Gastrointestinal Dysfunction Criteria in Critically Ill Children: The PODIUM Consensus Conference.

Context: Prior criteria to define pediatric multiple organ dysfunction syndrome (MODS) did not include gastrointestinal dysfunction.

Objectives: Our objective was to evaluate current evidence and to develop consensus criteria for gastrointestinal dysfunction in critically ill children.

Data Sources: Electronic searches of PubMed and EMBASE were conducted from January 1992 to January 2020, using medical subject heading terms and text words to define gastrointestinal dysfunction, pediatric critical illness, and outcomes.

Early factors for predicting discontinuation to subcutaneous Apomorphine infusion in Parkinson's disease: A prospective analysis of the Thai Apomorphine Registry.

Introduction: Although continuous subcutaneous apomorphine infusion (CSAI) is an effective therapy for Parkinson's disease (PD) with motor fluctuations, data from Asian cohorts is limited. The therapy is often discontinued due to the complexity of its delivery.

Methods: Fifty-one PD patients undergoing CSAI as an add-on therapy were enrolled in the Thai Apomorphine Registry, an electronic database that recorded clinical characteristics and parameters during the 14-consecutive-day titration and long-term follow-up.

Augmented Expansion of Treg Cells From Healthy and Autoimmune Subjects Adult Progenitor Cell Co-Culture.

Recent clinical experience has demonstrated that adoptive regulatory T (Treg) cell therapy is a safe and feasible strategy to suppress immunopathology induction of host tolerance to allo- and autoantigens. However, clinical trials continue to be compromised due to an inability to manufacture a sufficient Treg cell dose. Multipotent adult progenitor cells (MAPC) promote Treg cell differentiation , suggesting they may be repurposed to enhance expansion of Tregs for adoptive cellular therapy.

Access and Attitudes Toward Palliative Care Among Movement Disorders Clinicians.

Background: Neuropalliative care is an emerging field for those with neurodegenerative illnesses, but access to neuropalliative care remains limited.

Objective: We sought to determine Movement Disorder Society (MDS) members' attitudes and access to palliative care.

Methods: A quantitative and qualitative survey instrument was developed by the MDS Palliative Care Task Force and e-mailed to all members for completion.

Consensus-Based Recommendations for the Management of Hyperkalemia in the Hemodialysis Setting.

Hyperkalemia (serum K >5.0 mmol/L) is commonly observed among patients receiving maintenance hemodialysis and associated with increased risk of cardiac arrhythmias. Current international guidelines may not reflect the latest evidence on managing hyperkalemia in patients undergoing hemodialysis, and there is a lack of high-quality published studies in this area.

Evidence and Applicability of Stress Cardiovascular Magnetic Resonance in Detecting Coronary Artery Disease: State of the Art.

Cardiovascular magnetic resonance is increasingly used in clinical practice, as it has emerged over the years as an invaluable imaging technique for diagnosis and prognosis, with clear-cut applications in managing patients with both ischemic and non-ischemic heart disease. In this review, we focus on the evidence and clinical application of stress CMR in coronary artery disease from diagnosis to prognosis.

Patient preferences for stratified medicine in psoriasis: a discrete choice experiment.

Background: New technologies have enabled the potential for stratified medicine in psoriasis. It is important to understand patients' preferences to enable the informed introduction of stratified medicine, which is likely to involve a number of individual tests that could be collated into a prescribing algorithm for biological drug selection to be used in clinical practice.

Objectives: To quantify patient preferences for an algorithm-based approach to prescribing biologics ('biologic calculator') in psoriasis.

The role of gut dysbiosis in Parkinson's disease: mechanistic insights and therapeutic options.

Parkinson's disease is a common neurodegenerative disorder in which gastrointestinal symptoms may appear prior to motor symptoms. The gut microbiota of patients with Parkinson's disease shows unique changes, which may be used as early biomarkers of disease. Alterations in the gut microbiota composition may be related to the cause or effect of motor or non-motor symptoms, but the specific pathogenic mechanisms are unclear.

Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran-Results of the phase 1 inhibitor cohort.

Background: Fitusiran, an investigational small interfering RNA therapy, reduces antithrombin production to rebalance hemostasis in people with hemophilia A or B, with or without inhibitors.

Objectives: To evaluate the safety and efficacy of fitusiran treatment for people with moderate/severe hemophilia A or B with inhibitors.

Patients/methods: In this open-label phase 1, part D study, 17 males with hemophilia A or B with inhibitors received three once-monthly subcutaneous injections of fitusiran 50 mg (n = 6) or 80 mg (n = 11); followed for up to 112 days.

A Preliminary Investigation of the Views of People With Parkinson's (With and Without Psychosis) and Caregivers on Participating in Clinical Trials During the Covid-19 Pandemic: An Online Survey.

The coronavirus pandemic is having a profound impact on non-COVID-19 related research, including the delivery of clinical trials for patients with Parkinson's disease. A preliminary investigation to explore the views of Parkinson's disease (PD) patients, with and without experience of psychosis symptoms, and carers on the resumption of clinical research and adaptations to trials in light of COVID-19. An anonymous self-administered online survey was completed by 30 PD patients and six family members/carers via the Parkinson's UK Research Support Network to explore current perceptions on taking part in PD research and how a planned clinical trial for psychosis in PD may be adapted so participants feel safe.

Scaling-up Health-Arts Programmes: the largest study in the world bringing arts-based mental health interventions into a national health service.

The Scaling-up Health-Arts Programme: Implementation and Effectiveness Research (SHAPER) project is the world's largest hybrid study on the impact of the arts on mental health embedded into a national healthcare system. This programme, funded by the Wellcome Trust, aims to study the impact and the scalability of the arts as an intervention for mental health. The programme will be delivered by a team of clinicians, research scientists, charities, artists, patients and healthcare professionals in the UK's National Health Service (NHS) and the community, spanning academia, the NHS and the charity sector.

In chronic plaque psoriasis, roflumilast cream safely increased likelihood of clear or almost clear state at 6 weeks.

Lebwohl MG, Papp KA, Stein Gold L, et al. N Engl J Med. 2020;383:229-39.

Multimodal analysis of gene expression from postmortem brains and blood identifies synaptic vesicle trafficking genes to be associated with Parkinson's disease.

Objective: We aimed to identify key susceptibility gene targets in multiple datasets generated from postmortem brains and blood of Parkinson's disease (PD) patients and healthy controls (HC).

Methods: We performed a multitiered analysis to integrate the gene expression data using multiple-gene chips from 244 human postmortem tissues. We identified hub node genes in the highly PD-related consensus module by constructing protein-protein interaction (PPI) networks.

Type 1 diabetes can present before the age of 6 months and is characterised by autoimmunity and rapid loss of beta cells.

Aims/hypothesis: Diabetes diagnosed at <6 months of age is usually monogenic. However, 10-15% of affected infants do not have a pathogenic variant in one of the 26 known neonatal diabetes genes. We characterised infants diagnosed at <6 months of age without a pathogenic variant to assess whether polygenic type 1 diabetes could arise at early ages.

How "benign" is cutaneous mastocytosis? A Danish registry-based matched cohort study.

Background: There are limited estimates of the incidence rates (IRs) of mastocytosis, and only a few studies have addressed the long-term consequences of living with these diagnoses. Previous reports have shown that systemic mastocytosis is associated with leukemic transformations and an increased risk of death as opposed to cutaneous mastocytosis (CM) and indolent systemic mastocytosis (ISM), which have benign diagnoses with life expectancy rates similar to those of the background population.

Objective: This study aimed to analyze the incidence and mortality of mastocytosis.