Comparability of a provisioned device versus bring your own device for completion of patient-reported outcome measures by participants with chronic obstructive pulmonary disease: quantitative study findings.

Objective: To quantitatively compare equivalence and compliance of patient-reported outcome (PRO) data collected via provisioned device (PD) versus bring your own device (BYOD).

Methods: Participants with stable chronic obstructive pulmonary disease (COPD) completed the EXAcerbations of Chronic Pulmonary Disease Tool (EXACT) daily and COPD Assessment Test™ (CAT) and Patient Global Impression of Severity (PGIS) of COPD weekly on either PD or BYOD for 15 days, then switched device types for 15 days. EXACT was scored using the Evaluating Respiratory Symptoms in COPD (E-RS: COPD) algorithm and equivalence assessed using intraclass correlation coefficients (ICCs) adjusting for cross-over sequence, period, and time.

Present state bias in transition ratings was accurately estimated in simulated and real data.

Objective: Patient-reported transition ratings are supposed to reflect the change between a previous baseline health state and a present follow-up state, but may reflect the present state to a greater extent. This so-called "present state bias" (PSB) potentially threatens the validity of transition ratings. Several criteria have been proposed to assess PSB.

Circumventricular Organ Apelin Receptor Knockdown Decreases Blood Pressure and Sympathetic Drive Responses in the Spontaneously Hypertensive Rat.

The central site(s) mediating the cardiovascular actions of the apelin-apelin receptor (APJ) system remains a major question. We hypothesized that the sensory circumventricular organs (CVOs), interfacing between the circulation and deeper brain structures, are sites where circulating apelin acts as a signal in the central nervous system to decrease blood pressure (BP). We show that APJ gene () expression was elevated in the CVOs of spontaneously hypertensive rats (SHRs) compared to normotensive Wistar Kyoto (WKY) controls, and that there was a greater mean arterial BP (MABP) decrease following microinjection of [Pyr]apelin-13 to the CVOs of SHRs compared to WKY rats.

What is an estimand & how does it relate to quantifying the effect of treatment on patient-reported quality of life outcomes in clinical trials?

Background: Published in 2019, a new addendum to the ICH E9 guideline presents the estimand framework as a systematic approach to ensure alignment among clinical trial objectives, trial execution/conduct, statistical analyses, and interpretation of results. The use of the estimand framework for describing clinical trial objectives has yet to be extensively considered in the context of patient-reported outcomes (PROs). We discuss the application of the estimand framework to PRO objectives when designing clinical trials in the future, with a focus on PRO outcomes in oncology trial settings as our example.

Unlike ROC analysis, a new IRT method identified clinical thresholds unbiased by disease prevalence.

Objective: This study introduces a new method to establish clinical thresholds for multi-item tests, based on item response theory (IRT), as an alternative to receiver operating characteristic (ROC) analysis. The performance of IRT method was examined and compared with the ROC method across multiple simulated data sets and in a real data set.

Study Design And Setting: Simulated data sets (sample size: 1,000) varied in means and variability of the test scores and the prevalence of disease.

Increased apelin receptor gene expression in the subfornical organ of spontaneously hypertensive rats.

The vascular organ of the lamina terminalis, subfornical organ (SFO), and area postrema comprise the sensory circumventricular organs (CVO) which are central structures that lie outside the blood brain barrier and are thought to provide an interface between peripherally circulating signals and the brain through their projections to central autonomic structures. The SFO expresses mRNA for the G protein-coupled apelin receptor (APJ, gene name aplnr) and exogenous microinjection of the neuropeptide apelin (apln) to the SFO elicits a depressor effect. Here we investigated the expression and cellular distribution of aplnr, apln and the recently described ligand apela (apela) in the CVOs and investigated whether differences in the levels of expression of apelinergic gene transcripts in these regions might underlie the chronic elevated blood pressure seen in hypertension.

Exploring content and psychometric validity of newly developed assessment tools for itch and skin pain in atopic dermatitis.

Background: Atopic dermatitis (AD) is a common skin disorder characterized by chronic inflammation, altered skin barrier function, and inflammatory cell skin infiltration that decreases health-related quality of life (HRQoL). The study objective was to understand the patient perspective of AD burden and determine suitable patient-reported outcome (PRO) measures.

Methods: This mixed methods study involved the collection of qualitative and quantitative information from adults (≥ 18 years old) and adolescents (12 - 17 years old) with clinician-confirmed AD regarding their experiences of AD symptoms and its impact on HRQoL.

Blockade of Rostral Ventrolateral Medulla Apelin Receptors Does Not Attenuate Arterial Pressure in SHR and -NAME-Induced Hypertensive Rats.

Dysfunction of the apelinergic system, comprised of the neuropeptide apelin mediating its effects via the G protein-coupled apelin receptor (APJ), may underlie the onset of cardiovascular disease such as hypertension. Apelin expression is increased in the rostral ventrolateral medulla (RVLM) in spontaneously hypertensive rats (SHRs) compared to Wistar-Kyoto (WKY) normotensive rats, however, evidence that the apelinergic system chronically influences mean arterial blood pressure (MABP) under pathophysiological conditions remains to be established. In this study we investigated, in conscious unrestrained rats, whether APJ contributes to MABP and sympathetic vasomotor tone in the progression of two models of hypertension - SHR and -NAME-treated rats - and whether APJ contributes to the development of hypertension in pre-hypertensive SHR.

Expression and functional implications of the renal apelinergic system in rodents.

Apelin binds to the G protein-coupled apelin receptor (APJ; gene name aplnr) to modulate diverse physiological systems including cardiovascular function, and hydromineral and metabolic balance. Recently a second endogenous ligand for APJ, named apela, has been discovered. We confirm that apela activates signal transduction pathways (ERK activation) in cells expressing the cloned rat APJ.

Vasopressin V1a receptors mediate the hypertensive effects of [Pyr ]apelin-13 in the rat rostral ventrolateral medulla.

Key Points: Dysfunctions in CNS regulation of arterial blood pressure lead to an increase in sympathetic nerve activity that participates in the pathogenesis of hypertension. The apelin-apelin receptor system affects arterial blood pressure homeostasis; however, the central mechanisms underlying apelin-mediated changes in sympathetic nerve activity and blood pressure have not been clarified. We explored the mechanisms involved in the regulation of [Pyr ]apelin-13-mediated cardiovascular control within the rostral ventrolateral medulla (RVLM) using selective receptor antagonists.

The effect of coloured overlays and lenses on reading: a systematic review of the literature.

Purpose: There are many anecdotal claims and research reports that coloured lenses and overlays improve reading performance. Here we present the results of a systematic review of this literature and examine the quality of the evidence.

Methods: We systematically reviewed the literature concerning the effect of coloured lenses or overlays on reading performance by searching the PsychInfo, Medline and Embase databases.

Using coloured filters to reduce the symptoms of visual stress in children with reading delay.

The way citation distortion can create unfounded authority in a subject area is exemplified in the paper "Coloured filters to reduce the symptoms of visual stress in children with reading delay", published early online in the Scandinavian Journal of Occupational Therapy. The diagnostic criteria for visual stress remain unclear, for which reason the prevalence figures should be viewed with scepticism. Randomized controlled trials with placebo control groups consistently show improvements in experimental and control lenses.

Roles for learning in mammalian chemosensory responses.

This article is part of a Special Issue "Chemosignals and Reproduction". A rich variety of chemosignals have been identified that influence mammalian behaviour, including peptides, proteins and volatiles. Many of these elicit innate effects acting either as pheromones within species or allelochemicals between species.

Extraocular muscle atrophy and central nervous system involvement in chronic progressive external ophthalmoplegia.

Background: Chronic progressive external ophthalmoplegia (CPEO) is a classical mitochondrial ocular disorder characterised by bilateral progressive ptosis and ophthalmoplegia. These ocular features can develop either in isolation or in association with other prominent neurological deficits (CPEO+). Molecularly, CPEO can be classified into two distinct genetic subgroups depending on whether patients harbour single, large-scale mitochondrial DNA (mtDNA) deletions or multiple mtDNA deletions secondary to a nuclear mutation disrupting mtDNA replication or repair.

Surgery for traumatic optic neuropathy.

Background: Traumatic optic neuropathy (TON) is an important cause of severe visual loss following blunt or penetrating head trauma. Following the initial insult optic nerve swelling within the optic nerve canal or compression by bone fragments are thought to result in secondary retinal ganglion cell loss. Optic nerve decompression with steroids or surgical interventions or both have therefore been advocated to improve visual prognosis in TON.

Steroids for traumatic optic neuropathy.

Background: Traumatic optic neuropathy (TON) is an important cause of severe visual loss following blunt or penetrating head trauma. Following the initial injury, optic nerve swelling within the optic nerve canal can result in secondary retinal ganglion cell loss. Optic nerve decompression with steroids or surgical interventions or both has therefore been advocated as a means of improving visual prognosis in TON.

Raised intraocular pressure as a potential risk factor for visual loss in Leber Hereditary Optic Neuropathy.

Leber Hereditary Optic Neuropathy (LHON) is an important cause of inherited mitochondrial blindness among young adults. The majority of patients carry one of three mitochondrial DNA (mtDNA) point mutations: m.3460G>A, m.

Eyelid surgery in ocular myopathies.

Objective: To retrospectively analyse surgical outcome and complications in patients with ocular myopathy undergoing ptosis correction and to introduce preoperative prophylactic lower lid elevation in this group.

Methods: The medical records of all ocular myopathy patients who had undergone oculoplastic surgery between June 1995 and May 2006 were obtained. Patients' demographics, surgical details and measurements, and complications were recorded.

A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy.

Major advances in understanding the pathogenesis of inherited metabolic disease caused by mitochondrial DNA mutations have yet to translate into treatments of proven efficacy. Leber's hereditary optic neuropathy is the most common mitochondrial DNA disorder causing irreversible blindness in young adult life. Anecdotal reports support the use of idebenone in Leber's hereditary optic neuropathy, but this has not been evaluated in a randomized controlled trial.

Variation in MAPT is not a contributing factor to the incomplete penetrance in LHON.

Leber's hereditary optic neuropathy (LHON) is a common cause of inherited blindness, primarily due to one of three mitochondrial DNA (mtDNA) mutations. LHON, which has an unexplained variable penetrance and pathology, is characterised by disruption of the mitochondrial respiratory chain ultimately resulting in degeneration of the retinal ganglion cells. Phosphorylation of the tau protein is known to cause neurodegeneration and variation in MAPT has been associated with a range of neurodegenerative disorders.