International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA.

Introduction: The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.

Methods: Clinical outcome assessments for the top 10 priority domains identified in the Voice of the Patient Report for Rett syndrome were compiled and available psychometric data were extracted.

Evolving Food Allergy Clinical Trials to Become More Patient-Centered.

The current FDA paradigm may not fully capture important patient-centered outcomes or measure a primary outcome that is truly meaningful to patients. Patient reported outcome measures (PROMs) are standardized tools measuring the patient's experience in food allergy clinical trials, which can help support shared decision-making (SDM) and further our understanding of treatment impact. Food allergy PROMs include quality of life (QoL), health state utility (HSU), severity, and self-efficacy measures.

Creative Arts Therapy Reduces Psychological Distress in Non-Patient-Facing Healthcare Workers.

Objective: To assess the feasibility, acceptability, and efficacy of a 12-week in-person Creative Arts Therapy intervention in reducing psychological distress and burnout symptoms in non-patient-facing healthcare workers.

Background: Burnout and psychological distress among non-patient-facing healthcare workers are significant and understudied problems in healthcare systems.

Methods: Non-patient-facing healthcare workers with burnout symptoms were randomly assigned to one of four Creative Arts Therapy modalities (art, music, creative writing, or dance/movement) or a control group.

Children Requiring 3 or More Days of Invasive Ventilation: Secondary Analysis of Post-Discharge Change in Caregiver Employment.

Objectives: To describe frequency of, and risk factors, for change in caregiver employment among critically ill children with acute respiratory failure.

Design: Preplanned secondary analysis of prospective cohort dataset, 2018-2021.

Setting: Quaternary Children's Hospital PICU.

Caregiver worry about COVID-19 as a predictor of social mitigation behaviours and SARS-CoV-2 infection in a 12-city U.S. surveillance study of households with children.

Objective: Understanding compliance with COVID-19 mitigation recommendations is critical for informing efforts to contain future infectious disease outbreaks. This study tested the hypothesis that higher levels of worry about COVID-19 illness among household caregivers would predict lower (a) levels of overall and discretionary social exposure activities and (b) rates of household SARS-CoV-2 infections.

Methods: Data were drawn from a surveillance study of households with children ( = 1913) recruited from 12 U.

Neighborhood Disadvantage and Injury Mechanism, Severity, and Outcomes in Pediatric Trauma.

Background: Traumatic injuries remain the leading cause of death in children aged 1-14. Previous research demonstrates a link between lower socioeconomic status (SES) and higher pediatric injury morbidity and mortality. There is scant research exploring the relationship between neighborhood disadvantage and pediatric trauma.

Use of Right Ventricular Free-Wall Strain in a Multivariable Estimate of Right Ventricular-Arterial Coupling in Pediatric Pulmonary Arterial Hypertension.

Background: Right ventricular-arterial coupling (RVAC) describes the relationship between right ventricular contractility and pulmonary vascular afterload. Noninvasive surrogates for RVAC using echocardiographic estimates of right ventricular function, such as tricuspid annular plane systolic excursion (TAPSE), have been shown to correlate with invasively measured RVAC and predict clinical outcomes in pediatric pulmonary arterial hypertension. However, given the limitations of TAPSE at accurately estimating right ventricular function in children, we hypothesized that a multivariable estimate of RVAC using right ventricular free-wall longitudinal strain (RVFW-LS) may perform better than those utilizing TAPSE at predicting clinical outcomes.

Machine Learning-Based Prediction Model for ICU Mortality After Continuous Renal Replacement Therapy Initiation in Children.

Background: Continuous renal replacement therapy (CRRT) is the favored renal replacement therapy in critically ill patients. Predicting clinical outcomes for CRRT patients is difficult due to population heterogeneity, varying clinical practices, and limited sample sizes.

Objective: We aimed to predict survival to ICUs and hospital discharge in children and young adults receiving CRRT using machine learning (ML) techniques.

Staff and caregivers' perceptions of digital storytelling to increase influenza vaccine confidence in an urban safety-net healthcare system.

Background: Myriad risk factors contribute to pediatric influenza vaccination disparities. Digital stories are compelling accounts of lived experience that have been useful in health promotion, especially in minoritized communities. Little is known about how they are perceived as a behavioral intervention to improve influenza vaccination confidence in safety-net healthcare systems.

Bedside transfontanelle drain placement reduces the need for operative intervention in infants with subdural fluid collections.

Objective: Subdural fluid collection is a common neurosurgical condition in the pediatric population. Patients requiring surgical intervention have historically been managed with subdural drains, subdural-subgaleal shunting, subdural-peritoneal shunting, and mini-craniotomies. An alternative procedure for patients with an open anterior fontanelle is bedside transfontanelle drainage.

Synchrotron-Based Phase-Contrast Micro-CT Combined With Histology to Decipher Differences Between Hereditary and Sporadic Pediatric Pulmonary Veno-Occlusive Disease.

Pulmonary veno-occlusive disease (PVOD) is a lethal variant of pulmonary hypertension. The degree of pulmonary arterial involvement varies. Here, we compare two PVOD patients who were transplanted at 8 years of age, whereof one is a homozygous mutation carrier.

Cost-effectiveness of oral immunotherapy for egg allergy according to age of therapy commencement.

Background: Egg oral immunotherapy can induce desensitization or remission of egg allergy in children.

Objective: To determine the cost-effectiveness of OIT for raw egg allergy in school-aged children compared to egg avoidance, and the most cost-effective age at which to commence treatment.

Methods: A decision-analytic Markov model estimated the health and cost outcomes of 1000 children aged four years with egg allergy, comparing different ages of OIT commencement (from ages 4-12, inclusive) versus ongoing egg avoidance.

Having more resilience factors only gets you so far in the context of neighborhood disadvantage: understanding differential predictors of internalizing symptoms and body mass index in youth with spina bifida.

Objective: The primary aim of this study was to determine if neighborhood disadvantage predicted internalizing symptoms and body mass index (BMI) in youth with spina bifida (SB), while accounting for several sociodemographic factors. We also explored whether resilience factors helped explain associations between neighborhood disadvantage and internalizing symptoms or neighborhood disadvantage and BMI.

Methods: Participants (n = 69, Mage=14.

Asian neuropsychologists: Current state, gaps, barriers, and future directions.

As the field of neuropsychology continues expanding efforts to better recruit providers and serve individuals from diverse populations, understanding the training and practice experiences of neuropsychologists from diverse backgrounds is crucial. Given the diversity of Asian populations, the experiences of Asian neuropsychologists offer a unique opportunity to reflect on the progress made in addressing issues related to diversity, equity, and inclusion (DEI). This information will help address challenges related to education, training, and clinical practice, particularly in meeting growing demands for neuropsychological evaluations among Asian populations and addressing unique challenges.

Donor great vessel free arterial grafts for complex reconstruction during pediatric heart transplantation.

Objective: Prior studies suggest that prolonged donor heart warm ischemia time increases heart transplant mortality. Patients with single-ventricle heart disease requiring transplant with concomitant aortic arch or central pulmonary artery reconstruction present technical challenges that extend donor warm ischemia time using conventional techniques. Studies in larger pediatric and adult patients with single-ventricle anatomy have described the use of prosthetic material for concomitant great vessel reconstruction.

Imaging Findings and Management Strategies for Liver Masses in Children with Predisposition Disorders: A Review by the Pediatric LI-RADS Group.

Liver masses in children with underlying systemic disease or a predisposing syndrome can be benign or malignant, ranging from focal fat to hepatocellular carcinoma (HCC). Knowledge of the underlying condition, the pathophysiologic effect on the liver, and the development of liver disease and specific liver lesions allows radiologists to guide imaging with regard to modality and frequency and give recommendations for biopsy when appropriate. In some predisposition disorders, such as Beckwith Wiedemann spectrum, familial adenomatous polyposis syndrome, and tuberous sclerosis complex, established guidelines for imaging screening exist.

Cognitive Behavioral Therapy Factors Related to Outcomes in Depression Among Youth with HIV.

Purpose: This is a secondary analysis of a multi-site, cluster (site) randomized trial of the efficacy of a combined Health and Wellness Cognitive Behavior Therapy (H&W CBT) and medication management approach for depression in youth with HIV (YWH) compared to standard care. In this study, we explored the association between H&W CBT factors and depression outcomes after 24 weeks of treatment to discover treatment elements associated with symptom reduction.

Methods: Participants (12-24 years of age) were YWH in the United States (US) diagnosed with moderate to severe depression [Quick Inventory of Depressive Symptomatology (QIDS), Clinician-Rated score ≥ 11].

Alternative antibiotic selections during the 2022 amoxicillin shortage in the US.

This study of seven pediatric hospitals identified that the amoxicillin shortage in 2022 resulted in a decline in amoxicillin suspension prescribing by 30.8% with increases in use of amoxicillin non-suspension (+7.9) and broad-spectrum antibiotics.

Evaluating the safety and efficacy of proton radiotherapy for intracranial pediatric ependymomas: A single-arm meta-analysis.

Background: Ependymomas account for 6% to 10% of childhood central nervous system tumors. This study aimed to evaluate the safety and efficacy of proton radiotherapy in intracranial ependymoma patients.

Methods: We performed a systematic review and single-arm meta-analysis.