An international multicenter cohort study on implantable cardioverter-defibrillators for the treatment of symptomatic children with catecholaminergic polymorphic ventricular tachycardia.

Background: Catecholaminergic polymorphic ventricular tachycardia (CPVT) may cause sudden cardiac death (SCD) despite medical therapy. Therefore, implantable cardioverter-defibrillators (ICDs) are commonly advised. However, there is limited data on the outcomes of ICD use in children.

Refractive Error Change and Overminus Lens Therapy for Childhood Intermittent Exotropia.

Importance: Increased myopic shift was found to be associated with 1 year of overminus spectacle treatment for children with intermittent exotropia (IXT). Persistence of myopic shift after discontinuing overminus spectacles is unknown.

Objective: To compare refractive error change over 3 years in children with IXT originally treated with overminus vs nonoverminus spectacles.

Baclofen pumps do not increase risk of complications following spinal fusion.

Purpose: To assess the complication risks associated with intrathecal baclofen (ITB) pumps in cerebral palsy (CP) patients undergoing posterior spinal fusion (PSF) and to determine if timing of pump implantation before or during PSF impacts the risk of complications.

Methods: A prospectively collected multicenter database was retrospectively reviewed to identify CP patients undergoing PSF from 2008 to 2023. Patients were divided into 2 cohorts: those with an ITB pump (ITB cohort) and those without (non-ITB cohort).

Evaluation of the implementation of evidence-based pediatric asthma exacerbation treatments in a regional consortium of emergency medical Services Agencies.

Objective: Asthma exacerbations are a frequent reason for pediatric emergency medical services (EMS) encounters. The objective of this study was to examine the implementation of evidence-based treatments for pediatric asthma in a regional consortium of EMS agencies.

Methods: This retrospective study applied the Reach, Effectiveness, Adoption, Implementation, and Maintenance (RE-AIM) implementation framework to data from an EMS agency consortium in the Cincinnati, Ohio region.

Sphenochoanal polyps.

Nasal polyps are a common aetiology for persistent nasal obstruction. While antrochoanal polyps predominate the literature, the lesser known sphenochoanal polyp is equally as bothersome. To our knowledge, no prior dedicated review exists that characterises the patient population affected by this disease.

Parent-provided photographs as an outcome measure for childhood chalazia.

Purpose: To determine whether smartphone photographs of children's eyelids are reliable for diagnosing the presence of chalazia.

Methods: In this prospective cross-sectional study, 60 participants, 7 months to 16.5 years of age, at four sites were enrolled; all participants had a chalazion measuring at least 2 mm on at least one eyelid based on an in-person clinical examination by a pediatric eye care professional.

When successful, anterior vertebral body tethering (VBT) induces differential segmental growth of vertebrae: an in vivo study of 51 patients and 764 vertebrae.

Purpose: This study aimed to determine (1) does vertebral body tethering (VBT) produce differential growth modulation in individual vertebrae in patients with idiopathic scoliosis, (2) does VBT change disc shape, and (3) does VBT affect total spine length?

Methods: Patients with idiopathic scoliosis treated with VBT of the main thoracic curve and minimum 2-year follow-up were included. Vertebrae and discs were categorized as uninstrumented proximal thoracic, instrumented main thoracic, or uninstrumented thoracolumbar-lumbar. The left- and right-sided heights of each vertebra and disc were measured on subsequent radiographs to assess for differential growth.

Evaluation of age at symptom onset, proband status, and sex as predictors of disease severity in pediatric catecholaminergic polymorphic ventricular tachycardia.

Background: Children with catecholaminergic polymorphic ventricular tachycardia (CPVT) are at risk for sudden death, and a risk stratification tool does not exist.

Objective: The purpose of this study was to determine whether proband status, age at symptom onset, and/or sex are independent predictors of cardiac events.

Methods: A multicenter, ambispective, cohort of pediatric CPVT patients was categorized by sex, proband status, and age at symptom onset (D1: first decade of life [symptom onset <10 years] or D2: second decade of life [symptom onset 10-18 years, inclusive]).

Race as a factor in donor selection and survival of children with hematologic malignancies undergoing hematopoietic stem cell transplant in Florida.

Background: Previous studies have explored posthematopoietic cell transplant (HCT) outcomes by race in adults; however, pediatric data addressing this topic are scarce.

Procedure: This retrospective registry study included 238 White (W) and 57 Black (B) children with hematologic malignancies (HM) receiving first allogeneic HCT between 2010 and 2019 at one of the five Florida pediatric HCT centers.

Results: We found no differences between W and B children in transplant characteristics, other than donor type.

Traumatic injury to the unfused anterior arch of C1 in the setting of bipartite atlas.

We present a case of a pediatric patient with congenital unfused anterior and posterior arches of the atlas (C1), also known as bipartite atlas, who sustained a traumatic injury during gymnastics. A computed tomography (CT) scan of the cervical spine raised concern for abnormal separation of the midline cleft of the anterior arch of C1. Subsequent magnetic resonance imaging (MRI) showed focal, edema-like signal in the midline cleft of C1.

Overminus Lens Therapy for Children 3 to 10 Years of Age With Intermittent Exotropia: A Randomized Clinical Trial.

Importance: This is the first large-scale randomized clinical trial evaluating the effectiveness and safety of overminus spectacle therapy for treatment of intermittent exotropia (IXT).

Objective: To evaluate the effectiveness of overminus spectacles to improve distance IXT control.

Design, Setting, And Participants: This randomized clinical trial conducted at 56 clinical sites between January 2017 and January 2019 associated with the Pediatric Eye Disease Investigator Group enrolled 386 children aged 3 to 10 years with IXT, a mean distance control score of 2 or worse, and a refractive error between 1.

Genotype-Phenotype Correlations in Central Precocious Puberty Caused by MKRN3 Mutations.

Context: Loss-of-function mutations of makorin RING finger protein 3 (MKRN3) are the most common monogenic cause of familial central precocious puberty (CPP).

Objective: To describe the clinical and hormonal features of a large cohort of patients with CPP due to MKRN3 mutations and compare the characteristics of different types of genetic defects.

Methods: Multiethnic cohort of 716 patients with familial or idiopathic CPP screened for MKRN3 mutations using Sanger sequencing.

Simultaneous Diagnosis of Craniopharyngioma in 2 Brothers.

Craniopharyngioma is a rare suprasellar tumor. Approximately one-third of cases occur in pediatric patients. Depending on the size and extent of the lesion, the main treatment options include complete surgical removal of the tumor or limited surgery followed by radiotherapy.

Pediatric HCT in Florida (2014 -2016): A report from the FPBCC.

FPBCC was formed in 2018 by five pediatric transplant programs in Florida. One of the key objectives of the consortium is to provide outcome analyses by combining HCT data from all the participating centers in order to identify areas for improvement. In this first FPBCC landscape report we describe the patient and transplant characteristics of pediatric patients undergoing first allo and auto HCT between 2014 and 2016 in Florida.

Neuroimaging of Propofol Infusion Syndrome: A Case Report and Review of Literature.

A school-age boy with a complex medical history underwent a minor elective surgical procedure. Propofol was used for sedation during the procedure. The patient could not be awakened post-operatively.

Implementation of family psychosocial risk assessment in pediatric cancer with the Psychosocial Assessment Tool (PAT): study protocol for a cluster-randomized comparative effectiveness trial.

Background: Childhood cancer affects and is affected by multiple levels of the social ecology, including social and relational determinants of health (e.g., economic stability, housing, childcare, healthcare access, child and family problems).

Irinotecan, Temozolomide, and Dinutuximab With GM-CSF in Children With Refractory or Relapsed Neuroblastoma: A Report From the Children's Oncology Group.

Purpose: The combination of irinotecan, temozolomide, dintuximab, and granulocyte-macrophage colony-stimulating factor (I/T/DIN/GM-CSF) demonstrated activity in patients with relapsed/refractory neuroblastoma in the randomized Children's Oncology Group ANBL1221 trial. To more accurately assess response rate and toxicity, an expanded cohort was nonrandomly assigned to I/T/DIN/GM-CSF.

Patients And Methods: Patients were eligible at first relapse or first designation of refractory disease.

Linked-read genome sequencing identifies biallelic pathogenic variants in as a novel cause of Meier-Gorlin syndrome.

Material: Linked-read whole genome sequencing (WGS) presents a new opportunity for cost-efficient singleton sequencing in place of traditional trio-based designs while generating informative-phased variants, effective for recessive disorders when parental DNA is unavailable.

Methods: We have applied linked-read WGS to identify novel causes of Meier-Gorlin syndrome (MGORS), a condition recognised by short stature, microtia and patella hypo/aplasia. There are eight genes associated with MGORS to date, all encoding essential components involved in establishing and initiating DNA replication.

Author Correction: CHD3 helicase domain mutations cause a neurodevelopmental syndrome with macrocephaly and impaired speech and language.

The HTML and PDF versions of this Article were updated after publication to remove images of one individual from Figure 1.

Spatial Patterning of Molecular Cues and Vascular Cells in Fully Integrated Hydrogel Channels via Interfacial Bioorthogonal Cross-Linking.

Fully integrated hydrogel channels were fabricated via interfacial bioorthogonal cross-linking, a diffusion-controlled method for the creation and patterning of synthetic matrices based on the rapid bioorthogonal reaction between s-tetrazines (Tz) and trans-cyclooctene (TCO) dienophiles. Injecting an aqueous solution of a bisTCO cross-linker into a reservoir of tetrazine-modified hyaluronic acid (HA-Tz), while simultaneously drawing the syringe needle through the reservoir, yielded a cross-linked hydrogel channel that was mechanically robust. Fluorescent tags and biochemical signals were spatially patterned into the channel wall through time-dependent perfusion of TCO-conjugated molecules into the lumen of the channel.

Clinical and biological predictors of response to standardised paediatric colitis therapy (PROTECT): a multicentre inception cohort study.

Background: Lack of evidence-based outcomes data leads to uncertainty in developing treatment regimens in children who are newly diagnosed with ulcerative colitis. We hypothesised that pretreatment clinical, transcriptomic, and microbial factors predict disease course.

Methods: In this inception cohort study, we recruited paediatric patients aged 4-17 years with newly diagnosed ulcerative colitis from 29 centres in the USA and Canada.

Author Correction: CHD3 helicase domain mutations cause a neurodevelopmental syndrome with macrocephaly and impaired speech and language.

The original version of this Article contained an error in the spelling of the author Laurence Faivre, which was incorrectly given as Laurence Faive. This has now been corrected in both the PDF and HTML versions of the Article.