elevated conjugated bilirubin was defined as greater than 0.2 mg/dL and direct bilirubin was defined as greater than 0.5 mg/dL (>97.5th percentile for the assays). We performed simulations to estimate the anticipated number of false-positive screens. RESULTS: There were 32 cases of BA and 468 161 live births during the study period (1/14 700). There were 252 892 newborns with fractionated bilirubin assessed, including 26 of those subsequently confirmed to have BA. Conjugated or direct bilirubin was elevated in all 26 infants with BA and an additional 3246 newborns (1.3%) without BA. Simulated data suggest 9-21 per 1000 screened newborns will have an elevated conjugated or direct bilirubin using laboratory-based thresholds for a positive screen. Screening characteristics improved with higher thresholds without increasing false-negative tests. CONCLUSIONS: This study validates the previous findings that conjugated or direct bilirubin are elevated in the newborn period in patients with BA. A higher threshold for conjugated bilirubin improved screening performance. Future studies are warranted to determine the optimal screening test for BA and to assess the effectiveness and cost-effectiveness of implementing such a program.

Severe Acute Respiratory Syndrome Coronavirus-2 Infection in Children With Liver Transplant and Native Liver Disease: An International Observational Registry Study.

OBJECTIVE: Increased mortality risk because of severe acute respiratory syndrome coronavirus-2 (SARS-CoV2) infection in adults with native liver disease (LD) and liver transplant (LT) is associated with advanced age and comorbid conditions. We aim to report outcomes for children with LD and LT enrolled in the NASPGHAN/SPLIT SARS-CoV2 registry. METHODS: In this multicenter observational cohort study, we collected data from 91 patients <21 years (LD 44, LT 47) with laboratory-confirmed SARS-CoV2 infection between April 21 and September 17, 2020. RESULTS: Patients with LD were more likely to require admission (70% vs 43% LT, P = 0.007) and pediatric intensive care unit (PICU) management (32% vs 4% LT, P = 0.001). Seven LD patients required mechanical ventilation (MV) and 2 patients died; no patients in the LT cohort died or required MV. Four LD patients presented in pediatric acute liver failure (PALF), 2 with concurrent multisystem inflammatory syndrome in children (MIS-C); all recovered without LT. Two LD patients had MIS-C alone and 1 patient died. Bivariable logistic-regression analysis found that patients with nonalcoholic fatty LD (NAFLD) (odds ratio [OR] 5.6, P = 0.02) and LD (OR 6.1, P = 0.01, vs LT) had higher odds of severe disease (PICU, vasopressor support, MV, renal replacement therapy or death). CONCLUSIONS: Although not directly comparable, LT recipients had lower odds of severe SARS-CoV2 infection (vs LD), despite immunosuppression burden. NAFLD patients reported to the registry had higher odds of severe SARS-CoV2 disease. Future controlled studies are needed to evaluate effective treatments and further stratify LD and LT patients with SARS-CoV2 infection.

Revision of meso-Rex bypass utilizing a collateral vein in a patient with portal steal phenomenon after liver transplant: A case report.

INTRODUCTION: In children with extrahepatic portal vein obstruction or those who develop portal vein thrombosis after liver transplant, the use of Meso-Rex Bypass (MRB) creates a more physiological state by redirecting mesenteric blood flow back into the intrahepatic portal system via a venous conduit. PRESENTATION OF CASE: A 3-year-old female with biliary atresia associated with polysplenia syndrome and a surgical history of Kasai portoenterostomy procedure, and an ABO incompatible whole liver transplant. Within a year after transplant she presented with prehepatic portal hypertension, that was treated with MRB using a deceased donor ABO compatible iliac vein as conduit. Six months later, she was taken to the operating room for bypass revision, during the procedure the MRB showed no flow and no thrombus, and a large splenorenal collateral vein that was causing a portal perfusion steal phenomenon was observed. After dissecting the collateral vein, an 8 cm x8 mm segment of this vessel was used as an autologous conduit to re-do the Rex. DISCUSSION: Failed of MRB can be attributed to portal steal phenomenon, hypercoagulable disorders, bypass contraction or kinking. In this case we believe the culprit to be the former. When there is a history of longstanding portal hypertension, large collaterals develop; thus, intraoperative portal vein flow measurement is critical and ligation of large collaterals during liver transplantation and MRB should be performed to avoid portal steal phenomenon postprocedure. CONCLUSION: Using a collateral vein as an alternative autologous venous conduit is a feasible option that can have durable success.

The Impact of Increased Allocation Priority for Children Awaiting Liver Transplant: A Liver Simulated Allocation Model (LSAM) Analysis.

OBJECTIVE: The aim of the study was to investigate the impact of prioritizing infants, children, adolescents, and the sickest adults (Status 1) for deceased donor livers. We compared outcomes under two "SharePeds" allocation schema, which prioritize children and Status 1 adults for national sharing and enhanced access to pediatric donors or all donors younger than 35 years, to outcomes under the allocation plan approved by the Organ Procurement and Transplant Network in December 2017 (Organ Procurement and Transplantation Network [OPTN] 12-2017). METHODS: The 2017 Liver Simulated Allocation Model and Scientific Registry of Transplant Recipients data on all US liver transplant candidates and liver offers 7/2013 to 6/2016 were used to predict waitlist deaths, transplants, and post-transplant deaths under the OPTN 12-2017 and SharePeds schema. RESULTS: Prioritizing national sharing of pediatric donor livers with children (SharePeds 1) would decrease waitlist deaths for infants (<2 years, P = 0.0003) and children (2-11 years, P = 0.001), with no significant change for adults (P = 0.13). Prioritizing national sharing of all younger than 35-year-old deceased donor livers with children and Status 1A adults (SharePeds 2) would decrease waitlist deaths for infants, children, and all Status 1A/B patients (P < 0.0001 for each). SharePeds 1 and 2 would increase the number of liver transplants done in infants, children, and adolescents compared to the OPTN-2017 schema (P < 0.00005 for all age groups). Both SharePeds schema would increase the percentage of pediatric livers transplanted into pediatric recipients. CONCLUSIONS: Waitlist deaths could be significantly decreased, and liver transplants increased, for children and the sickest adults, by prioritizing children for pediatric livers and with broader national sharing of deceased donor livers.

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.

Identification of Pediatric Patients With Celiac Disease Based on Serology and a Classification and Regression Tree Analysis.

BACKGROUND & AIMS: Celiac disease is detected using serology and endoscopy analyses. We used multiple statistical analyses of a geographically isolated population in the United States to determine whether a single serum screening can identify individuals with celiac disease. METHODS: We performed a retrospective study of 3555 pediatric patients (18 years old or younger) in the intermountain West region of the United States from January 1, 2008, through September 30, 2013. All patients had undergone serologic analyses for celiac disease, including measurement of antibodies to tissue transglutaminase (TTG) and/or deamidated gliadin peptide (DGP), and had duodenal biopsies collected within the following year. Modified Marsh criteria were used to identify patients with celiac disease. We developed models to identify patients with celiac disease using logistic regression and classification and regression tree (CART) analysis. RESULTS: Single use of a test for serum level of IgA against TTG identified patients with celiac disease with 90% sensitivity, 90% specificity, a 61% positive predictive value (PPV), a 90% negative predictive value, and an area under the receiver operating characteristic curve value of 0.91; these values were higher than those obtained from assays for IgA against DGP or IgG against TTG plus DGP. Not including the test for DGP antibody caused only 0.18% of celiac disease cases to be missed. Level of TTG IgA 7-fold the upper limit of normal (ULN) identified patients with celiac disease with a 96% PPV and 100% specificity. Using CART analysis, we found a level of TTG IgA 3.2-fold the ULN and higher to most accurately identify patients with celiac disease (PPV, 89%). Multivariable CART analysis showed that a level of TTG IgA 2.5-fold the ULN and higher was sufficient to identify celiac disease in patients with type 1 diabetes (PPV, 88%). Serum level of IgA against TTG in patients with versus those without trisomy 21 did not affect diagnosis predictability in CART analysis. CONCLUSIONS: In a population-based study, we found that serum level of IgA against TTG can identify patients with celiac disease with PPVs of about 90%. Predictive values increase greatly when levels are markedly above the ULN or when the assay is used in combination with other variables. Measurement of IgG against TTG or DGP does not increase the accuracy of detection of celiac disease based against TTG IgA levels. There is a low risk of false-positive results from serologic analysis in patients with type I diabetes or persistent increases in antibody against TTG on repeat testing.

Perceptions of gender equality in work-life balance, salary, promotion, and harassment: results of the NASPGHAN task force survey.

OBJECTIVES: Gender equality in the workplace has not been described in pediatric gastroenterology. METHODS: An electronic survey that explored perceptions of career parity, work-life balance, and workplace harassment was sent to all members of the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition. Reponses were anonymous. RESULTS: Of the 303 respondents (21%), there was an even distribution across geographic region, age, and gender (54% men). Gender affected perception of salary and promotion; 46% of men but only 9% of women feel that "women earn the same as men" (P < 0.001). Similarly, 48% of men but only 12% of women feel that "women rise at the same rate as men" (P < 0.001). Both genders of academic practice respondents, compared with other practice models, perceived men were promoted more quickly than women (P = 0.008). Women had higher dissatisfaction with mentoring than men (29% vs 13%, P = 0.03). Significantly more men than women reported spouses with "flexible jobs" (35% vs 14%, P < 0.001). Having a spouse with "flexible job" or having children (preschool or school age), however, did not affect satisfaction with work-life balance for either gender. Overall, women are more likely to be dissatisfied with work-life balance than men (P = 0.046). CONCLUSIONS: Satisfaction with work-life balance is lower among women versus men pediatric gastroenterologists, but does not correlate with flexibility of spouse's job or caring for young children. Gender-divergent perception of promotion, parity of compensation, and mentoring requires further investigation.

Renal replacement therapy in infants and children with hepatorenal syndrome awaiting liver transplantation: a case-control study.

Limited data on short- and long-term outcomes of renal replacement therapy (RRT) in pediatric liver transplantation (LT) patients exist. We evaluated risk factors for RRT in pediatric LT recipients with hepatorenal syndrome (HRS) and described the outcomes. We performed a single-center, case-control study of LT recipients who required RRT for HRS from 1999 to 2011. Three controls who did not receive RRT were matched with each case on the basis of age, diagnosis, and LT date. We identified 8 recipients among 133 recipients of 152 LT cases [6%, 95% confidence interval = 2%-10%; mean age = 7.7 years, range = 0.5-19.8 years) who required RRT before LT for HRS. Four patients were <1 year old and weighed 5.6 to 6.6 kg. Biliary atresia was the most common LT indication. Cases had higher Model for (Pediatric) End-Stage Liver Disease scores at listing (26 versus 16, P = 0.01) and lower glomerular filtration rates (GFRs; 15 versus 102 mL/minute/1.73 m(2) , P < 0.001) at RRT initiation or LT. Ascites, gastrointestinal bleeding, and infections occurred more commonly among cases: (100% versus 54%, P = 0.03; 100% versus 46%, P = 0.01; and 88% versus 33%, P = 0.01, respectively). Cases also experienced toxic vancomycin troughs more frequently (38% versus 0%, P = 0.01) and received RRT for a median of 21 days (range = 3-355 days). The case mortality rate was 37.5% (3/8 at 1, 26, and 346 days after LT) and 0% for controls. The 4 infants required 0 to 3 dialysis catheter replacements during RRT. Cases and controls had similar median follow-ups [3.2 years (range = 1.5-7.6 years) versus 4.9 years (range = 0.2-11 years), P = 0.29]. After LT, they also had similar GFRs (83 versus 99 mL/minute/1.73 m(2) at 1 month, P = 0.19; 80 versus 107 mL/minute/1.73 m2 at 1 year, P > 0.99; and 97 versus 114 mL/minute/1.73 m2 at the most recent follow-up, P = 0.09). The case survival rates were 75% and 63% at 1 month and 1 year, respectively; 4 cases required antihypertensives and diuretics 1 month after LT, but at the last follow-up, only 1 case required antihypertensive therapy, and none required diuretics. In conclusion, pediatric patients with HRS, including infants, benefit from RRT. Although HRS decreases survival, patients with HRS who undergo LT generally recover renal function within 1 month that persists during long-term follow-up.

Cholangiocarcinoma and high-grade dysplasia in young patients with primary sclerosing cholangitis.

INTRODUCTION: Cholangiocarcinoma (CCA) is very often an adulthood disease with primary sclerosing cholangitis (PSC) as one of the risk factors. It is rarely seen in the pediatric population, and when it is diagnosed before adulthood, it can be associated with PSC as well as HIV infection, biliary atresia, radiation therapy, and choledochal cyst. Although there have been some case reports of pediatric CCA, cases of childhood CCA associated with PSC are still relatively rare. AIM: To describe the clinical and pathologic features of CCA in pediatric patients with previously diagnosed PSC. METHODS: Retrospective study RESULTS: Four patients with PSC (age range 15-18, mean 17 years) were included in this study. All patients underwent ERCP for diagnosis. Tissue samples obtained included routine cytology and FISH. ERCP was used to target sites for tissue acquisition in all patients. 3/4 of patients have inflammatory bowel disease (two Crohn's disease and one ulcerative colitis). Alkaline phosphatase was elevated in 3/4 patients, aspartate aminotransferase/alanine aminotransferase were elevated in 2/4 patients, and total bilirubin/direct bilirubin were elevated in 2/4 patients. 4/4 patients had positive FISH studies, and 3/4 patients had brush cytology concerning for CCA. 2/4 patients received chemotherapy, one patient underwent orthotopic liver transplant, and one patient underwent Whipple procedure. Two patients died soon after being diagnosed. CONCLUSIONS: Young patients with PSC can develop CCA. This finding has implications for both screening and surveillance for cancer in pediatric patients with PSC.

Outcome after discontinuation of immunosuppression in children with autoimmune hepatitis: a population-based study.

OBJECTIVE: To assess sustained immunosuppression-free remission (SIFR) in children with autoimmune hepatitis (AIH). STUDY DESIGN: We retrospectively reviewed all children with AIH in the region between 1986 and 2011 using a population-based methodology. RESULTS: We identified 56 children with AIH (62.5% females; median age, 11.1 years [IQR, 5.7-14.4 years], followed for a median of 5.6 years [IQR, 2.8-8.6 years]). Liver disease was characterized by type II AIH in 8.9%, cirrhosis in 14.0%, and primary sclerosing cholangitis in 21.4%. Coexisting nonhepatic immune-mediated diseases occurred in 37.5%. Biochemical remission on immunosuppressive therapy was achieved in 76.4% of all patients with AIH at a median of 1.2 years (IQR, 0.4-3.6 years); 23.1% of these patients experienced a subsequent relapse. Discontinuation of all immunosuppressive medications was attempted in 16 patients and was successful in 14 patients (87.5%) with type 1 AIH (median age at discontinuation, 8.9 years [IQR, 3.5-17.9 years], treated for a median of 2.0 years [IQR, 1.3-3.5 years] after diagnosis), with SIFR occurring at a median of 3.4 years (IQR, 2.6-5.8 years) of follow-up. Excluding patients with inflammatory bowel disease who received immunosuppressive therapy independent of their liver disease, the probability of achieving SIFR within 5 years of diagnosis of AIH was 41.6% (95% CI, 25.3%-62.9%). Baseline patient characteristics associated with an inability to achieve biochemical remission on immunosuppression or SIFR were elevated international normalized ratio, positive antineutrophil cytoplasmic antibody titer, cirrhosis, and a nonhepatic autoimmune disorder. CONCLUSION: We found a high rate of successful discontinuation of all immunosuppressive medications in carefully selected patients with AIH in a population-based cohort. SIFR is an achievable goal for children with AIH, particularly those with type I disease in stable biochemical remission on immunosuppressive therapy.

Neonatal Cholestasis Caused by Undiagnosed Maternal Graves' Disease.

Neonatal cholestasis results from a variety of etiologies, including anatomic, infectious, and metabolic abnormalities. Hyperthyroidism, in contrast to hypothyroidism, is infrequently associated with neonatal cholestasis. Newborn screening is an important tool to detect newborn metabolic disorders, including thyroid dysfunction. However, one must exercise caution when interpreting these reports; typically only high thyroid stimulating hormone (TSH) levels are flagged as abnormal, while low or undetectable levels may not be. We present a unique case of cholestasis in a hyperthyroid neonate of an untreated, undiagnosed mother with Graves' disease; the infant's metabolic screen was not flagged as abnormal.

Effect of decreased parenteral soybean lipid emulsion on hepatic function in infants at risk for parenteral nutrition-associated liver disease: a pilot study.

PURPOSE: We performed a pilot trial to compare reduced dose versus standard soybean lipid emulsion in neonates at risk for parenteral nutrition-associated liver disease. METHODS: A prospective randomized controlled trial was performed (2009-2011) enrolling surgical patients ≥ 26 weeks' gestation anticipated to require >50% of daily caloric intake from parenteral nutrition (PN) for at least 4 weeks. Randomization occurred into either reduced (1.0 g/kg/day) or standard (3g/kg/day) groups. Co-primary outcomes for interpretation of the results were conjugated bilirubin and total bile acids. Additional outcomes included ALT, AST, GGT, alkaline phosphatase, growth, and essential fatty acid levels. Outcomes were compared between treatment groups using Wilcoxon rank sums tests. RESULTS: Twenty-eight patients (47% enrollment rate) were included in the study with an average treatment duration of 5.4 weeks. Groups had similar PN calories and protein intake throughout the study. Total increase from baseline was smaller in the reduced vs. standard group for conjugated bilirubin (p=0.04) and total bile acids (p=0.02). Weight z-score increased more in the standard group, and no patient experienced essential fatty acid deficiency. CONCLUSION: Markers of cholestasis rose at a slower rate using reduced lipid doses. This pilot study demonstrates feasibility and need for a larger study evaluating the effects of reduced lipids in patients at risk for developing parenteral nutrition-associated liver disease.

Primary sclerosing cholangitis, autoimmune hepatitis, and overlap in Utah children: epidemiology and natural history.

UNLABELLED: The epidemiology and natural history of pediatric primary sclerosing cholangitis (PSC), autoimmune sclerosing cholangitis (ASC), and autoimmune hepatitis (AIH) are not well characterized. Using multiple, overlapping search strategies followed by a detailed records review, we identified all cases of pediatric PSC, ASC, AIH, and inflammatory bowel disease (IBD) in a geographically isolated region of the United States. We identified 607 cases of IBD, 29 cases of PSC, 12 cases of ASC, and 44 cases of AIH. The mean age at diagnosis was 13.0 years for PSC, 11.3 years for ASC, and 9.8 years for AIH. The incidence and prevalence of PSC, ASC, and AIH were 0.2 and 1.5 cases, 0.1 and 0.6 cases, and 0.4 and 3.0 cases per 100,000 children, respectively. The mean duration of follow-up was 5.9 years. The probability of developing complicated liver disease within 5 years of the diagnosis of liver disease was 37% [95% confidence interval (CI) = 21%-58%] for PSC, 25% (95% CI = 7%-70%) for ASC, and 15% (95% CI = 7%-33%) for AIH. The 5-year survival rates with the native liver were 78% (95% CI = 54%-91%) for PSC, 90% (95% CI = 47%-99%) for ASC, and 87% (95% CI = 71%-95%) for AIH. Cholangiocarcinoma developed in 2 of the 29 PSC patients (6.9%). PSC occurred in 9.9% of patients with ulcerative colitis (UC) and in 0.6% of patients with Crohn's disease (CD). ASC occurred in 2.3% of UC patients and 0.9% of CD patients. AIH occurred in 0.4% of UC patients and in 0.3% of CD patients. Liver disease occurred in 39 of 607 IBD patients (6.4%) overall. CONCLUSION: Immune-mediated liver diseases are important sources of morbidity in children. Using a population-based design, this study quantifies the burden and natural history of immune-mediated liver disease in children.

Liver transplantation in a 7-month-old girl with Caroli's disease.

Caroli's disease (including Caroli's syndrome) is a rare autosomal recessive disorder of the liver characterized by diffuse cystic dilatation of the intrahepatic bile ducts. The disease may present at any age and is characterized by recurrent episodes of biliary obstruction, cholangitis, hepaticolithiasis, and liver abscesses. Caroli's syndrome is further associated with congenital hepatic fibrosis and portal hypertension. Patients with recurrent complications or cirrhosis may die because of recurrent infection, portal hypertension, liver failure, or cholangiocarcinoma. Liver transplantation is the treatment of choice for these complicated patients. Here we describe the youngest reported patient with Caroli's syndrome treated successfully using liver transplantation and review the recent literature.

Clinical variables as prognostic tools in pediatric-onset ulcerative colitis: a retrospective cohort study.

BACKGROUND: Clinical variables may identify a subset of patients with pediatric-onset ulcerative colitis (UC) (≤18 years at diagnosis) at risk for adverse outcomes. We postulated that routinely measured clinical variables measured at diagnosis would predict colectomy in patients with pediatric-onset UC. METHODS: We conducted a chart review of patients with pediatric-onset UC at a single center over a 10-year period. We compared patients with and without colectomy across several variables, used proportional hazards regression to adjust for potential confounders, and assessed the ability of a UC risk score to predict colectomy. RESULTS: Among 470 patients with inflammatory bowel disease ICD9-coded encounters, 155 patients had UC and 135 were eligible for analysis. The 1- and 3-year colectomy rates were 16.7% (95% confidence interval [CI]: 11.0%-24.8%) and 35.6% (26.7%-45.4%). White blood cell (WBC) count and hematocrit measured at diagnosis were associated with colectomy at 3 years, even after correcting for potential confounding variables. A UC Risk Score derived from the WBC count and hematocrit was strongly associated with colectomy risk, with a high negative predictive value (NPV) for colectomy at 1 and 3 years (NPV = 0.95 and 0.89, respectively), but low positive predictive value (PPV = 0.22 and 0.38, respectively). CONCLUSIONS: A risk score calculated from WBC and hematocrit measured at diagnosis was associated with, but incompletely predictive of, colectomy in pediatric-onset UC. These data suggest 1) routinely measured clinical variables may have a prognostic role in risk stratification, and 2) multicenter prospective studies are needed to optimize risk stratification in pediatric UC. Our findings have impact on the design of such studies.

Novel screening assay performance in pediatric celiac disease and adult dermatitis herpetiformis.

OBJECTIVES: : Several serologic assays are commercially available to aid in the diagnosis of gluten-sensitive enteropathy (GSE). Our objective in this study was to assess the performance of a novel combined antigen-screening assay for GSE. PATIENTS AND METHODS: : Deidentified sera from 111 pediatric patients suspected of having celiac disease (CD), 130 adults diagnosed with dermatitis herpetiformis (DH), and 77 pediatric and 49 adult normal controls were included in the study. Sera from 10 patients submitted to our laboratory for GSE testing with IgA deficiency and IgG antibodies against 1 or more of the traditional serologic markers associated with GSE were also included. All sera were screened for antibodies (IgA and IgG) against tissue transglutaminase (tTG) and deamidated gliadin peptides (DGP) by enzyme immunoassay (EIA) in a single test well. In addition, all sera were assessed for each individual marker and isotype using separate EIAs. RESULTS: : The IgA/IgG anti-tTG/DGP EIA screen was 92.6% sensitive and 94.3% specific in pediatric CD and detected 1 patient (Marsh 3c) who was IgA anti-tTG negative; this patient was not IgA deficient (<7.0 mg/dL). All 10 IgA-deficient sera gave positive results by the tTG/DGP EIA screen. Sensitivity and specificity of the tTG/DGP EIA screen in retrospective and prospective DH were 65% and 100% versus 62% and 100%, respectively. CONCLUSIONS: : The new IgA/IgG anti-tTG/DGP EIA screen was slightly more sensitive than IgA anti-tTG alone in pediatric CD. This novel screening assay may allow the current recommendation of measuring total serum IgA in suspected GSE patients to be eliminated.

Elimination of soybean lipid emulsion in parenteral nutrition and supplementation with enteral fish oil improve cholestasis in infants with short bowel syndrome.

BACKGROUND: Parenteral nutrition-associated liver disease (PNALD) is a potentially fatal complication for children with intestinal failure. Fish oil-based lipid emulsions have shown promise for the treatment of PNALD but are not readily available. Six cases are presented in which cholestasis resolved after soybean lipid emulsion (SLE) was removed from parenteral nutrition (PN) and enteral fish oil was given. METHODS: A retrospective review at a tertiary children's hospital (July 2003 to August 2008) identified 6 infants with intestinal failure requiring PN for >6 months who developed severe hepatic dysfunction that was managed by eliminating SLE and providing enteral fish oil. RESULTS: Twenty-three infants with short bowel syndrome requiring prolonged PN developed cholestasis. SLE was removed in 6 of these patients, and 4 of the 6 received enteral fish oil. Standard PN included 2-3 g/kg/d SLE with total PN calories ranging from 57 to 81 kcal/kg/d at the time of SLE removal. Hyperbilirubinemia resolved after elimination of SLE within 1.8-5.4 months. Total PN calories required to maintain growth generally did not change. CONCLUSIONS: Temporary elimination of SLE and supplementation with enteral fish oil improved cholestasis in PN-dependent infants. Further trials are needed to evaluate this management strategy.