Efficacy and safety of macitentan in Fontan-palliated patients: 52-week randomized, placebo-controlled RUBATO Phase 3 trial and open-label extension.

Objectives: The efficacy and safety of macitentan, an endothelin receptor antagonist, were assessed in a 52-week, prospective, multicenter, double-blind, randomized, placebo-controlled, parallel-group study assessing the efficacy and safety of macitentan in Fontan-palliated adult and adolescent patients (RUBATO-DB) and an open-label extension trial (RUBATO-OL).

Methods: Patients aged 12 years and older with New York Heart Association functional class II or III underwent total cavopulmonary connection more than 1 year before screening and showed no signs of Fontan failure/clinical deterioration. In RUBATO-DB, the primary efficacy end point was change in peak oxygen consumption from baseline to week 16; secondary end points were change from baseline over 52 weeks in peak oxygen consumption and change in mean count/minute of daily physical activity via accelerometer from baseline to week 16.

Reduced Likelihood of Hospitalization With the JN.1 or HV.1 Severe Acute Respiratory Syndrome Coronavirus 2 Variants Compared With the EG.5 Variant.

Within a multistate viral genomic surveillance program, we evaluated whether proportions of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infections attributed to the JN.1 variant and to XBB-lineage variants (including HV.1 and EG.

Medical Therapy and Monitoring in Adult Congenital Heart Disease Heart Failure.

Heart failure (HF) in adult congenital heart disease (ACHD) is an increasingly common problem facing ACHD and advanced heart disease and transplant providers. Patients are highly nuanced, and therapies are poorly studied. Standard HF medications are often used in patients who are not targets of large clinical trials.

A Guide to Expanding the Use of Buprenorphine Beyond Standard Initiations for Opioid Use Disorder.

Buprenorphine has become an important medication in the context of the ongoing opioid epidemic. However, complex pharmacologic properties and varying government regulations create barriers to its use. This narrative review is intended to facilitate buprenorphine use-including non-traditional initiation methods-by providers ranging from primary care providers to addiction specialists.

Safety and Feasibility of Photodynamic Therapy for Ablation of Peripheral Lung Tumors.

Background: Newer navigational bronchoscopy technologies render peripheral lung lesions accessible for biopsy and potential treatment. We investigated whether photodynamic therapy (PDT) delivered via navigational bronchoscopy is feasible and safe for ablation of peripheral lung tumors.

Methods: Two studies evaluated PDT in patients with solid peripheral lung tumors followed by clinical follow-up (nonresection study, N=5) or lobectomy (resection study, N=10).

Temporal trends and predictors of surgical ablation for atrial fibrillation across a multistate healthcare system.

Background: Multiple class I and class IIa recommendations exist related to surgical ablation (SA) of atrial fibrillation (AF) in patients undergoing cardiac surgery.

Objective: Examine temporal trends and predictors of SA for AF in a large US healthcare system.

Methods: We retrospectively analyzed data from the Society for Thoracic Surgery (STS) Adult Cardiac Surgery Database for 21 hospitals in the Providence St.

Treatment of tibial plateau fractures with a novel fenestrated screw system for delivery of bone graft substitute.

Purpose: The purpose of this study was to describe the incidence of subsidence in patients with AO/OTA 41 (tibial plateau) fractures which were repaired with a novel fenestrated screw system to used to deliver CaPO4 bone substitute material to fill the subchondral void and support the articular reduction.

Methods: Patients with unicondylar and bicondylar tibial plateau fractures were treated according to the usual technique of two surgeons. After fixation, the Zimmer Biomet N-Force Fixation System®, a fenestrated screw that allows for the injection of bone substitute was placed and used for injection of the proprietary calcium phosphate bone graft substitute into the subchondral void.

Potential implications of DMET ontogeny on the disposition of commonly prescribed drugs in neonatal and pediatric intensive care units.

: Pediatric patients, especially neonates and infants, are more susceptible to adverse drug events as compared to adults. In particular, immature small molecule drug metabolism and excretion can result in higher incidences of pediatric toxicity than adults if the pediatric dose is not adjusted.: We reviewed the top 29 small molecule drugs prescribed in neonatal and pediatric intensive care units and compiled the mechanisms of their metabolism and excretion.

Congenital myasthenic syndrome caused by a frameshift insertion mutation in .

Objective: Description of a new variant of the glutamine-fructose-6-phosphate transaminase 1 () gene causing congenital myasthenic syndrome (CMS) in 3 children from 2 unrelated families.

Methods: Muscle biopsies, EMG, and whole-exome sequencing were performed.

Results: All 3 patients presented with congenital hypotonia, muscle weakness, respiratory insufficiency, head lag, areflexia, and gastrointestinal dysfunction.

Safety of dabigatran etexilate for the secondary prevention of venous thromboembolism in children.

This open-label, single-arm, prospective cohort trial is the first phase 3 safety study to describe outcomes in children treated with dabigatran etexilate for secondary venous thromboembolism (VTE) prevention. Eligible children aged 12 to <18 years (age stratum 1), 2 to <12 years (stratum 2), and >3 months to <2 years (stratum 3) had an objectively confirmed diagnosis of VTE treated with standard of care (SOC) for ≥3 months, or had completed dabigatran or SOC treatment in the DIVERSITY trial (NCT01895777) and had an unresolved clinical thrombosis risk factor requiring further anticoagulation. Children received dabigatran for up to 12 months, or less if the identified VTE clinical risk factor resolved.

Reduction in coronary artery bypass grafting surgery mortality and morbidity during a 3-year multicenter quality improvement project.

Objective: Risk-adjusted operative mortality is a key quality measure for isolated coronary artery bypass grafting. Through a multicenter quality improvement initiative, we sought to improve this measure at 14 surgical programs within a large and geographically dispersed health care system.

Methods: Observed mortality and combined mortality/morbidity rates for isolated coronary artery bypass grafting were collected from January 2014 to June 2017.

and Mutations Implicate RAB5 Regulation in Nephrotic Syndrome.

Background: Steroid-resistant nephrotic syndrome (SRNS) is a frequent cause of CKD. The discovery of monogenic causes of SRNS has revealed specific pathogenetic pathways, but these monogenic causes do not explain all cases of SRNS.

Methods: To identify novel monogenic causes of SRNS, we screened 665 patients by whole-exome sequencing.

Clinical Course of a Patient With Kidney Failure Due to Isolated Bilateral Renal Extramedullary Plasmacytomas.

Plasmacytomas are rare immunoproliferative monoclonal plasma cell diseases of lymphoid lineage that may present in an isolated or systemic manner. Systemic involvement is much more common than occurrences isolated to a particular organ, and for this reason, it is imperative to rule out systemic involvement for osseous and nonosseous isolated neoplasms. These neoplasms present unique challenges due to their location, extent of involvement, vague presentation, and dearth of treatment protocol.

Prediction of clinical deterioration after admission from the pediatric emergency department.

Background: An ongoing threat to hospitalized patients is delayed recognition of clinical deterioration and its association with increased morbidity and mortality.

Objective: This study evaluated the ability of Pediatric Emergency Medicine (PEM) clinicians to predict clinical deterioration of patients admitted from the Pediatric Emergency Department (PED). Clinical deterioration was defined as unanticipated transfer to an Intensive Care Unit (ICU) within 12 h of PED-to-ward admission.

Effects of macro- and micronutrients on exercise-induced hepcidin response in highly trained endurance athletes.

Iron deficiency has ergolytic effects on athletic performance. Exercise-induced inflammation impedes iron absorption in the digestive tract by upregulating the expression of the iron regulatory protein, hepcidin. Limited research indicates the potential of specific macro- and micronutrients on blunting exercise-induced hepcidin.

A Randomized Controlled Trial of a Citywide Emergency Department Care Coordination Program to Reduce Prescription Opioid Related Emergency Department Visits.

Background: Increasing prescription overdose deaths have demonstrated the need for safer emergency department (ED) prescribing practices for patients who are frequent ED users.

Objectives: We hypothesized that the care of frequent ED users would improve using a citywide care coordination program combined with an ED care coordination information system, as measured by fewer ED visits by and decreased controlled substance prescribing to these patients.

Methods: We conducted a multisite randomized controlled trial (RCT) across all EDs in a metropolitan area; 165 patients with the most ED visits for complaints of pain were randomized.

-associated developmental disorders exhibit distinct classes of mutations with variable expression and tissue distribution.

Mosaicism is increasingly recognized as a cause of developmental disorders with the advent of next-generation sequencing (NGS). Mosaic mutations of have been associated with the widest spectrum of phenotypes associated with overgrowth and vascular malformations. We performed targeted NGS using 2 independent deep-coverage methods that utilize molecular inversion probes and amplicon sequencing in a cohort of 241 samples from 181 individuals with brain and/or body overgrowth.

Multicenter Prospective Clinical Series Evaluating Radiofrequency Ablation in the Treatment of Painful Spine Metastases.

Background: Radiofrequency ablation (RFA) of vertebral body metastases (VBM) has been reported as safe and effective in retrospective studies. This single-arm prospective multicenter clinical study evaluates RFA in the treatment of painful VBM.

Methods: Fifty patients with VBM were prospectively enrolled during a 13-month period at eight US centers under an IRB-approved study.

The data do not seem to support a benefit to BCAA supplementation during periods of caloric restriction.

J Int Soc Sports Nutr 13:1-015-0112-9, 2016 describe the efficacy of branched chain amino acid (BCAA) supplementation and resistance training for maintaining lean body mass during a calorie-restricted diet, and claim that this occurs with concurrent losses in fat mass. However, the reported results appear to be at odds with the data presented on changes in fat mass. This letter discusses the issues with the paper.

Pediatric Ingestions: Emergency Department Management.

Pediatric ingestions present a common challenge for emergency clinicians. Each year, more than 50,000 children aged less than 5 years present to emergency departments with concern for unintentional medication exposure, and nearly half of all calls to poison centers are for children aged less than 6 years. Ingestion of magnetic objects and button batteries has also become an increasing source of morbidity and mortality.

Long-Term Results of Stenting versus Endarterectomy for Carotid-Artery Stenosis.

Background: In the Carotid Revascularization Endarterectomy versus Stenting Trial, we found no significant difference between the stenting group and the endarterectomy group with respect to the primary composite end point of stroke, myocardial infarction, or death during the periprocedural period or any subsequent ipsilateral stroke during 4 years of follow-up. We now extend the results to 10 years.

Methods: Among patients with carotid-artery stenosis who had been randomly assigned to stenting or endarterectomy, we evaluated outcomes every 6 months for up to 10 years at 117 centers.

Pilot Study of Adding Vincristine, Topotecan, and Cyclophosphamide to Interval-Compressed Chemotherapy in Newly Diagnosed Patients With Localized Ewing Sarcoma: A Report From the Children's Oncology Group.

Background: The combination of topotecan and cyclophosphamide is active in relapsed Ewing sarcoma family of tumors (ESFT). The feasibility of adding these agents combined with vincristine (vincristine-topotecan-cyclophosphamide [VTc]) to standard five-drug chemotherapy with vincristine-doxorubicin-cyclophosphamide (VDC) and ifosfamide-etoposide (IE) administered in an interval-compressed (2-week instead of 3-week intervals) schedule was investigated.

Procedure: Newly diagnosed patients with localized ESFT < 31 years, with good performance status and adequate organ function were eligible.