Fulminant Liver Failure in a Child With ß-Thalassemia on Deferasirox: A Case Report.

Deferesirox (DFX), an oral chelating agent, is used to treat chronic iron overload in several hematological diseases such as ß-thalassemia, sickle cell disease, and myelodysplastic anemia. DFX is generally well tolerated with the exception of gastrointestinal disturbances and rash, although cases of renal toxicity, as well as acute and chronic liver failure, have been reported in adults and children. Here we describe a 3-year-old girl with ß-thalassemia undergoing treatment with DFX who presented with acute liver failure and Fanconi's syndrome. It is important for pediatric gastroenterologists, hepatologists, and hematologists to be aware that the commonly used drug DFX can lead to acute liver failure in children, and liver function should be monitored closely in all patients taking DFX.

Transition of Inflammatory Bowel Disease Care: Assessment of Transition Readiness Factors and Disease Outcomes in a Young Adult Population.

BACKGROUND: Limited data exist on what factors impact transition readiness and how readiness impacts short-term disease outcomes. METHODS: Patients between the ages of 18 and 25 with an established inflammatory bowel disease diagnosis completed questionnaires at the time of an outpatient visit in the pediatric or adult setting, which included the Transition Readiness Assessment Questionnaire (TRAQ). After 6 months, electronic medical records were reviewed. RESULTS: A total of 95 patients were enrolled, 46 in the adult care setting and 49 in the pediatric care setting. Patients in the adult setting had a significantly higher overall TRAQ score compared with the pediatric setting (median: 4.42 [IQR: 3.9-4.6] versus 4.06 [IQR: 3.4-4.4], P < 0.001). Logistic regression analysis demonstrated that age was independently associated with higher TRAQ scores (odds ratio: 1.49; 95 confidence interval%, 1.1-2.02). Nonadherent patients scored lower on the Managing Medications subscale (median: 4.25 [IQR: 3.3-4.8] versus 4.75 [IQR: 4.3-5.0], P < 0.01). Logistic regression showed that patients who scored <4.75 on the Medication Management subscale were 3.8 times more likely to be nonadherent than patients who scored ≥4.75 (95% confidence interval, 1.4-10.3). This remained significant after adjusting for gender and age. During the 6-month follow-up period, 9/95 patients (10%) had hospitalizations or ED visits related to inflammatory bowel disease. There were no associations between TRAQ scores and hospitalizations/ED visits. CONCLUSIONS: Age is the primary factor that drives transition readiness. Our findings suggest that administering the medication management portion of the TRAQ can be used to identify patients at risk for nonadherence. Follow-up studies are needed to determine how readiness impacts long-term disease outcomes.

Home Nasogastric Feeds: Feeding Status and Growth Outcomes in a Pediatric Population.

BACKGROUND: Home enteral nutrition (HEN) is a safe method for providing nutrition to children with chronic diseases. Advantages of HEN include shorter hospitalizations, lower cost, and decreased risk of malnutrition-associated complications. Follow-up after hospital discharge on HEN is limited. The purpose of this study was to look at children discharged on nasogastric (NG) feeds to assess follow-up feeding status and impact on growth. METHODS: A retrospective chart review was conducted of pediatric patients discharged from Mount Sinai Medical Center on NG feeds between January 2010 and March 2013. RESULTS: A total of 87 patients were included. Average age was 1.2 years. The most common diagnoses were congenital heart disease (47%), metabolic disease (17%), neurologic impairment (10%), liver disease (9%), prematurity (8%), and inflammatory bowel disease (6%). At most recent follow-up, 44 (50.6%) were on full oral feeds, 8 (9.2%) were still on NG feeds, 9 (10.3%) had a gastrostomy tube placed, 9 (10.3%) were deceased, and 17 (19.5%) had transferred care or were lost to follow-up. Average time to discontinuation of NG feeds was 4.8 months. Change in body mass index from hospital discharge to follow-up visit 6 to 12 weeks after discharge was statistically significant, from a mean (SD) of 13.78 (2.82) to 14.58 (2.1) (P = .02). Change in weight z score was significant for neurologic impairment (-1.35 to -0.04; P = .03). Height z score change was significant for prematurity (-3.84 to -3.34; P = .02). There was no significant change in height or weight z scores for the other diagnoses. CONCLUSIONS: NG feeds can help to improve short-term growth after hospital discharge in children with chronic illnesses.

IgG4-sclerosing cholangitis in a pediatric patient.

IgG4 sclerosing cholangitis (IgG4-SC) is an immune-mediated process that results in inflammation and fibrosis of the pancreatobiliary tract. Although IgG4-SC is predominantly associated with autoimmune pancreatitis, IgG4-SC as its own entity can be difficult to diagnose. Patients with IgG4-SC are typically men over the age of 60, and present clinically with obstructive jaundice, abdominal pain, and weight loss. The diagnosis of IgG4-SC may be difficult to differentiate from primary sclerosing cholangitis (PSC) or cholangiocarcinoma. IgG4-SC is morphologically characterized by dense lymphoplasmacellular infiltration, particularly IgG4+ plasma cells and CD4+ T cells, extensive fibrosis in bile duct walls, and obliterative phlebitis. In contrast to PSC, those with IgG4-SC often have elevated serum IgG4 and can be successfully treated with immunosuppression. Here, we present the first reported case of IgG4-SC in a pediatric patient with asymptomatic elevation in liver enzymes, bile duct strictures on imaging, characteristic pathology findings, elevated serum IgG4, and excellent response to corticosteroids. Pediatric gastroenterologists and hepatologists, as well as pediatric hepatopathologists, need to be aware of IgG4-SC as a disease entity. Although certain clinical and imaging findings mimic PSC, diagnosis of IgG4-SC and its appropriate treatment with corticosteroids often lead to remission and reversal of disease.

Balloon-occluded retrograde transvenous obliteration for recurrent fundal gastric variceal bleeding in an adolescent.

Gastric variceal bleeding is associated with high morbidity and mortality. Balloon-occluded retrograde transvenous obliteration is a relatively new treatment used to control bleeding gastric varices that involves transvenous sclerosis of gastric varices through a spontaneous gastrorenal shunt. Here, we report on a 14-yr-old patient that underwent balloon-occluded retrograde transvenous obliteration for refractory bleeding fundal varices in the setting of esophageal varices and cirrhosis, which did not respond to medical management or endoscopic injection. This case report serves as a reminder that balloon-occluded retrograde transvenous obliteration can successfully control fundal variceal bleeding in pediatric patients and may serve as a bridge to liver transplantation.

Analysis of current treatments used in clinical practice in a pediatric summer camp population for children with inflammatory bowel disease.

BACKGROUND: Many treatment options exist for children with inflammatory bowel disease (IBD), yet the lack of clinical guidelines for management has lead to great variation in care. The purpose of this project was to evaluate current treatment modalities in children from the Northeast US who applied to the 2010 session of Camp Oasis, a Crohn's and Colitis Foundation of America (CCFA)-sponsored camp for children ages 8-17 with medically stable IBD. METHODS: Patient demographics, medical history, and current medications were entered into the camp database. The subjects were divided into two groups; Crohn's disease (CD) or ulcerative colitis/indeterminate colitis (UC/IC). In all, 164 applicants were included, 121 (74%) with CD and 43 (26%) with UC/IC. RESULTS: There were no significant differences between the two groups with respect to median age at the time of camp, median age at diagnosis, or median length of illness. Of the 121 applicants with CD, 13 (10.7%) were on an antibiotic, 56 (46.3%) were on a 5-aminosalicylate (5-ASA), 10 (8.3%) were on corticosteroids, 57 (47.1%) were on immunomodulators, and 44 (36.4%) were on a biologic agent. Six (5%) were on both an immunomodulator and a biologic agent. Of the 43 subjects with UC/IC, 27 (62.7%) were on a 5-ASA, two (4.7%) were on corticosteroids, 13 (30.2%) were on an immunomodulator, and four (9.3%) were on a biologic agent. The groups were similar with regard to surgery (20.7% for CD and 18.6% for UC/IC). CONCLUSIONS: Identifying current treatment patterns may serve to highlight variations in care among this pediatric IBD population.