Histoplasmosis in a Solid Organ Transplant Patient in a Non-Endemic Region in New York City.

Objective: To present the case of a solid organ transplant recipient with Histoplasmosis in New York City.

Case Report: We present a 39-year-old female liver transplant recipient, who experienced a two-week history of progressive shortness of breath and dyspnea on exertion that interfered with all activities of daily living. Physical examination by the team revealed the patient had a WBC of 11.

Association of age, race, and ethnicity with access, response, and toxicities from CAR-T therapy in children and adults with B-cell malignancies: a review.

Chimeric antigen receptor T cell (CAR-T) therapies are now standard-of-care for several B-cell malignancies, and additional indications are being evaluated. In this review, we survey data on how outcomes after CAR-T therapies vary according to age, race, and ethnicity. We also review the representation of age, racial, and ethnic groups in key CAR-T clinical trials.

Sclerotherapy combined with sirolimus for the treatment of complex cervicofacial lymphatic malformations in infants: avoiding the need for tracheostomy.

Background: Lymphatic malformations (LMs) are low-flow, congenital lesions commonly presenting as asymptomatic masses in the head and neck. However, large lymphangiomas can significantly affect breathing or swallowing, posing considerable treatment challenges.

Methods: A retrospective analysis of complex cervicofacial LMs in infants was conducted over the past 8 years at the Department of Radiology.

Physiologic Pulmonary Phenotyping of Infants Born Preterm and Post-Discharge Respiratory Morbidity.

Objectives: To determine whether airway and parenchymal function identifies subgroups of infants born preterm according to the predominant pulmonary pathophysiology, and whether these subgroups have different risks for respiratory disease during infancy.

Study Design: We prospectively enrolled a cohort of 125 infants born preterm with planned clinical follow-up after NICU discharge. The study included monthly questionnaires for wheeze and visits to a physician or care provider for any respiratory illness.

Pediatric Transplant and Cellular Therapy Consortium RESILIENT Conference on Pediatric Chronic Graft-versus-Host Disease Survivorship after Hematopoietic Cell Transplantation: Part II. Organ Dysfunction and Immune Reconstitution Considerations for children with chronic GVHD after Hematopoietic Cell Transplantation.

While highly morbid forms of chronic graft versus host disease (cGVHD) and severe late effects of allogeneic hematopoietic cell transplant (HCT) can impact children and adults alike, unique considerations arise in pediatric cases regarding diagnosis, monitoring, treatment, and likelihood of resolution. As children can present with atypical features of cGVHD, and with more significant disease due to inability to communicate symptoms, they may be at increased risk for highly morbid forms of cGVHD and incur greater subsequent late effects, which may be more pronounced in those with underlying chromosomal breakage syndromes, with higher prevalence in pediatric HCT recipients. The long-term effects of cGVHD and its therapies include impaired immune reconstitution, leading to increased risks of infection and secondary malignant neoplasms.

Phospho-flow cytometry assays for diagnostic use - A discussion of assay utility and assay development and validation challenges.

Detection of changes in phosphorylation of cell signaling molecules using flow cytometry is termed "phosphoflow" or "phospho-flow cytometry". Phosphoflow has wide application for basic research into the mechanics of cell signaling, for evaluating aberrant signaling in cancerous cells and tissues, for studying efficacy or off-target effects during drug and vaccine development, and for functional assessment of pathogenic variants of genes that are known to play a role in development or function of the immune system. Phosphoflow has not been widely adopted in clinical laboratories owing to the challenges with developing and validating robust assays consistent with clinical laboratory regulatory standards.

Diagnostic testing for hemophagocytic lymphohistiocytosis.

Hemophagocytic lymphohistiocytosis (HLH) is a rare clinical syndrome caused by severe systemic hyperinflammation. HLH can be rapidly fatal if unrecognized or inadequately treated. It is important that clinicians are able to utilize diagnostic testing to assess for HLH and determine the underlying causes including possible inborn errors of immunity (IEI).

Development and internal validation of a nomogram for predicting recurrent respiratory tract infections in children.

Objective: This study aimed to develop and internally validate a nomogram in predicting the risk of recurrent respiratory tract infection (RRTI) in children.

Methods: A retrospective analysis was performed, involving 150 children with RRTI and 151 healthy controls, aged 0-14 years, admitted to or selected from the Pediatric Department of Yixing Hospital of Traditional Chinese Medicine between June 2022 and June 2023. Data were gathered through a comprehensive questionnaire survey on risk factors associated with RRTI.

Simulation-based training improves developmental hip dysplasia examination and diagnosis skills on newborns.

Background: Examination maneuvers used to diagnose developmental hip dysplasia (DDH) translate poorly to video and written curricula. This poses a challenge to teaching the infant hip exam to orthopedic, family medicine, and pediatric trainees. This work investigated the impact of the MiHip simulation-based training program on residents' knowledge, confidence, and exam skills in the simulated setting, and translation of these skills to the clinical setting.

The role of non-respiratory arousals in residual daytime sleepiness in patients with obstructive sleep apnea treated with positive airway pressure: An analysis of the European Sleep Apnea Database registry.

Study Objectives: To assess the impact of the non-respiratory arousal burden at baseline polysomnography (PSG) on residual daytime sleepiness in positive airway pressure (PAP)-treated obstructive sleep apnea (OSA).

Methods: We included OSA patients from the European Sleep Apnea Database registry with available arousal data who had at least 2 treatment follow-up visits. The primary outcome was the Epworth Sleepiness Scale (ESS) score under PAP.

Trajectories of intrinsic connectivity one year post pediatric mild traumatic brain injury: Neural injury superimposed on neurodevelopment.

The developing brain undergoes rapid changes throughout middle childhood and adolescence. The disambiguation of long-term changes in intrinsic activity following pediatric mild traumatic brain injury (pmTBI) from typical development can therefore only be ascertained in longitudinal studies with large sample size and at least three serial assessments. A comprehensive clinical battery and resting-state fMRI data were collected approximately 1-week (N = 263; 8-18 years old), 4-months (N = 192) and 1-year (N = 153) post-injury, with identical visits in a large cohort (N = 228) of age- and sex-matched healthy controls (HC).

Global, regional and national burden of neuroblastoma and other peripheral nervous system tumors, 1990 to 2021 and predictions to 2035: visualizing epidemiological characteristics based on GBD 2021.

Background: Neuroblastoma (NB) is the most common extracranial malignant solid tumor in children, accounting for >15 % of cancer-related deaths in children. We analyzed the epidemiological statistical indicators of neuroblastoma and other peripheral nervous system tumors patients from 1990 to 2021 in Global Burden of Disease (GBD) 2021 database, aiming to provide valuable insights for public health interventions and clinical practices.

Methods: Based on the GBD 2021 database, this study analyzed the incidence, mortality, prevalence, and Disability-Adjusted Life-Years (DALYs) of neuroblastoma and other peripheral nervous system tumors from 1990 to 2021, stratified by sociodemographic development index (SDI) and geographic regions.

Paediatric strategy forum for medicinal product development in diffuse midline gliomas in children and adolescents ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration.

Fewer than 10 % of children with diffuse midline glioma (DMG) survive 2 years from diagnosis. Radiation therapy remains the cornerstone of treatment and there are no medicinal products with regulatory approval. Although the biology of DMG is better characterized, this has not yet translated into effective treatments.

The outcomes of implementing clinical guidelines to manage pediatric diabetic ketoacidosis in emergency department.

Background: Type 1 diabetes is the most common endocrine health condition among youth. Healthcare professionals must consider evidence-based guidelines in managing children and adolescents with diabetic ketoacidosis (DKA). The current study aims to assess the outcomes of implementing clinical guidelines by the American Diabetes Association to manage DKA among pediatrics in an emergency department in Palestine.

One hundred thirty-four germ line PU.1 variants and the agammaglobulinemic patients carrying them.

Leukopoiesis is lethally arrested in mice lacking the master transcriptional regulator PU.1. Depending on the animal model, subtotal PU.

Enhancing trauma cardiopulmonary resuscitation simulation training with the use of virtual reality (Trauma SimVR): Protocol for a randomized controlled trial.

Background: With the increasing availability and use of digital tools such as virtual reality in medical education, there is a need to evaluate their impact on clinical performance and decision-making among healthcare professionals. The Trauma SimVR study is investigating the efficacy of virtual reality training in the context of traumatic in-hospital cardiac arrest.

Methods And Analysis: This study protocol (clinicaltrials.

Efficacy and safety of creatine phosphate sodium in the treatment of viral myocarditis: A systematic review and meta-analysis.

Purpose: To systematically evaluate the efficacy and safety of creatine phosphate sodium in the treatment of viral myocarditis, and to provide guidance for its clinical treatment.

Methods: We conducted a search of The Cochrane Library, PubMed, EMbase, and Web of Science databases to retrieve randomized controlled trials (RCTs) on the use of creatine phosphate sodium (CPS) in the treatment of viral myocarditis. The search was conducted up to April 2024.

Respiratory extracellular vesicle isolation optimization through proteomic profiling of equine samples and identification of candidates for cell-of-origin studies.

Growing evidence supports the importance of extracellular vesicle (EV) as mediators of communication in pathological processes, including those underlying respiratory disease. However, establishing methods for isolating and characterizing EVs remains challenging, particularly for respiratory samples. This study set out to address this challenge by comparing different EV isolation methods and evaluating their impacts on EV yield, markers of purity, and proteomic signatures, utilizing equine/horse bronchoalveolar lavage samples.

A 5-year Single-center Experience on the Use of Emicizumab Prophylaxis in Children With Severe Hemophilia A With and Without Factor VIII Inhibitors.

Objective: Emicizumab promotes efficacious hemostasis in persons with hemophilia A persons with hemophilia A with and without inhibitors. Primary analyses of real-world data and clinical trials have shown emicizumab efficacy and safety; however, long-term data are limited.

Methods: This retrospective study was conducted to assess real-world long-term outcomes of pediatric patients on emicizumab in our hemophilia center between the period of February 2018 and September 2023.

Association of Influenza Vaccination During Pregnancy with Health Outcomes in Mothers and Children: A Population-Based Cohort Study.

Immunization rates of maternal influenza vaccination during pregnancy remain suboptimal, with concerns about potential harm to the mothers and their offspring. We conducted a population-based cohort study, using mother-child linked database in Korea: (a) maternal cohort between December 2019, and March 2022; (b) neonatal cohort between September 2020, and June 2021. Exposure was defined as influenza vaccination during pregnancy.

Randomised In Vitro Study Investigating PEEP-Stability During Application of CPAP With Binasal Prongs and Face Masks.

Aim: Face masks and binasal prongs are commonly used interfaces for applying continuous positive airway pressure (CPAP) in neonatology. We aimed to assess CPAP stability in a randomised controlled in vitro study.

Methods: In a simulated resuscitation scenario of a 1000-g preterm infant with respiratory distress, 20 operators (10 with/without neonatology experience) aimed to maintain a CPAP of 5 cmHO as precisely as possible using face masks or binasal prongs in random order.

Disco-Interacting Protein 2 Homolog B CGG Repeat Expansion in Siblings with Neurodevelopmental Disability and Progressive Movement Disorder.

Background: Trinucleotide repeat expansions are an emerging class of genetic variants associated with various movement disorders. Unbiased genome-wide analyses can reveal novel genotype-phenotype associations and provide a diagnosis for patients and families.

Objective: The aim was to identify the genetic cause of a severe progressive movement disorder phenotype in 2 affected brothers.

Toward Equitable Access to Psychosocial Screening in Canada: Validation of the French-Canadian Psychosocial Assessment Tool.

Objective: The Psychosocial Assessment Tool (PAT) is a brief caregiver report, family-centered, psychosocial risk screening tool widely used in pediatrics and available in many languages. Although French is an official language of Canada, a French-Canadian version of the PAT has not yet been validated, which impedes access to this tool for family psychosocial screening. This study aimed to translate, adapt as necessary, and validate the French-Canadian version of the PAT.

Fixed dosing of alpelisib for children with vascular anomalies: Can we do better?

Severe forms of vascular malformations (VM) can highly impact patients' quality of life and lead to life-threatening organ dysfunction. Numerous VM are caused by somatic activating mutations in the PI3K/AKT/mTOR signalling pathway. Alpelisib, a PIK3CA inhibitor was recently FDA-approved for paediatric PIK3CA-related overgrowth syndrome (PROS).

Neonatal Nutrition and Brain Structure at 7 Years in Children Born Very Preterm.

Importance: Neonatal protein intake following very preterm birth has long lasting effects on brain development. However, it is uncertain whether these effects are associated with improved or impaired brain maturation.

Objective: To assess the association of neonatal protein intake following very preterm birth with brain structure at 7 years of age.