International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA.

Introduction: The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.

Methods: Clinical outcome assessments for the top 10 priority domains identified in the Voice of the Patient Report for Rett syndrome were compiled and available psychometric data were extracted.

Expert-based physical activity guidelines for metabolic and bariatric surgery patients: a systematic review of randomized controlled trials.

Patients undergoing metabolic and bariatric surgery (MBS) can improve outcomes through a physically active lifestyle. Despite ongoing research, clinical recommendations for physical activity (PA) are not fully developed. For this review, 39 articles representing 24 randomized clinical trials satisfied inclusion criteria.

Advanced strategies for intensive care management of acute liver failure.

Acute liver failure (ALF) is defined as the loss of hepatic function in conjunction with hepatic encephalopathy and coagulopathy. There is histological evidence of profound hepatocyte damage. If it is not aggressively managed, ALF can be fatal within a few days.

AAHPM Assessment Workgroup: Hospice and Palliative Medicine Fellowship Assessment Needs & Directions.

Context: Medical education is moving towards competency based medical education (CBME). This provides opportunities and challenges for the field of Hospice and Palliative Medicine (HPM). The American Academy of Hospice and Palliative Medicine (AAHPM) created the Assessment Workgroup to spearhead a shared vision and process to implement high-quality assessment of fellow performance.

The effects of plasma exchange and glucocorticoids on early kidney function among patients with ANCA-associated vasculitis in the PEXIVAS trial.

Therapeutic plasma exchange (PLEX) is an adjunctive treatment for patients with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis and kidney involvement. Little is known about the effect of PLEX on early changes in kidney function. This post-hoc analysis of the PEXIVAS trial investigated the effects of PLEX on changes in kidney function within 12 months.

Neonatal Seizures and Associated Neurobehavioral Profiles in Preschool Age Children.

Background: Neonatal seizures are common with acute brain injury. Up to 25% of survivors develop postneonatal epilepsy. We hypothesized postneonatal epilepsy diagnosed by age 24 months would increase risk for early markers of neurobehavioral disorders than acute provoked neonatal seizures alone.

Pressure to evade cell-autonomous innate sensing reveals interplay between mitophagy, IFN signaling, and SARS-CoV-2 evolution.

SARS-CoV-2 emerged, and continues to evolve, to efficiently infect humans worldwide. SARS-CoV-2 evades early innate recognition, interferon signaling occurring only in bystander cells. How the virus continues to evolve in the face of innate responses has important consequences, but the pathways involved are incompletely understood.

An expert consensus statement on biomarkers of ageing for use in intervention studies.

Biomarkers of ageing serve as important outcome measures in longevity-promoting interventions. However, there is limited consensus on which specific biomarkers are most appropriate for human intervention studies. This work aimed to address this need by establishing an expert consensus on biomarkers of ageing for use in intervention studies via the Delphi method.

Dynamic contrast-enhanced magnetic resonance lymphangiography: a simple algorithm for image interpretation.

Dynamic contrast-enhanced magnetic resonance lymphangiography is a high-resolution imaging technique that has emerged as the preferred method for evaluating lymphatic anatomy and flow dynamics due to its precise anatomical detail. The lymphatic system has a complex anatomical distribution, and variability is common among individuals with cardiac abnormalities, particularly congenital heart disease. Lymphatic imaging has recently been revolutionized by the introduction of MR lymphangiography.

Rare dysfunctional SCN2A variants are associated with malformation of cortical development.

Objective: SCN2A encodes the voltage-gated sodium (Na+) channel α subunit Na1.2, which is important for the generation and forward and back propagation of action potentials in neurons. Genetic variants in SCN2A are associated with a spectrum of neurodevelopmental disorders.

Understanding Daily Care Experience Preferences Across the Lifespan of Older Adults: Application of Natural Language Processing.

Introduction: Older adults are a heterogeneous group, and their care experience preferences are likely to be diverse and individualized. Thus, the aim of this study was to identify categories of older adults' care experience preferences and to examine similarities and differences across different age groups.

Methods: The initial categories of older adults' care experience preferences were identified through a qualitative review of narrative text (n = 3134) in the ADVault data set.

Annual Research Review: Cash transfer programs and young people's mental health - a review of studies in the United States.

Worldwide, more than one in 10 children or adolescents is diagnosed with a mental disorder. Cash transfer programs, which aim to reduce poverty and improve life outcomes by providing direct cash assistance to families and incentivizing or enabling spending on education, health service use, dietary diversity and savings, have been shown to improve the mental health and well-being of young people in low- and middle-income countries. The goal of this review is to describe cash transfer programs in the United States, to describe potential mechanisms by which cash transfer programs could improve child and adolescent mental health and to summarize any evidence of the impact of cash transfer programs.

Intracranial multimodal monitoring in neurocritical care (Neurocore-iMMM): an open, decentralized consensus.

Background: Intracranial multimodal monitoring (iMMM) is increasingly used in neurocritical care, but a lack of standardization hinders its evidence-based development. Here, we devised core outcome sets (COS) and reporting guidelines to harmonize iMMM practices and research.

Methods: An open, decentralized, three-round Delphi consensus study involved experts between December 2023 and June 2024.

Differential diagnosis and comparison of diagnostic algorithms in children and adolescents with autoimmune encephalitis in Spain: a prospective cohort study and retrospective analysis.

Background: The usefulness of current diagnostic approaches in children with suspected autoimmune encephalitis is unknown. We aimed to assess the diagnosis of autoimmune encephalitis in clinical practice and to compare the performance of two international diagnostic algorithms (one intended for patients of any age [general], the other intended for paediatric patients), with particular emphasis on the evaluation of patients with probable antibody-negative autoimmune encephalitis because this diagnosis suggests that immunotherapy should be continued or escalated but is difficult to establish.

Methods: We did a prospective cohort study that included all patients (<18 years of age) with suspected autoimmune encephalitis recruited at 40 hospitals in Spain whose physicians provided clinical information every 6 months for 2 years or more.

Vagus nerve stimulation in treatment-resistant depression: A one-year, randomized, sham-controlled trial.

Background: Few treatments are available for individuals with marked treatment-resistant depression (TRD).

Objective: Evaluate the safety and effectiveness of FDA-approved adjunctive vagus nerve stimulation (VNS) in patients with marked TRD.

Methods: This 12-month, multicenter, double-blind, sham-controlled trial included 493 adults with marked treatment-resistant major depression who were randomized to active or no-stimulation sham VNS for 12 months.

Evaluating post-cardiac arrest blood pressure thresholds associated with neurologic outcome in children: Insights from the pediRES-Q database.

Background: Current Pediatric Advanced Life Support Guidelines recommend maintaining blood pressure (BP) above the 5th percentile for age following return of spontaneous circulation (ROSC) after cardiac arrest (CA). Emerging evidence suggests that targeting higher thresholds, such as the 10th or 25th percentiles, may improve neurologic outcomes. We aimed to evaluate the association between post-ROSC BP thresholds and neurologic outcome, hypothesizing that maintaining mean arterial pressure (MAP) and systolic blood pressure (SBP) above these thresholds would be associated with improved outcomes at hospital discharge.

Randomized trial investigating the utility of a liver tissue transcriptional biomarker in identifying adult liver transplant recipients not requiring maintenance immunosuppression.

The maintenance of stable allograft status in the absence of immunosuppression, known as operational tolerance, can be achieved in a small proportion of liver transplant recipients, but we lack reliable tools to predict its spontaneous development. We conducted a prospective, multi-center, biomarker-strategy design, immunosuppression withdrawal clinical trial to determine the utility of a predictive biomarker of operational tolerance. The biomarker test, originally identified in a patient cohort with high operational tolerance prevalence, consisted of a 5-gene transcriptional signature measured in liver tissue collected before initiating immunosuppression weaning.