Safety and efficacy of valoctocogene roxaparvovec with prophylactic glucocorticoids: 1-year results from the phase 3b, single-arm, open-label GENEr8-3 study.

Background: Valoctocogene roxaparvovec, an adeno-associated virus vector that transfers a human factor VIII (FVIII) coding sequence to hepatocytes, provides bleeding protection for people with severe hemophilia A (HA).

Objective: Determine the efficacy and safety of valoctocogene roxaparvovec with concomitant prophylactic glucocorticoids in the open-label, single-arm, phase 3b GENEr8-3 trial.

Methods: Participants with severe HA who were using HA prophylaxis received one 6x10 vg/kg infusion of valoctocogene roxaparvovec concomitantly with daily prophylactic glucocorticoids (40 mg prednisolone equivalent/d weeks 0‒8; taper to 5 mg/d weeks 9‒19).

Allogeneic haematopoietic stem-cell transplantation for children with refractory systemic juvenile idiopathic arthritis and associated lung disease: outcomes from an international, retrospective cohort study.

Background: Systemic juvenile idiopathic arthritis-related lung disease (sJIA-LD) is a severe complication in patients with treatment-refractory systemic juvenile idiopathic arthritis (sJIA). The objective of this study was to evaluate the effect of allogeneic haematopoietic stem-cell transplantation (HSCT) in a cohort of children with sJIA-LD.

Methods: This international, retrospective cohort study was performed in nine hospitals across the USA and Europe in children with sJIA-LD who had received allogeneic HSCT.

Sexual and reproductive health considerations in the care of young adults with inflammatory bowel disease: A multidisciplinary conversation.

The effects of inflammatory bowel disease (IBD) and the medications used to treat it on sexual and reproductive health can be significant, impacting the quality of life of patients across gender identities. This article presents insights from a roundtable discussion facilitated by the Crohn's and Colitis Young Adults Network (CCYAN) between young adult patients with IBD and medical professionals, including physicians, nurses, psychologists, and trainees/medical students. It underscores the distinction between sexual and reproductive health, emphasizing the need to address both aspects comprehensively.

Non-invasive removal of a misplaced and knotted guidewire during ultrasound-guided central venous catheter insertion in a hybrid operating room: a case report.

Background: The standard of care for placement of a central venous catheter (CVC) includes a real-time ultrasound (US)-guided technique. We describe a rare case in which the guidewire penetrated the posterior wall of the vessel, forming a knot, which precluded simple removal. This occurred despite the procedure being performed under real-time US guidance.

Bleed treatment with eptacog beta (rFVIIa) results in a low incidence of rebleeding in adult and adolescent patients with haemophilia A or B with inhibitors.

Introduction: Eptacog beta is a novel human recombinant FVIIa approved for use in the United States, European Union, United Kingdom and Mexico for the treatment and control of bleeding in patients with haemophilia A or B with inhibitors (≥12 years). It is also indicated for perioperative care in the same patient population in Europe and the United Kingdom.

Aim: To assess the incidence of rebleeding and review treatment outcomes in subjects with haemophilia with inhibitors enrolled in the phase 3 PERSEPT 1 clinical trial.

Antibiotic Management and Imaging Practices of Sinusitis-Related Epidural Empyema Among Pediatric Centers.

A survey of pediatric infectious diseases clinicians from 49 children's hospitals demonstrated variability across institutions in the treatment of epidural empyema from sinus extension, including antibiotic route, selection, and impact of neuroimaging on determining duration of therapy.

Behavioral Economic Strategies Increase Adverse Event Reporting in Pediatric Anesthesia.

Introduction: Identification and reporting of severe adverse events (SAEs) during anesthesia care remains critical in identifying areas of improvement in perioperative patient care. Although many healthcare organizations rely on the self-reporting of SAEs, under-reporting may limit the identification of the true incidence of these events. To circumvent these barriers, many healthcare systems leverage the Electronic Medical Record (EMR) by incorporating an Anesthesia Information Management System (AIMS).

Default Antibiotic Order Durations for Skin and Soft Tissue Infections in Outpatient Pediatrics: A Cluster Randomized Trial.

Background: Antibiotic durations for uncomplicated skin/soft tissue infections (SSTI) often exceed the guideline-recommended 5-7 days. We assessed the effectiveness of a default duration order panel in the Electronic Health Record (EHR) to reduce long prescriptions.

Methods: Cluster randomized trial of a SSTI order panel with default antibiotic durations (implemented 12/2021), compared to a control panel (no decision support) in 14 pediatric primary care clinics.

Remote monitoring of social attention in neurogenetic syndromes and idiopathic neurodevelopmental disability.

Social attention is a key aspect of neurodevelopment and is significantly altered in neurodevelopmental genetic syndromes and many individuals with idiopathic autism spectrum disorder (ASD). The primary aim of the present study was to examine the psychometric properties of webcam-collected social attention measurements, including four new specific aspects of social attention, in three genetic syndromes (PTEN Hamartoma Tumor Syndrome-PHTS; Malan Syndrome-NFIX; and SYNGAP1-related disorder-SYNGAP1), a mixed group of other neurodevelopmental genetic syndromes (Other NDGS), and individuals with a range of idiopathic neurodevelopmental disorder (NDD). The secondary aim was to evaluate the construct validity of these social attention measurements, including evaluating known-groups validity across study groups and concurrent validity for separating ASD and non-ASD cases.

Point of Care Ultrasonography for the Evaluation of Ocular Emergencies.

Ocular point-of-care ultrasound (POCUS) has emerged as a pivotal tool in evaluating ocular emergencies in the emergency department (ED), addressing millions of annual visits for eye complaints in the United States. Compared to traditional imaging methods, ocular POCUS's advantages include efficiency, repeatability, and safety. Adequate patient preparation, positioning, and transducer selection are essential for obtaining high-quality images.

Neoantigen DNA vaccines are safe, feasible, and induce neoantigen-specific immune responses in triple-negative breast cancer patients.

Background: Neoantigen vaccines can induce or enhance highly specific antitumor immune responses with minimal risk of autoimmunity. We have developed a neoantigen DNA vaccine platform capable of efficiently presenting both HLA class I and II epitopes and performed a phase 1 clinical trial in triple-negative breast cancer patients with persistent disease on surgical pathology following neoadjuvant chemotherapy, a patient population at high risk of disease recurrence.

Methods: Expressed somatic mutations were identified by tumor/normal exome sequencing and tumor RNA sequencing.

Liberation from Respiratory Support in Bronchopulmonary Dysplasia.

Objective: To estimate the association between the mode of respiratory support administered at 36 weeks' post-menstrual age (PMA) with time-to-liberation from respiratory support (LRS) in infants with grade 2/3 bronchopulmonary dysplasia (BPD).

Study Design: Daily respiratory support data were abstracted for infants born <32 weeks' gestation with grade 2/3 BPD enrolled in the Children's Hospitals Neonatal Database between 2017 and 2022. The main exposure was the mode of respiratory support received at 36 weeks' PMA: high flow nasal cannula >2 L/min (HFNC), continuous positive airway pressure (CPAP), non-invasive positive pressure ventilation (NIPPV), or mechanical ventilation (MV).

Transition to Enteral Triazole Antifungal Therapy for Pediatric Invasive Candidiasis: Secondary Analysis of a Multicenter Cohort Study Conducted by the Pediatric Fungal Network.

Of 319 children with invasive candidiasis, 67 (21%) transitioned from intravenous to enteral antifungal therapy. Eight (12%) transitioned back to intravenous antifungal therapy, one due to perceived treatment failure defined by clinical progression or worsening. Global treatment response at study completion was successful in 66 participants who transitioned to enteral therapy.

Development of a Patient Guidebook for Inpatient Child and Adolescent Psychiatry Units.

An inpatient psychiatric stay for youth should go beyond acute containment of risk and pharmacologic changes and, instead, offer tailored evidence-based treatments in a manner that is individualized for the patient and caregivers. A unique, transdiagnostic guidebook can be utilized within the inpatient hospitalization structure in a way that adds value to the treatment but does not burden the clinical floor staff. This study describes the process of creating a patient-centered youth guidebook, implementation, and recommendations for others wanting to consider the use of a similar modality.

Concurrent nodular lymphocytic myositis and myasthenia gravis. A case report.

Localized painful nodules associated with focal myositis have been previously documented, either as isolated occurrences or in conjunction with systemic illnesses, such as polymyositis [1-4]. These nodules typically manifest over the course of 2-8 weeks and exhibit histological features consistent with inflammatory myositis. Here we present a unique case of a patient with a history of myasthenia gravis, who experienced exacerbation of myasthenic symptoms, accompanied by the development of palpable, tender nodules on her thighs and proximal weakness.