Contiguous Xp21 deletion involving Duchenne muscular dystrophy and McLeod neuroacanthocytosis syndrome results in rapidly progressive and fatal cardiomyopathy.

Dilated cardiomyopathy is an expected manifestation and common cause of death in patients with Duchenne muscular dystrophy. We present an unusually rapid progression of cardiomyopathy in a boy with Duchenne muscular dystrophy. Expanded genetic testing revealed a contiguous Xp21 deletion involving dystrophin and XK genes, responsible for Duchenne muscular dystrophy and McLeod neuroacanthocytosis syndrome, respectively, resulting in a more severe cardiac phenotype.

Stress and value: the student perspective on utilizing real vs. actor patients in objective structured clinical examinations.

Background: Studies have shown objective structured clinical examinations (OSCEs) to be one of the most reliable tools in assessing clinical performance; however in Pediatrics they primarily use manikins, simulators or parent actors which limits the comprehensiveness of the assessment. In our Pediatric Clerkship, medical students are evaluated using a standardized rubric during a clinical evaluation exercise (CEX) with real patients. This study assessed medical students' perceived stress levels and the educational value of the CEX compared an OSCE.

American College of Allergy, Asthma and Immunology members' preferred steps 1 to 3 asthma maintenance and reliever therapy and incomplete insurance coverage indicated as main practice hurdle.

Background: New asthma guidelines (GINA, 2022; NAEPP EPR-4, 2020) include considerable changes in treatment recommendations, specifically regarding anti-inflammatory rescue and Single MAintenance and Reliever Therapy (SMART).

Objective: To explore American College of Allergy, Asthma and Immunology members' preferred treatment and perceived hurdles.

Methods: A survey (SurveyMonkey) regarding steps 1 to 3 asthma therapy was e-mailed to American College of Allergy, Asthma and Immunology members.

Use of Image Guidance During Pediatric Venovenous Extracorporeal Membrane Oxygenation Cannulation: A Survey of the American Pediatric Surgical Association.

This study evaluated practices for image guidance during placement of bicaval dual-lumen (BCDL) venovenous extracorporeal membrane oxygenation (VV-ECMO) cannulas in pediatric and adolescent patients and elucidates reasoning behind surgeon practices. A survey covering VV-ECMO practice and opinions was distributed by the American Pediatric Surgical Association (APSA) to all attending members. A total of 110 pediatric surgeons responded (11.

Takotsubo cardiomyopathy in a 4-year-old female with pneumococcal meningitis.

A previously healthy 4-year-old female presented in cardiogenic shock with pneumococcal meningitis. Findings on echocardiogram raised suspicion for takotsubo cardiomyopathy. With supportive care, left ventricular systolic function normalised.

Discontinuation of the OriGen® Dual-Lumen Right Atrial Cannula Decreased Venovenous ECMO Usage in Neonates and Older Children: A Survey of the American Pediatric Surgical Association.

Introduction: Dual-lumen cannulas for venovenous (VV)-ECMO are widely used in pediatric patients. The popular OriGen® dual-lumen right atrial cannula was discontinued in 2019 without a comparable replacement.

Methods: A survey covering VV-ECMO practice and opinions was distributed to attending members of the American Pediatric Surgical Association.

Ensuring equitable access to guideline-based asthma care across the lifespan: Tips and future directions to the successful implementation of the new NAEPP 2020 guidelines, a Work Group Report of the AAAAI Asthma, Cough, Diagnosis, and Treatment Committee.

The most recent recommendations from the 2020 National Asthma Education and Prevention Program Update and Global Initiative for Asthma 2021 guide evidence-based clinical decision making. However, given the present state of health disparities by age, income, and race, the equitable implementation and dissemination of these guidelines will be unlikely without further guidance. This work group report reviews the current state of the new asthma guideline implementation; presents updated evidence-based therapeutic options with attention to specific patient populations; and addresses barriers to the implementation of these guidelines in minoritized, historically marginalized, and underresourced communities.

Medulloblastoma Presenting As Isolated Leptomeningeal Enhancement With No Primary Mass.

Medulloblastoma presenting with diffuse leptomeningeal enhancement and no identified intra-parenchymal primary mass is extremely rare. A 14-year-old previously healthy boy presented with a three-week history of symptoms consistent with increased intracranial pressure (ICP). Magnetic resonance imaging (MRI) revealed diffuse leptomeningeal enhancement which prompted consideration of infectious, inflammatory, and neoplastic etiologies.

Financial Implications of Short Stay Pediatric Hospitalizations.

Background: Observation status (OBS) stays incur similar costs to low-acuity, short-stay inpatient (IP) hospitalizations. Despite this, payment for OBS is likely less and may represent a financial liability for children's hospitals. Thus, we described the financial outcomes associated with OBS stays compared to similar IP stays by hospital and payer.

A Smartphone-Based Self-management Intervention for Individuals With Bipolar Disorder (LiveWell): Protocol Development for an Expert System to Provide Adaptive User Feedback.

Background: Bipolar disorder is a severe mental illness that results in significant morbidity and mortality. While pharmacotherapy is the primary treatment, adjunctive psychotherapy can improve outcomes. However, access to therapy is limited.

A Smartphone-Based Self-Management Intervention for Individuals with Bipolar Disorder (LiveWell): Qualitative Study on User Experiences of the Behavior Change Process.

Background: Bipolar disorder is a severe mental illness characterized by recurrent episodes of depressed, elevated, and mixed mood states. The addition of psychotherapy to pharmacological management can decrease symptoms, lower relapse rates, and improve quality of life; however, access to psychotherapy is limited. Mental health technologies such as smartphone apps are being studied as a means to increase access to and enhance the effectiveness of adjunctive psychotherapies for bipolar disorder.

A Smartphone-Based Self-management Intervention for Bipolar Disorder (LiveWell): User-Centered Development Approach.

Background: Bipolar disorder is a serious mental illness that results in significant morbidity and mortality. Pharmacotherapy is the primary treatment for bipolar disorder; however, adjunctive psychotherapy can help individuals use self-management strategies to improve outcomes. Yet access to this therapy is limited.

Development of Coaching Support for LiveWell: A Smartphone-Based Self-Management Intervention for Bipolar Disorder.

Despite effective pharmacological treatment, bipolar disorder is a leading cause of disability due to recurrence of episodes, long episode durations, and persistence of interepisode symptoms. While adding psychotherapy to pharmacotherapy improves outcomes, the availability of adjunctive psychotherapy is limited. To extend the accessibility and functionality of psychotherapy for bipolar disorder, we developed LiveWell, a smartphone-based self-management intervention.

Factors Influencing Poor Outcomes in Synthetic Tissue-Engineered Tracheal Replacement.

Objectives: Humans receiving tissue-engineered tracheal grafts have experienced poor outcomes ultimately resulting in death or the need for graft explantation. We assessed the performance of the synthetic scaffolds used in humans with an ovine model of orthotopic tracheal replacement, applying standard postsurgical surveillance and interventions to define the factors that contributed to the complications seen at the bedside.

Study Design: Large animal model.

Quantification of tissue-engineered trachea performance with computational fluid dynamics.

Objectives/hypothesis: Current techniques for airway characterization include endoscopic or radiographic measurements that produce static, two-dimensional descriptions. As pathology can be multilevel, irregularly shaped, and dynamic, minimal luminal area (MLA) may not provide the most comprehensive description or diagnostic metric. Our aim was to examine the utilization of computational fluid dynamics (CFD) for the purpose of defining airway stenosis using an ovine model of tissue-engineered tracheal graft (TETG) implantation.

Outcomes in pediatric patients with abdominal compartment syndrome following urgent exploratory laparotomy.

Background/purpose: Abdominal compartment syndrome (ACS) is a serious condition with high mortality in critically ill children. Our objectives were to characterize the incidence of ACS in pediatric patients who underwent urgent exploratory laparotomy and to compare outcomes of patients with and without ACS.

Methods: This retrospective review examined pediatric patients (0-18years) who underwent urgent exploratory laparotomy over a 2-year period.

A review of calcium channel antagonists in the treatment of pediatric hypertension.

All children aged > or = 3 years should have an annual blood pressure (BP) measurement taken during a routine physical examination. Physicians should become familiar with recommended pediatric normative BP tables. BP above the 95th percentile may require drug therapy.

Exercise assessment in infants after cardiac transplantation.

Background: Few data describe exercise performance after cardiac transplantation during infancy. The aim of this study was to compare the cardiorespiratory response to exercise in healthy subjects with that of subjects who had undergone heart transplantation during infancy to treat hypoplastic left heart syndrome.

Methods: Subjects (24 heart transplant recipients and 25 healthy controls) exercised on a treadmill using pediatric ramp protocols.

Use of a consensus building approach to plan speech services for children with cleft palate in India.

Cleft lip and palate (CLP) is a common birth defect worldwide. While surgical repair can normalize appearance, debilitating speech disorders frequently persist. Speech-language pathology (SLP) services are needed to address these disorders.