Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis.

Background & Aims: PEDFIC 2, an ongoing, open-label, 72-week study, evaluates odevixibat, an ileal bile acid transporter inhibitor, in patients with progressive familial intrahepatic cholestasis.

Methods: PEDFIC 2 enrolled and dosed 69 patients across two cohorts; all received odevixibat 120 μg/kg per day. Cohort 1 comprised children from PEDFIC 1, and cohort 2 comprised new patients (any age).

Long-term results of liver transplantation for maple syrup urine disease: A single-center experience in Turkey.

Objectives: Maple syrup urine disease (MSUD) is an autosomal recessive inherited disorder. Despite the advances in medical nutrition therapies, classical phenotype causes severe neurological disorders and sudden death. It is known that MSUD patients do not experience metabolic attacks despite their free diet after liver transplantation (LT).

Intestinal Mucormycosis in a Child With Maple Syrup Urine Disease After Orthotopic Liver Transplant.

Mucormycosis can result in serious morbidity and mortality, especially in transplant recipients. In this case report, we present a 3-year-old female patient with maple syrup urine disease who developed mucormycosis infection after deceased donor split liver transplant. Progressive segmental necrosis of the small intestines and new ischemic areas were observed after repeated abdominal surgeries.

Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial.

Background: Progressive familial intrahepatic cholestasis (PFIC) is a group of inherited paediatric liver diseases resulting from mutations in genes that impact bile secretion. We aimed to evaluate the effects of odevixibat, an ileal bile acid transporter inhibitor, versus placebo in children with PFIC.

Methods: Patients eligible for this 24-week, randomised, double-blind, completed, phase 3 study were paediatric outpatients diagnosed with PFIC1 or PFIC2 who had pruritus and elevated serum bile acids at screening.

Exocrine pancreas functions in children with type 1 diabetes mellitus.

Background And Study Aim: We evaluated exocrine pancreas functions using a noninvasive indicator in a case-control study conducted on children and adolescents diagnosed with type 1 diabetes mellitus.

Patients And Methods: Sixty-seven patients who participated in a summer camp were enrolled in this study. Nineteen healthy children in the same age group were assigned to the control group.

Broadly effective metabolic and immune recovery with C5 inhibition in CHAPLE disease.

Complement hyperactivation, angiopathic thrombosis and protein-losing enteropathy (CHAPLE disease) is a lethal disease caused by genetic loss of the complement regulatory protein CD55, leading to overactivation of complement and innate immunity together with immunodeficiency due to immunoglobulin wasting in the intestine. We report in vivo human data accumulated using the complement C5 inhibitor eculizumab for the medical treatment of patients with CHAPLE disease. We observed cessation of gastrointestinal pathology together with restoration of normal immunity and metabolism.

Psychopathology, quality of life, and related factors in pediatric liver transplantation candidates and recipients.

Background: Liver transplantation (LT) has been accepted as a standard treatment of pediatric liver diseases that can progress to end-stage liver disease or lead to acute liver failure. However, there is a lack of studies clarifying quality of life (QoL) and the characteristics and the prevalence of common psychiatric disorders in children before and/or after LT. Thus, this study aimed to investigate QoL and the prevalence of anxiety, depression, and post-traumatic stress disorder (PTSD) in children and adolescents before and after LT and to compare them with healthy controls.

Role of Soluble Adiponectin Receptor 2 in Non-Alcoholic Fatty Liver Disease in Children.

Purpose: The incidence of non-alcoholic fatty liver disease (NAFLD) in children is gradually increasing. The aim of this study was to investigate the use of serum adiponectin and soluble adiponectin receptor 2 (soluble Adipo R2) levels for the diagnosis of fatty liver disease in obese and overweight children.

Methods: The study included 51 obese and overweight children between the ages of 6 and 18 years diagnosed with NAFLD using ultrasonography and 20 children without fatty liver disease.

Liver transplantation from a live donor to a patient with maple syrup urine disease: Two case reports.

Liver transplantation were reported in patients with classic maple syrup urine disease in the literature. Branched chain alpha keto acid dehydrogenase activity can be improved in patients after transplant, and a protein-restricted diet is usually not needed. The first patient was a boy aged 2,5 years who presented with frequent ketosis attacks and epileptic seizures, and the second patient was an 11-month-old boy who also presented with frequent ketosis episodes, both despite adherence to diet therapy.

Gastritis Associated with Initially Pediatric Crohn's Disease and Ulcerative Colitis.

Purpose: The aim of this study is to determine the involvement of the upper gastrointestinal system (GIS) in patients diagnosed with Crohn's disease (CD), ulcerative colitis (UC), and non-inflammatory bowel disease (IBD) and to compare their differences.

Methods: This study included patients aged between 2 and 18 years who underwent colonoscopy and esophagogastroduodenoscopy (EGD) for the first time due to the prediagnosis of IBD. In EGD, samples were taken from duodenum, antrum, corpus, and esophagus; and gastritis, duodenitis, and esophagitis were identified through histopathologic examination.

The incidence of HLA-DQ2/DQ8 in Turkish children with celiac disease and a comparison of the geographical distribution of HLA-DQ.

Introduction: Celiac disease (CD) is an auto-immune enteropathy that occurs in genetically pre-disposed people as a result of the consumption of gluten-containing foods.

Aim: To identify the incidence of HLA-DQ2 and HLA-DQ8 observed in children with CD.

Material And Methods: In this study, we focused on children ranging in age from 2 to 18 years and diagnosed with celiac disease.

Investigation of Efficacy of Lidocaine Spray for Sedated Esophagogastroduodenoscopy in Children.

Purpose: Our aim in this study is to investigate efficacy of topical lidocaine spray for sedated esophagogastroduodenoscopy (EGD) in children.

Methods: The endoscopy of children aged between 3-18 years who underwent EGD in our endoscopy unit. Intravenous (IV) midazolam and ketamine were used for sedation.

Investigation of the efficacy of synbiotics in the treatment of functional constipation in children: a randomized double-blind placebo-controlled study.

Background/aims: The aim of this study was to demonstrate the efficacy of synbiotic (Lactobacillus casei, L. rhamnosus, L. plantarum, and Bifidobacterium lactis and prebiotics [fiber, polydextrose, fructo-oligosaccharides, and galacto-oligosaccharides]) treatment in children with functional constipation.

Efficacy and safety of midazolam and ketamine in paediatric upper endoscopy.

Background And Study Aim: Upper endoscopy can be successfully carried out in children under deep sedation and anaesthesia. However, the best method of upper endoscopy for children who require gastrointestinal intervention has yet to be defined. The aim of this study is to investigate the efficacy and safety of the sedation induced by intravenous midazolam and ketamine during upper endoscopy in children.

Pediatric Liver Transplantation: Our Experiences.

Objective: The aim of our study was to evaluate our liver transplant pediatric patients and to report our experience in the complications and the long-term follow-up results.

Materials And Methods: Patients between the ages of 0 and 18 years, who had liver transplantation in the organ transplantation center of our university hospital between 1997 and 2016, were included in the study. The age, sex, indications for the liver transplantation, complications after the transplantation, and long-term follow-up findings were retrospectively evaluated.

Efficacy of synbiotic, probiotic, and prebiotic treatments for irritable bowel syndrome in children: A randomized controlled trial.

Background/aims: Irritable bowel syndrome (IBS) is an important health problem that presents serious social burdens and high costs. Our study investigated the efficacy of synbiotic (Bifidobacterium lactis B94 with inulin), probiotic (B. lactis B94), and prebiotic (inulin) treatment for IBS in a pediatric age group.

Acute vanishing bile duct syndrome after the use of ibuprofen.

We present a case report of a 7-year-old patient who developed toxic epidermal necrolysis (TEN) and vanishing bile duct syndrome (VBDS) after oral ibuprofen intake. Acute VBDS is a rare disease with unknown aetiology, often presenting with progressive loss of the intrahepatic biliary tract. TEN is an immune complex-mediated hypersensitivity reaction involving the skin and mucosa, which is induced by drugs or infectious diseases, sometimes leading to systemic symptoms.

Total oxidant status, total antioxidant capacity and ischemia modified albumin levels in children with celiac disease.

In our study, we aimed to investigate ischemia modified albumin (IMA) as an oxidative stress marker, as well as other oxidant and antioxidant markers that have not been evaluated in children with celiac disease. A total of 37 pediatric patients who were diagnosed with celiac disease (CD) and 29 healthy children were enrolled in this prospective study. We evaluated the IMA, total oxidant status, total antioxidant capacity, sulfhydryl, and advanced oxidation protein products in all of the subjects.