Relationships between patient and physician global assessments in anti-neutrophil cytoplasmic antibody-associated vasculitis.

Introduction/objectives: Patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV) experience reduced health-related quality of life (HRQoL). Global assessment tools use visual analogue scales to provide patients' and physicians' views on disease activity or the patient's overall health and well-being. The aim of this study was to examine the relationships between patient and physician global assessments (PtGA and PhGa) as they relate to overall health in people with AAV.

Effectiveness and safety of intravenous immunoglobulin for peripheral neuropathy in EGPA patients: a retrospective study.

Eosinophilic granulomatosis with polyangiitis (EGPA) is a chronic inflammatory disease belonging to the spectrum of small-vessel vasculitis associated with the presence of anti-neutrophil cytoplasmic antibodies (ANCAs), also characterized by eosinophilic infiltration of target organs. Peripheral neuropathy (PN) affects about 2/3 of the patients as a presenting symptom and typically represents a vasculitic involvement. A few studies have addressed the role of intravenous immunoglobulin (IVIg) for the treatment of PN in EGPA.

Sacituzumab Govitecan in Patients With Relapsed/Refractory Advanced Head and Neck Squamous Cell Carcinoma: Results From the Phase 2 TROPiCS-03 Basket Study.

Purpose: Treatment options for advanced head and neck squamous cell carcinoma (HNSCC) previously treated with platinum-based chemotherapy and a programmed death-1 (PD-1) inhibitor are limited. Trophoblast cell-surface antigen 2 (Trop-2) is highly expressed in HNSCC. Sacituzumab govitecan (SG) is a Trop-2-directed antibody-drug conjugate approved for patients with certain previously treated solid tumors.

The association between glycaemic variability and sleep quality and quantity in adults with type 1 and type 2 diabetes: A systematic review.

Aims: Individuals with diabetes frequently encounter sleep disturbances, which can detrimentally impact glycaemic management. We reviewed the relationship between sleep outcomes and glycaemic variability in adults with diabetes.

Methods: We systematically searched Medline, EMBASE and Cochrane Library (2002-March 2023) for studies evaluating sleep and glycaemic variability in adults with type 1 and type 2 diabetes.

A systematic review of the efficacy of TYK2 inhibitors in patients with dermatological disease.

This study systematically reviews existing data on the efficacy of Tyrosine Kinase 2 (TYK2) inhibitors in comparison to placebo or standard treatments for therapeutic benefit and improving quality of life in dermatological diseases. Seventeen records representing 13 clinical trials, one matching-adjusted indirect comparison, and one case study were included. Results indicate that Deucravacitinib is superior to placebo, Apremilast and Adalimumab in treating adult patients with moderate-to-severe plaque psoriasis and superior to placebo in the treatment of adults with systemic lupus erythematosus.

Ontology-based integration and querying of heterogeneous rare disease data sources - POLVAS perspective.

The integration of rare disease medical databases belonging to different countries is an important problem, as a large number of observations are required for reliable statistical inference of patient data in order to facilitate clinical research. Such integration of national registry data, which requires harmonization of the heterogeneous data sets into a unified view, is facilitated in the European FAIRVASC project by developing a domain-specific ontology. The FAIRVASC project is dedicated to the rare disease of anti-neutrophil cytoplasmic antibody (ANCA) associated vasculitis (AAV).

Procalcitonin-guided antibiotic treatment in patients with cancer: a patient-level meta-analysis from randomized controlled trials.

Background: Use of serum procalcitonin (PCT), an inflammatory biomarker for bacterial infections, has shown promising results for early stopping antibiotic treatment among patients with respiratory infections and sepsis. There is need for additional data regarding effectiveness and safety of this concept among patients with cancer.

Methods: Individual data of patients with a documented diagnosis of cancer and proven or suspected respiratory infection and/or sepsis were extracted from previous trials where adult patients were randomized to receive antibiotic treatment based on a PCT protocol or usual care (control group).

Real-world rates and risk factors for subsequent treatment with vertebroplasty or balloon kyphoplasty after initial vertebral augmentation: a retrospective cohort study.

Unlabelled: The purpose of this study was to determine the real-world incidence and predictors of additional vertebroplasty or balloon kyphoplasty after initial vertebral augmentation, as a proxy for subsequent symptomatic vertebral fracture. Of patients, 15.5% underwent subsequent vertebral augmentation.

Neutralizing the IL-7Rα limits injury in experimental ANCA-associated glomerulonephritis.

Background And Hypothesis: Increased T cell IL-7Rα signaling is associated with a poorer prognosis in ANCA-associated vasculitis. These studies examined the functional role of IL-7Rα (CD127) in experimental glomerulonephritis mediated by anti-MPO T cell autoimmunity. We hypothesized that T cells would express IL-7Rα in the kidney and that blocking the function of IL-7Rα, without cellular depletion, would be protective.

Epilepsy and Cardiac Arrhythmias: A State-of-the-Art Review.

Epilepsy is an important cause of disability and mortality worldwide. It can be frequently misdiagnosed, and detailed history and relevant investigations are needed to differentiate epilepsy from syncope. Electroencephalogram is a key noninvasive assessment of neurological function, and the diagnostic yield is increased when performed for an extended period in the ambulatory setting with concurrent electrocardiogram and video monitoring.

Antibiotic Treatment for 7 versus 14 Days in Patients with Bloodstream Infections.

Background: Bloodstream infections are associated with substantial morbidity and mortality. Early, appropriate antibiotic therapy is important, but the duration of treatment is uncertain.

Methods: In a multicenter, noninferiority trial, we randomly assigned hospitalized patients (including patients in the intensive care unit [ICU]) who had bloodstream infection to receive antibiotic treatment for 7 days or 14 days.

Tailoring metabolic activity assays for tumour-engineered 3D models.

Monitoring cell behaviour in hydrogel-based 3D models is critical for assessing their growth and response to cytotoxic treatment. Resazurin-based PrestoBlue and AlamarBlue reagents are frequently used metabolic activity assays when determining cell responses. However, both assays are largely applied to cell monolayer cultures but yet to have a defined protocol for use in hydrogel-based 3D models.

Evaluation of Myocarditis in Patients With Still Disease: Clinical Findings From the Multicenter International AIDA Network Still Disease Registry.

Objective: We aimed to (1) evaluate the cardiac involvement, with a focus on myocarditis, in patients with Still disease included in the multicenter Autoinflammatory Disease Alliance (AIDA) Network Still disease registry; and (2) assess the predictive factors for myocarditis by deriving a clinical risk patient profile for this severe manifestation.

Methods: A multicenter observational study was established, in which consecutive patients with Still disease in the AIDA Network Still disease registry were characterized by cardiac involvement. Cardiac involvement was defined according to the presence of pericarditis, tamponade, myocarditis, and/or aseptic endocarditis.

Leader cells promote immunosuppression to drive ovarian cancer progression in vivo.

Over 75% of patients with ovarian cancer present with late-stage disease, often accompanied by extensive metastasis. The metastatic cascade is driven by a sub-population of transcriptionally plastic cells known as "leader cells" (LCs), which play a critical role in collective invasion yet remain poorly understood. LCs are marked by the expression of keratin-14 (KRT14), which determines their migratory and invasive capacity in ovarian cancer.

Managing Urticarial Vasculitis: A Clinical Decision-Making Algorithm Based on Expert Consensus.

Urticarial vasculitis (UV) is a rare and difficult-to-treat, small-vessel leukocytoclastic vasculitis presenting with recurrent long-lasting wheals. So far, no guidelines and treatment algorithms exist that could help clinicians with the management of UV. In this review, we describe evidence on systemic treatments used for UV and propose a clinical decision-making algorithm for UV management based on the Urticarial Vasculitis Activity Score assessed for 7 days (UVAS7).

Goal-Directed Rehabilitation Versus Standard Care for Individuals with Hereditary Cerebellar Ataxia: A Multicenter, Single-Blind, Randomized Controlled Superiority Trial.

Objective: Rehabilitation is thought to reduce ataxia severity in individuals with hereditary cerebellar ataxia (HCA). This multicenter, randomized controlled superiority trial aimed to examine the efficacy of a 30-week goal-directed rehabilitation program compared with 30 weeks of standard care on function, ataxia, health-related quality of life, and balance in individuals with an HCA.

Methods: Individuals with an autosomal dominant or recessive ataxia (aged ≥15 years) were enrolled at 5 sites in Australia.

From 'strong recommendation' to practice: A pre-test post-test study examining adherence to stroke guidelines for fever, hyperglycaemia, and swallowing (FeSS) management post-stroke.

Background: The Quality in Acute Stroke Care (QASC) Trial demonstrated that assistance to implement protocols to manage Fever, hyperglycaemia (Sugar) and Swallowing (FeSS) post-stroke reduced death and disability. In 2017, a 'Strong Recommendation' for use of FeSS Protocols was included in the Australian Clinical Guidelines for Stroke Management. We aimed to: i) compare adherence to FeSS Protocols pre- and post-guideline inclusion; ii) determine if adherence varied with prior participation in a treatment arm of a FeSS Intervention study, or receiving treatment in a stroke unit; and compare findings with our previous studies.