Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA.

Background And Aims: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the G lobal AL agille A lliance (GALA) study.

Specific inhibitor of Wnt/beta-catenin pathway can alter behavioral responses in young rats with malformed cortices.

The Wnt/beta-catenin pathway plays a crucial role in regulating cellular processes and has been implicated in neural activity-dependent learning as well as anxiety. However, the role of this pathway in young children with abnormal cortical development is unknown. Cortical malformations at early development, behavioral abnormalities, and a susceptibility to seizures have been reported in rats prenatally exposed to methylazoxymethanol.

A Report on a Nationwide Surveillance System for Pediatric Acute Hepatitis of Unknown Etiology in Korea.

Background: Several cases of pediatric acute hepatitis of unknown etiology related to adenoviral infections have been reported in Europe since January 2022. The aim of this study was to compare the incidence, severity, possible etiology, and prognosis of the disease with those in the past in Korea.

Methods: The surveillance group collected data between May and November 2022 using a surveillance system.

Does brace treatment following closed reduction of developmental dysplasia of the hip improve acetabular coverage?

Aims: Abduction bracing is commonly used to treat developmental dysplasia of the hip (DDH) following closed reduction and spica casting, with little evidence to support or refute this practice. The purpose of this study was to determine the efficacy of abduction bracing after closed reduction in improving acetabular index (AI) and reducing secondary surgery for residual hip dysplasia.

Methods: We performed a retrospective review of patients treated with closed reduction for DDH at a single tertiary referral centre.

Impact of the Coronavirus Disease 2019 Pandemic on Pediatric Gastrointestinal Endoscopy: A Questionnaire-based Internet Survey of 162 Institutional Experiences in Asia Pacific.

Purpose: The impact of coronavirus 2019 (COVID-19) on gastrointestinal (GI) endoscopy procedures in adults has been reported, with a drastic reduction in the number of procedures. However, there are no sufficient data regarding the impact on pediatric GI endoscopy. Here, we aimed to report that impact in the Asia-Pacific region.

Outcomes at 18-24 Months of Infants with Birth Weight under 500 g Born in Korea during 2013-2017: A Nationwide Cohort Study.

Introduction: This study aimed to investigate the outcomes of infants at 18-24 months born in the Korean Neonatal Network with a birth weight <500 g.

Methods: The anthropometric and neurodevelopmental data of infants with a birth weight <500 g at a gestational age of ≥22 weeks who were registered in the Korean Neonatal Network 2013-2017 and followed up at a corrected age of 18-24 months were reviewed. Neurodevelopmental impairment was defined as the presence of any of the following: (1) cerebral palsy; (2) severe visual impairment; (3) hearing impairment; or (4) cognitive impairment.

Disseminated Intravascular Coagulation as a Risk Factor for Clinical Outcome After Liver Transplantation in Pediatric Patients With Kasai Portoenterostomy Failure.

Background: Disseminated intravascular coagulation (DIC) is a serious complication in critically ill pediatric patients. This study aimed to evaluate the association between pretransplant DIC and perioperative clinical outcomes of liver transplantation (LT) in pediatric patients with Kasai portoenterostomy (KPE) failure.

Methods: We enrolled pediatric patients who received LT after KPE failure between January 2005 and April 2021.

Early echocardiographic pulmonary artery measurements as prognostic indicators in left congenital diaphragmatic hernia.

Background: To predict whether the left pulmonary artery (LPA) to the main pulmonary artery (MPA) ratio measured by echocardiography in left congenital diaphragmatic hernia (CDH) was related to death or need for extracorporeal membrane oxygenation (ECMO).

Methods: This retrospective study analyzed neonates with left CDH born between 2018 and 2022 in a single tertiary medical institution. Echocardiography was performed immediately after birth.

Kidney involvement in children during the SARS-CoV-2 Omicron variant pandemic.

Background: As the coronavirus disease-2019 (COVID-19) pandemic continues, driven by the Omicron variant, infection rates in children have recently rapidly surged compared with previous years. We aimed to investigate the presentation of kidney involvement in children after Omicron variant severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) infection.

Methods: We retrospectively reviewed the medical records of pediatric patients who presented with kidney disease with a temporal relationship with COVID-19 between January and August 2022 in a single tertiary center in Korea.

Development of a Minimum Dataset for the Monitoring of Recombinant Human Growth Hormone Therapy in Children with Growth Hormone Deficiency: A GloBE-Reg Initiative.

Introduction: Although there are some recommendations in the literature on the assessments that should be performed in children on recombinant human growth hormone (rhGH) therapy, the level of consensus on these measurements is not clear. The objective of the current study was to identify the minimum dataset (MDS) that could be measured in a routine clinical setting across the world, aiming to minimise burden on clinicians and improve quality of data collection.

Methods: This study was undertaken by the growth hormone (GH) scientific study group in GloBE-Reg, a new project that has developed a common registry platform that can support long-term safety and effectiveness studies of drugs.

A Phenomenologic Study of the Experiences of Pediatric Cancer Survivors After Hematopoietic Stem Cell Transplantation.

Purpose: To explore the experiences of pediatric cancer survivors in South Korea after hematopoietic stem cell transplantation.

Participants & Setting: 14 survivors of childhood cancer who had undergone hematopoietic stem cell transplantation at a pediatric hematopoietic stem cell transplantation center.

Methodologic Approach: Participants underwent in-depth face-to-face or online interviews from October 2020 to January 2021.

The Difference in Tibial Tuberosity to Trochlear Groove Distance Between CT and MRI Arises From the Degree of Knee Flexion During Imaging.

Background: Tibial tuberosity to trochlear groove distance (TT-TG) has been reported to have different values according to imaging modalities, usually higher in computed tomography (CT) than in magnetic resonance imaging (MRI). This difference is thought to be caused by the degree of knee flexion during imaging, but few studies have aimed to elucidate the cause.

Methods: Five hundred eight patients with knee CT or MRI performed between ages of 6 to 16 years without underlying diseases affecting the musculoskeletal system were included.

Comparison of the clinical characteristics and clinical outcomes of culture-positive septic shock and culture-negative septic shock among pediatric patients.

Objectives: Among pediatric patients with septic shock, culture-negative septic shock (CNSS) is common but there have been limited data on its clinical characteristics and prognosis. We compared the clinical characteristics and clinical outcomes between culture-positive septic shock (CPSS) and CNSS in pediatric patients.

Design: Retrospective single-center study.

Redo pull-through for postoperative complications following pull-through in Hirschsprung disease: a single center experience.

Purpose: Although surgical management of Hirschsprung disease (HD) is effective in most patients, some patients experience long-term postoperative complications, and require redo pull-through (PT). The present study evaluated clinical outcomes of redo PT in HD patients at a single center.

Methods: Patients with HD who underwent redo PT procedures between 2003 and 2019 were retrospectively reviewed.

Electrophysiological network predicts clinical response to vigabatrin in epileptic spasms.

Purpose: This study aimed to discover electrophysiologic markers correlated with clinical responses to vigabatrin-based treatment in infants with epileptic spasms (ES).

Method: The study involved a descriptive analysis of ES patients from a single institution, as well as electroencephalogram (EEG) analyses of 40 samples and 20 age-matched healthy infants. EEG data were acquired during the interictal sleep state prior to the standard treatment.

Corrigendum: Mineral bone disorder in children with chronic kidney disease: data from the KNOW-Ped CKD (Korean cohort study for outcome in patients with pediatric chronic kidney disease) study.

[This corrects the article DOI: 10.3389/fped.2023.

Sterile Silicone Ring Tourniquets in Limb Surgery: A Prospective Clinical Trial in Pediatric Patients Undergoing Orthopedic Surgery.

Sterile silicone ring tourniquets (SSRTs) reduce intraoperative bleeding and provide a wide surgical view. Moreover, they reduce the risk of contamination and are cheaper than conventional pneumatic tourniquets. Our study describes the perioperative outcomes of sterile silicone ring tourniquet placement in pediatric patients undergoing orthopedic surgery.

Association between serum total cholesterol and chronic kidney disease progression in children: results from the KNOW-PedCKD.

Background: Dyslipidemia can cause cardiovascular disease and increase the fatality rate among children with chronic kidney disease (CKD); this makes early screening and treatment of dyslipidemia crucial. This study aimed to assess the association between the changes in serum total cholesterol levels over time and the degree of CKD progression in children.

Methods: From April 2011 to August 2021, 379 of the 432 participants enrolled in the KoreaN cohort study for Outcomes in patients With Pediatric CKD (KNOW-PedCKD) were included and divided into 4 categories based on total cholesterol levels (< 170 mg/dL, acceptable; 170-199, borderline; 200-239, high; and ≥ 240, very high).

Increased PD-1 expression of bone marrow T-cells in acute myeloid leukaemia patients after stem cell transplantation, and its association with overall survival.

Background: Immune checkpoints are involved in mechanisms by which tumours escape from the host immune system. Our aim was to evaluate acute myeloid leukaemia (AML) patients to determine expression levels of checkpoint molecules according to diagnosis and treatments, and to identify optimal candidates for checkpoint blockade.

Methods: Bone marrow (BM) samples were obtained from 279 AML patients at different disease status and from 23 controls.

Ogden Type IV Tibial Tuberosity Fractures in Healthy Adolescents: Preoperative Magnetic Resonance Imaging and 2-Year Clinical Follow-up Study.

Background: Ogden type IV tibial tuberosity fractures, defined as a type of fracture with posterior-inferior metaphyseal extension (Salter-Harris type II variant), are uncommon but challenging pediatric fractures. The purpose of this study was to investigate the clinical and radiological presentation and associated surgical outcomes.

Methods: Ten previously healthy patients who had been surgically treated at the authors' institution between 2015 and 2018 with at least 2 years of postoperative follow-up were included.

A new cyclin-dependent kinase-9 inhibitor A09-003 induces apoptosis in acute myeloid leukemia cells with reduction of myeloid cell leukemia sequence-1 protein.

Acute myeloid leukemia (AML) is the most common type of hematological disease in adults, and has a very poor outcome [1]. Based on its wide range of efficacy in AML models, a small-molecule inhibitor of the anti-apoptotic protein BCL-2, venetoclax (ABT-199/GDC-0199), was developed for clinical trials. However, venetoclax showed limited monotherapy activity [2].