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.

Food insecurity screening practices in a pediatric gastroenterology population.

Food insecurity is a rising concern for US households and leads to adverse child health outcomes. Pediatric gastroenterology providers are uniquely equipped to help guide families experiencing this challenge given their specialized training in nutritional support and dietary therapy for disease management. Hence, this study aimed to evaluate food insecurity screening practices from the perspectives of patient caregivers and healthcare providers in a tertiary pediatric gastroenterology practice.

Comprehensive transition of care for polycystic ovary syndrome from adolescence to adulthood.

Polycystic ovary syndrome (PCOS) is a lifelong chronic condition that affects one in ten females and can be diagnosed in adolescence. As adolescents with PCOS transition to adulthood, counselling for lifestyle management and mental health concerns often transition from involving the family unit to increasingly individual-focused approaches. PCOS is associated with a large range of comorbidities affecting reproductive, metabolic, dermatological, and psychological health.

Actigraphy methodology in the Kids Mod PAH trial: Physical activity as a functional endpoint in pediatric clinical trials.

Pulmonary vasodilator treatment can improve hemodynamics, right ventricular function, symptoms, and survival in pediatric pulmonary hypertension (PH). However, clinical trial data are lacking due to many constraints. One major limitation is the lack of relevant trial endpoints reflective of hemodynamics or functional status in patients in whom standard exercise testing is impractical, unreliable, or not reproducible.

Patient Screening for Self-Expanding Percutaneous Pulmonary Valves Using Virtual Reality.

Background: In recent years, self-expanding technology to treat pulmonary regurgitation in the native right ventricular outflow tract became Food and Drug Administration approved in the United States and is now routinely used. The current practice for selection of patients who are candidates for these devices includes screening for "anatomic fit," performed by each of the manufacturing companies. Our study aims to validate the use of virtual reality (VR) as a tool for local physician-led screening of patients.

Considerations for Gender-Affirming Hormonal and Surgical Care Among Transgender and Gender Diverse Adolescents and Adults With Congenital Heart Disease.

Background: Transgender and gender diverse (TGD) individuals and long-term survivors with adult congenital heart disease (ACHD) are both growing populations with specialized needs. No studies assess temporal trends or evaluate the care of TGD individuals with ACHD.

Methods And Results: Meetings between congenital cardiology and gender-affirming care specialists identified unique considerations in TGD individuals with ACHD.

Kids Mod PAH trial: A multicenter trial comparing mono- versus duo-therapy for initial treatment of pediatric pulmonary hypertension.

Pulmonary hypertension (PH) is a significant health problem that contributes to high morbidity and mortality in diverse cardiac, pulmonary, and systemic diseases in children. Evidence-based advances in PH care have been challenged by a paucity of quality endpoints for assessing clinical course and the lack of robust clinical trial data to guide pharmacologic therapies in children. While the landmark adult AMBITION trial demonstrated the benefit of up-front combination PH therapy with ambrisentan and tadalafil, it remains unknown whether upfront combination therapy leads to more rapid and sustained clinical benefits in children with various categories of PH.

Parental age effects and Rett syndrome.

Rett syndrome (RTT) is a progressive neurodevelopmental disorder, and pathogenic Methyl-CpG-binding Protein 2 (MECP2) variants are identified in >95% of individuals with typical RTT. Most of RTT-causing variants in MECP2 are de novo and usually on the paternally inherited X chromosome. While paternal age has been reported to be associated with increased risk of genetic disorders, it is unknown whether parental age contributes to the risk of the development of RTT.

Twitter Trends for Celiac Disease and the Gluten-Free Diet: Cross-sectional Descriptive Analysis.

Background: Few studies have systematically analyzed information regarding chronic medical conditions and available treatments on social media. Celiac disease (CD) is an exemplar of the need to investigate web-based educational sources. CD is an autoimmune condition wherein the ingestion of gluten causes intestinal damage and, if left untreated by a strict gluten-free diet (GFD), can result in significant nutritional deficiencies leading to cancer, bone disease, and death.

See one, see one, teach one - Decisions on allocating intubation opportunities in pediatric emergency medicine.

Background: Decisions about who should perform tracheal intubation in academic settings must balance the needs of trainees to develop competency in pediatric intubation with patient safety. Airway protocols during the COVID-19 pandemic may have reduced opportunities for trainees, representing an opportunity to examine the impact of shifting laryngoscopy responsibilities away from trainees.

Methods: This observational study combined data from 11 pediatric emergency departments in North America participating in either the National Emergency Airway Registry for Children (NEAR4KIDS) or a national pediatric emergency medicine airway education collaborative.

Megacystis Associated With an Underlying ACTA2 Variant and Diagnosis of Multisystemic Smooth Muscle Dysfunction Syndrome: A Case Report.

Fetal megacystis, or an enlarged fetal bladder, is most often attributed to embryological defects, occurring early in gestation. Recent investigations have demonstrated that the underlying etiology of megacystis may be more myriad than originally thought. We present the third reported patient with megacystis due to an ACTA2 Arg179 substitution variant causing Multisystemic Smooth Muscle Dysfunction Syndrome.

Clinical approach to mechanical circulatory support in the transplant patient from the Pediatric Heart Transplant Society.

The use of mechanical circulatory support (MCS) for pediatric patients who have undergone heart transplant has grown rapidly in the past decade. This includes support in the immediate post-transplant period and "rescue" therapy for patient later in their transplant course. Extracorporeal membrane oxygenation (ECMO) remains a standard modality of support for intraoperative concerns and for acute decompensation in the immediate post-transplant period.

Clinical approach to vasoplegia in the transplant patient from the Pediatric Heart Transplant Society.

This manuscript outlines a clinical approach to vasoplegia incorporating the current state of knowledge regarding vasoplegia in pediatric patients immediately post-transplant and to identify modifiable factors both pre- and post-transplant that may reduce post-operative morbidity, end-organ dysfunction, and mortality. Centers participating in the Pediatric Heart Transplant Society (PHTS) were asked to provide their internal protocols and rationale for vasoplegia management, and applicable adult and pediatric data were reviewed. The authors synthesized the above protocols and literature into the following description of clinical approaches to vasoplegia highlighting areas of both broad consensus and of significant practice variation.

Psychosocial predictors of problematic eating in young adults who underwent adolescent bariatric surgery.

Introduction: This study examined problematic eating and eating-related psychopathology among young adults who underwent adolescent bariatric surgery including concurrent and prospective associations with psychosocial factors and weight change.

Methods: VIEW point is a 6-year follow-up study within a prospective observational study series observing adolescents with severe obesity who had bariatric surgery ( = 139) or who presented to nonsurgical lifestyle modification programs ( = 83). Participants completed height/weight measurements, questionnaires, and diagnostic interviews.

Riociguat in children with pulmonary arterial hypertension: The PATENT-CHILD study.

Riociguat, a soluble guanylate cyclase stimulator, is approved for treatment of adults with pulmonary arterial hypertension (PAH). The safety, tolerability, and pharmacokinetics (PK) of oral riociguat in a pediatric population with PAH was assessed in PATENT-CHILD (NCT02562235), a multicenter, single-arm, 24-week, open-label, Phase 3 study. Patients aged 6-17 years in World Health Organization functional class (WHO-FC) I-III treated with stable endothelin receptor antagonists and/or prostacyclin analogs received riociguat equivalent to 0.