Adding hydroxyurea to chronic transfusion therapy for sickle cell anemia reduces transfusion burden.

Background: Chronic red blood cell (RBC) transfusion is an established therapy to prevent stroke in patients with sickle cell anemia (SCA). It is unclear if adding daily hydroxyurea treatment to chronic transfusion is beneficial.

Study Design And Methods: We conducted a phase 2 clinical trial (NCT03644953) investigating the addition of dose-escalated hydroxyurea to chronic transfusion for patients with SCA receiving simple chronic transfusion for stroke prevention.

A randomized controlled educational study to evaluate an e-learning module to teach the physical examination of the temporomandibular joint in juvenile idiopathic arthritis.

Background: The aim of the study was to evaluate the effectiveness of a novel e-learning module in teaching the physical exam of the temporomandibular joint (TMJ) in Juvenile idiopathic arthritis (JIA.).

Methods: An e-learning module was developed to convey the TMJ physical examination maneuvers that are considered to be best practice in JIA.

Development and Validation of a Pediatric Celiac Disease-Specific Quality of Life Measure.

Introduction: Quality of life (QOL) is critical in screening and management of chronic medical conditions, including celiac disease (CD). The aim of this project was to develop a CD-specific pediatric QOL measure (Celiac Disease Life Inventory of Family Experiences [CDLIFE]) with parallel self-report and parent-report forms by generating items through concept elicitation interviews, iterative refinement using cognitive debriefing interviews, and evaluating its psychometric properties and validity.

Methods: Concept elicitation interviews were conducted to develop items (9 youth ages 8-19 years with CD; 10 parents of youth with CD), followed by cognitive interviews with additional stakeholders (3 youth with CD, 3 parents, and 8 clinicians) and item administration (parent/youth reports: n = 103/102).

Integrating a Breastfeeding Peer Counselor Into an Academic Hospital's Obstetric Clinic: A Pilot Study.

Black recipients of the Special Supplemental Nutrition Program for Women, Infants, and Children (WIC) have low breastfeeding rates. The aim of this pilot study was to determine if prenatal education by a breastfeeding peer counselor in an academic obstetric clinic is feasible and could improve WIC-eligible participants' breastfeeding self-efficacy and in-hospital breastfeeding rates. Pregnant participants ( = 57) were randomized into either an intervention group, which spoke briefly with a breastfeeding peer counselor immediately after their clinic appointment, or a comparison group, which received usual prenatal obstetric care only.

Blood volume collected for cultures in infants with suspected neonatal sepsis.

Objectives: To evaluate blood culture sample volumes, identify factors linked to insufficient samples, and compare volumes among neonates treated for culture-negative-sepsis, sepsis-rule-outs, and bloodstream infections (BSI).

Methods: Observational cohort of blood cultures collected during NICU stay. Association of age, weight, gender, source, and collection time with lower-than-recommended volumes was determined by logistic regression.

Efficient exact exchange using Wannier functions and other related developments in planewave-pseudopotential implementation of RT-TDDFT.

The plane-wave pseudopotential (PW-PP) formalism is widely used for the first-principles electronic structure calculation of extended periodic systems. The PW-PP approach has also been adapted for real-time time-dependent density functional theory (RT-TDDFT) to investigate time-dependent electronic dynamical phenomena. In this work, we detail recent advances in the PW-PP formalism for RT-TDDFT, particularly how maximally localized Wannier functions (MLWFs) are used to accelerate simulations using the exact exchange.

Applying a Trauma-Informed Lens to Challenging Adolescent Encounters: A Faculty Development Session for Pediatricians.

Introduction: Patient encounters perceived to be challenging are common and contribute to both suboptimal patient health outcomes and provider burnout. A trauma-informed care (TIC) approach to these encounters is critical, as many of the characteristics associated with challenging patient encounters can be linked to a history of trauma exposure.

Methods: Our team created and delivered a 1-hour synchronous virtual session intended to bolster provider knowledge of TIC principles and their application to challenging adolescent encounters.

Antibody-displaying extracellular vesicles for targeted cancer therapy.

Extracellular vesicles (EVs) function as natural delivery vectors and mediators of biological signals across tissues. Here, by leveraging these functionalities, we show that EVs decorated with an antibody-binding moiety specific for the fragment crystallizable (Fc) domain can be used as a modular delivery system for targeted cancer therapy. The Fc-EVs can be decorated with different types of immunoglobulin G antibody and thus be targeted to virtually any tissue of interest.

Pubertal Suppression in Early Puberty Followed by Testosterone Mildly Increases Final Height in Transmasculine Youth.

Context: Treatment for transmasculine youth (TMY) can involve testosterone treatment and is sometimes preceded by gonadotropin-releasing hormone agonist (GnRHa) treatment for puberty blockade. GnRHas can increase final height in birth-assigned females with central precocious puberty. Maximizing final adult height (FAH) is an important outcome for many TMY.

Application of the endogenous CRISPR-Cas type I-D system for genetic engineering in the thermoacidophilic archaeon .

CRISPR (clustered regularly interspaced short palindromic repeats)-Cas systems are widely distributed among bacteria and archaea. In this study, we demonstrate the successful utilization of the type I-D CRISPR-Cas system for genetic engineering in the thermoacidophilic archaeon . Given its extreme growth conditions characterized by a temperature of 75°C and pH 3, an uracil auxotrophic selection system was previously established, providing a basis for our investigations.

Household laundry detergents disrupt barrier integrity and induce inflammation in mouse and human skin.

Background: Epithelial barrier impairment is associated with many skin and mucosal inflammatory disorders. Laundry detergents have been demonstrated to affect epithelial barrier function in vitro using air-liquid interface cultures of human epithelial cells.

Methods: Back skin of C57BL/6 mice was treated with two household laundry detergents at several dilutions.

Assessing the Effect of Augmented Reality on Procedural Outcomes During Ultrasound-Guided Vascular Access.

Objective: Augmented reality devices are increasingly accepted in health care, though most applications involve education and pre-operative planning. A novel augmented reality ultrasound application, HoloUS, was developed for the Microsoft HoloLens 2 to project real-time ultrasound images directly into the user's field of view. In this work, we assessed the effect of using HoloUS on vascular access procedural outcomes.

Identification of scaffold proteins for improved endogenous engineering of extracellular vesicles.

Extracellular vesicles (EVs) are gaining ground as next-generation drug delivery modalities. Genetic fusion of the protein of interest to a scaffold protein with high EV-sorting ability represents a robust cargo loading strategy. To address the paucity of such scaffold proteins, we leverage a simple and reliable assay that can distinguish intravesicular cargo proteins from surface- as well as non-vesicular proteins and compare the EV-sorting potential of 244 candidate proteins.

Cardiac evaluation of hospitalized children with 2019 coronavirus (COVID-19) infection at a single large quaternary center.

Background: Cardiac complications of serious SARS-CoV-2 infections, especially Multisystem Inflammatory Syndrome of Children (MIS-C) are well described, however current studies have not considered pediatric patients hospitalized with no cardiac concerns. We established a protocol for cardiac evaluation of all admitted COVID-19 patients three weeks post-discharge, irrespective of cardiac concerns. We assessed cardiovascular outcomes and hypothesized that patients with absent cardiac concerns are at lower risk for cardiac abnormalities.

Pediatric Lyme disease: systematic assessment of post-treatment symptoms and quality of life.

Background: Lyme disease is common among children and adolescents. Antibiotic treatment is effective, yet some patients report persistent symptoms following treatment, with or without functional impairment. This study characterized long-term outcome of pediatric patients with Lyme disease and evaluated the case definition of post-treatment Lyme disease (PTLD) syndrome.

Assessing trainee critical thinking skills using a novel interactive online learning tool.

Background: Critical thinking is essential for the accurate diagnosis and management of patients. It is correlated with academic success.

Objective: Our objective was to design a novel tool for interactive online learning to improve knowledge and to assess trainees' critical thinking skills using the framework of the American Philosophical Association (APA).

Interprofessional Extracorporeal Membrane Oxygenation Cardiopulmonary Resuscitation Simulations Aimed at Decreasing Actual Cannulation Times: A Longitudinal Study.

Background: Since 2013, the cardiac intensive care unit (CICU) at Children's National has conducted annual extracorporeal membrane oxygenation cardiopulmonary resuscitation (ECPR) simulations that focus on team dynamics, room setup, and high-quality CPR. In 2019 and 2020, the simulations were expanded to include the surgical and extracorporeal membrane oxygenation (ECMO) teams in an effort to better understand and improve this process.

Methods: During a 4-week period in 2019, 7 peripheral ECPR simulations were conducted, and through a 3-week period in 2020, 7 central ECPR simulations were conducted.

Cell-specific targeting of extracellular vesicles through engineering the glycocalyx.

Extracellular vesicles (EVs) are promising carriers for the delivery of a variety of chemical and biological drugs. However, their efficacy is limited by the lack of cellular specificity. Available methods to improve the tissue specificity of EVs predominantly rely on surface display of proteins and peptides, largely overlooking the dense glycocalyx that constitutes the outermost layer of EVs.

Examining the Association Between MIS-C and the Child Opportunity Index at a Single Center.

Objective: To describe associations between the Child Opportunity Index (COI) and multisystem inflammatory syndrome of childhood (MIS-C) diagnosis among hospitalized children.

Methods: We used a retrospective case control study design to examine children ≤21 years hospitalized at a single, tertiary care children's hospital between March 2020 and June 2021. Our study population included children diagnosed with MIS-C (n = 111) and a control group of children hospitalized for MIS-C evaluation who had an alternative diagnosis (n = 61).

Pediatric screen exposure and school related headache disability.

Background And Objectives: Prolonged screen exposure is often cited as a trigger for pediatric headache. We present initial findings evaluating the association between adolescent screen use type, duration, and school disability.

Methods: New patients aged 12-17 years presenting to a headache clinic were screened and surveyed regarding headache characteristics, behavioral habits, school attendance, and screen utilization.

An educational intervention to facilitate appropriate subspecialty referrals: a study assessing resident communication skills.

Background: Our goal was to improve pediatric residents' advanced communication skills in the setting of referral to address the entrustable professional activity of subspecialty referral identified by the American Board of Pediatrics. To accomplish this aim, we created a referral and consultation curriculum to teach and assess core communication skills in subspecialty referral involving an adolescent with syncope, an anxiety-provoking symptom that is rarely associated with serious pathology.

Methods: We utilized blended multimodal educational interventions to improve resident communication skills in referral of patients.