Outcomes in Hematopoietic Stem Cell Transplantation for Congenital Amegakaryocytic Thrombocytopenia.

Congenital amegakaryocytic thrombocytopenia (CAMT) is a rare, inherited bone marrow failure syndrome. Hematopoietic stem cell transplantation (HSCT) is considered a curative treatment option, but existing descriptions of patient and transplant characteristics and outcomes after related and unrelated donor HSCT are sparse. We describe outcomes after HSCT for congenital amegakaryocytic thrombocytopenia (CAMT; n = 86) from 2000 to 2018.

Welcoming Healthcare Into Our Virtual Meeting Rooms: Perspective and Perseverance in Pediatric Rheumatology.

Purpose Of Review: We highlight practice changes adopted to increased use of telemedicine, look at precision, and accuracy in using a virtual visit to evaluate and treat rheumatic disease, and describe our shift in engaging patients and their families in supporting our research aims.

Recent Findings: Telemedicine visits increased substantially with the start of the SARS-CoV-2 pandemic. With this change came the need for significant advances to our telemedicine practices to allow for quality patient visits and continued research collection.

Epilepsy Surgery: Special Circumstances.

Epilepsy surgery has proven to be very effective in treating refractory focal epilepsies in children, producing seizure freedom or partial seizure control well beyond any other medical or dietary therapies. While surgery is mostly utilized in certain clinical phenotypes, either based on the location such as temporal lobe epilepsy, or based on the presence of known epileptogenic lesions such as focal cortical dysplasia, tumors or hemimegalencephaly, there is a growing body of evidence to support the role of surgery in other patients' cohorts that were classically not thought of as surgical candidates. These include patients with rare genetic disorders, electrical status epilepticus in sleep, status epilepticus and the very young patients.

Provider Perspectives on the Acceptability, Appropriateness, and Feasibility of Teleneonatology.

Objective: We aimed to measure provider perspectives on the acceptability, appropriateness, and feasibility of teleneonatology in neonatal intensive care units (NICUs) and community hospitals.

Study Design: Providers from five academic tertiary NICUs and 27 community hospitals were surveyed using validated implementation measures to assess the acceptability, appropriateness, and feasibility of teleneonatology. For each of the 12 statements, scale values ranged from 1 to 5 (1 = strongly disagree; 5 = strongly agree), with higher scores indicating greater positive perceptions.

Uncharted waters: mesenchymal stem cell treatment for pediatric refractory rheumatic diseases; a single center case series.

Background: With the advent of innovative therapies including biologics and Janus kinase inhibitors, children with rheumatic diseases are more likely to have improved outcomes. Despite these advances, some children do not respond, or they, or their parents fear adverse events and seek other alternatives. Increasingly, private companies are offering mesenchymal stem cells (MSC) as an alternative, which are described as natural therapies for rheumatic diseases, often insinuating them as a cure.

Determination of language dominance in pediatric patients with epilepsy for clinical decision-making: Correspondence of intracarotid amobarbitol procedure and fMRI modalities.

Identification of the language dominant hemisphere is an essential part of the evaluation of potential pediatric epilepsy surgery patients. Historically, language dominance has been determined using the intracarotid amobarbitol procedure (IAP), but use of functional Magnetic Resonance Imaging (fMRI) scanning is becoming more common. Few studies examine the correspondence between fMRI and IAP in pediatric samples.

Drivers of Variation in Diagnosis and Management of Eosinophilic Esophagitis: A Survey of Pediatric Gastroenterologists.

Background: Widespread variation in the diagnosis and treatment of eosinophilic esophagitis (EoE) has previously been reported among adult gastroenterologists; however, variation in EoE practice in among pediatric populations is poorly characterized. The study objectives were to describe guideline adherence and understand reasons for variation in EoE practice among pediatric gastroenterologists following publication of the updated 2018 international EoE guidelines.

Methods: We developed and administered a 28-item survey to pediatric gastroenterologists via an email listserv using the PEDGI Bulletin Board from 03/2019 to 04/2019.

Development of best practices in the utilization and implementation of pediatric cervical spine traction: a modified Delphi study.

Objective: Cervical traction in pediatric patients is an uncommon but invaluable technique in the management of cervical trauma and deformity. Despite its utility, little empirical evidence exists to guide its implementation, with most practitioners employing custom or modified adult protocols. Expert-based best practices may improve the care of children undergoing cervical traction.

A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One Allele.

Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and safe in patients ≥12 years of age with cystic fibrosis and at least one (cystic fibrosis transmembrane conductance regulator) allele, but it has not been evaluated in children <12 years of age. To assess the safety, pharmacokinetics, and efficacy of ELX/TEZ/IVA in children 6 through 11 years of age with -minimal function or - genotypes. In this 24-week open-label phase 3 study, children ( = 66) weighing <30 kg received 50% of the ELX/TEZ/IVA adult daily dose (ELX 100 mg once daily, TEZ 50 mg once daily, and IVA 75 mg every 12 h) whereas children weighing ⩾30 kg received the full adult daily dose (ELX 200 mg once daily, TEZ 100 mg once daily, and IVA 150 mg every 12 h).

ASE Statement on Adapting Pediatric, Fetal, and Congenital Heart Disease Echocardiographic Services to the Evolving COVID-19 Pandemic.

Over the 12 months since the start of the coronavirus disease 2019 pandemic, an explosion of investigation and an increase in experience have led to vast improvement in our knowledge about this disease. However, coronavirus disease 2019 remains a huge public health threat.

The Impact of Erythropoietin on Short- and Long-Term Kidney-Related Outcomes in Neonates of Extremely Low Gestational Age. Results of a Multicenter, Double-Blind, Placebo-Controlled Randomized Clinical Trial.

Objective: To evaluate whether extremely low gestational age neonates (ELGANs) randomized to erythropoietin have better or worse kidney-related outcomes during hospitalization and at 22-26 months of corrected gestational age (cGA) compared with those randomized to placebo.

Study Design: We performed an ancillary study to a multicenter double-blind, placebo-controlled randomized clinical trial of erythropoietin in ELGANs.

Results: The prevalence of severe (stage 2 or 3) acute kidney injury (AKI) was 18.

Editorial: Analyzing Treatment and Prescribing in Large Administrative Datasets With a Lens on Equity.

In this issue of the Journal, Bushnell and colleagues present findings from their analysis of a commercial insurance administrative dataset, examining the ways that antipsychotics are used in young children (aged 2-7 years) in the United States. From 2009 to 2017, they find that the use of antipsychotics decreased and there was a shift toward use of medications in alignment with evidence-based standards. The most common conditions for use of antipsychotics included pervasive developmental disorders, externalizing disorders, and attention-deficit/hyperactivity disorder.

Mechanisms of Motivational Interviewing: a Conceptual Framework to Guide Practice and Research.

Reviews of the motivational interviewing (MI) training literature demonstrate MI is a nuanced skill set that takes carefully planned didactic training, application of skills in context-specific practice settings, and ongoing support to promote reflective practice and sustained proficiency. Despite the robust knowledge base related to training and how MI works to achieve favorable outcomes, these two literature bases are not well integrated. In an effort to inform and guide future research, we propose the mechanisms of motivational interviewing (MMI) conceptual framework, which expands upon previous work.

Multiple hippocampal transections for refractory pediatric mesial temporal lobe epilepsy: seizure and neuropsychological outcomes.

Objective: Temporal lobe epilepsy (TLE) is the most common focal epilepsy across adult and pediatric age groups. It is also the most amenable to surgery, with excellent long-term seizure outcome. Most TLE cases have an epileptogenic zone in the mesial temporal structures, namely the hippocampus.

Prevalence of acute kidney injury (AKI) in extremely low gestational age neonates (ELGAN).

Background: To determine the prevalence and severity of acute kidney injury (AKI) at different time frames in relation to gestational age (GA) and birthweight (BW) in extremely low gestational age neonates (ELGAN). Our hypothesis is that ELGAN with lower GA and lower BW have higher AKI rates.

Methods: A total of 923 ELGAN enrolled in the Preterm Erythropoietin Neuroprotection Trial were evaluated from birth until death or hospital discharge.

Sugammadex and neuromuscular reversal: special focus on neonatal and infant populations.

Purpose Of Review: Sugammadex is a novel selective muscle relaxant binding agent capable of reversing deep neuromuscular blockade from rocuronium or vecuronium. It has not been approved for use in children by the US FDA because of lack of literature regarding pediatric patients.

Recent Findings: A series of new studies has reported sugammadex administration to increasingly younger patients ranging in the newborn period.

Clinical Consensus Statement: Ankyloglossia in Children.

Objective: To identify and seek consensus on issues and controversies related to ankyloglossia and upper lip tie in children by using established methodology for American Academy of Otolaryngology-Head and Neck Surgery clinical consensus statements.

Methods: An expert panel of pediatric otolaryngologists was assembled with nominated representatives of otolaryngology organizations. The target population was children aged 0 to 18 years, including breastfeeding infants.

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report.

Wiskott-Aldrich syndrome (WAS) is an X-linked disease caused by mutations in the WAS gene, leading to thrombocytopenia, eczema, recurrent infections, autoimmune disease, and malignancy. Hematopoietic cell transplantation (HCT) is the primary curative approach, with the goal of correcting the underlying immunodeficiency and thrombocytopenia. HCT outcomes have improved over time, particularly for patients with HLA-matched sibling and unrelated donors.

Evaluating the nutritional content of an insect-fortified food for the child complementary diet in Ghana.

Background: Due to rising food insecurity, natural resource scarcity, population growth, and the cost of and demand for animal proteins, insects as food have emerged as a relevant topic. This study examines the nutrient content of the palm weevil larva (), a traditionally consumed edible insect called in Ghana, and assesses its potential as an animal-source, complementary food.

Methods: in two "unmixed" forms (raw, roasted) and one "mixed" form (-groundnut paste) were evaluated for their macronutrient, micronutrient, amino acid, and fatty acid profiles.

Delayed initiation of enteral feeds is associated with postnatal growth failure among preterm infants managed at a rural hospital in Uganda.

Background: Prematurity is the leading cause of mortality in children under 5 years of age globally and is also frequently associated with postnatal growth failure (PGF). Although most preterm births occur in low resource settings, little is known about their postnatal growth outcomes especially in rural areas. We evaluated the incidence and factors associated with PGF among preterm infants managed at a rural hospital in Uganda.