Abstract Background Individuals with Friedreich ataxia (FRDA) can find it difficult to access specialized clinical care. To facilitate best practice in delivering healthcare for FRDA, clinical management guidelines (CMGs) were developed in 2014. However, the lack of high-certainty evidence and the inadequacy of accepted metrics to measure health status continues to present challenges in FRDA and other rare diseases. To overcome these challenges, the Grading of Recommendations Assessment and Evaluation (GRADE) framework for rare diseases...

Abstract Background Core outcome sets (COSs) aim to reduce outcome heterogeneity in clinical practice and research by suggesting a minimum number of agreed-upon outcomes in clinical trials. Most COSs in the musculoskeletal field are developed for specific conditions. We propose that there are likely to be common core domains within existing musculoskeletal COSs that may be used as a starting point in the development of future COSs. We aim to identify common core domains from...

Abstract Background Core outcome sets (COSs) aim to reduce outcome heterogeneity in clinical practice and research by suggesting a minimum number of agreed-upon outcomes in clinical trials. Most COSs in the musculoskeletal field are developed for specific conditions. We propose that there are likely to be common core domains within existing musculoskeletal COSs that may be used as a starting point in the development of future COSs. We aim to identify common core domains from...

Supplementary data to Entry of the antipsychotic drug, olanzapine, into the developing rat brain in mono- and

A systematic review was conducted to describe, and evaluate the effectiveness and cost-effectiveness of, intermediary services to support people with a disability to implement individualized funding plans. We included six records, including one subanalysis of randomized trial data, three qualitative studies, and two systematic reviews (reporting on 73 and 18 studies, respectively). No studies directly compared “consumer-directed plan plus intermediary services” to “consumer-directed plan with no/alternative intermediary,” so effectiveness of these interventions is uncertain. There...

Abstract Background Hepatic arterial infusion chemotherapy (HAIC) with oxaliplatin and 5-fluorouracil was effective in unresectable hepatocellular carcinoma (HCC). The program of FOLFOX-HAIC in HCC was performed for 1 day (HAIC 1d) or 2 days (HAIC 2d). We hereby retrospectively compared the efficacy and safety between these two treatment regimens and explored the predictive power of thymidylate synthase (TYMS), an enzyme involved in the DNA synthesis process and metabolism of fluorouracil. Methods This study included patients...

Measuring and correcting wavefront aberrations is an important process in a wide variety of disciplines, from ophthalmology, laser cutting and astronomy to free-space communication and microscopy, and always relies on measuring intensities to infer phase. One approach is to use the transport-of-intensity as a means for phase retrieval, exploiting the connection between observed energy flow in optical fields and their wavefronts. Here we present a simple scheme, using a digital micro-mirror device (DMD), to perform...

Background:Tenecteplase administered to patients with ischaemic stroke in a mobile stroke unit (MSU) has been shown to reduce the perfusion lesion volumes and result in ultra-early recovery. We now seek to assess the cost-effectiveness of tenecteplase in the MSU.Methods:A within-trial (TASTE-A) economic analysis and a model-based long-term cost-effectiveness analysis were performed. This post hoc within-trial economic analysis utilised the patient-level data (intention to treat, ITT) prospectively collected over the trial to calculate the difference in...

Light sheet microscopy combined with microchip is an emerging tool in biomedical research that notably improves efficiency. However, microchip-enhanced light-sheet microscopy is limited by noticeable aberrations induced by the complex refractive indices in the chip. Herein, we report a droplet microchip that is specifically engineered to be capable of large-scale culture of 3D spheroids (over 600 samples per chip) and has a polymer index matched to water (difference <1%). When combined with a lab-built open-top...

The National Transfusion Dataset (NTD) will form the first integrated national database of blood usage in Australia. The NTD aims to collect information about where, when, and how blood products are used across all clinical settings. This will address Australia’s absence of an integrated national database to record blood usage with the ability to link with clinical outcomes. The dataset will be an invaluable resource towards a comprehensive understanding of how and why blood products...

The National Transfusion Dataset (NTD) will form the first integrated national database of blood usage in Australia. The NTD aims to collect information about where, when, and how blood products are used across all clinical settings. This will address Australia’s absence of an integrated national database to record blood usage with the ability to link with clinical outcomes. The dataset will be an invaluable resource towards a comprehensive understanding of how and why blood products...

Young children have rights; they are agents and active constructors of their social worlds. Despite well-established theoretical foundations, the ‘methods’ and ‘ethics’ of qualitative research to elicit young children’s voice require further exploration to ensure young children are central to our research endeavors. This systematic review examined studies that sought to capture young children’s (3–6 years) voice in Early Childhood Education and Care settings. Fifty-eight studies met the inclusion criteria. Interview was found to be...

Young children have rights; they are agents and active constructors of their social worlds. Despite well-established theoretical foundations, the ‘methods’ and ‘ethics’ of qualitative research to elicit young children’s voice require further exploration to ensure young children are central to our research endeavors. This systematic review examined studies that sought to capture young children’s (3–6 years) voice in Early Childhood Education and Care settings. Fifty-eight studies met the inclusion criteria. Interview was found to be...

Objective:The Strengths and Difficulties Questionnaire is a widely used screening tool for emotional and behavioural problems in children. Recent quantitative analyses have raised concerns regarding its structural validity in Aboriginal and Torres Strait Islander communities. This paper aims to extend upon existing findings by analysing the factor structure of both the parent- and teacher-reported Strengths and Difficulties Questionnaire in this population across a broader age range than in previous studies.Methods:Participants were the caregivers and teachers...

Abstract Background Limited evidence is available about the association between tropical cyclones and dengue incidence. This study aimed to examine the effects of tropical cyclones on the incidence of dengue and to explore the vulnerable populations in Guangzhou, China. Methods Weekly dengue case data, tropical cyclone and meteorological data during the tropical cyclones season (June to October) from 2015 to 2019 were collected for the study. A quasi-Poisson generalized linear model combined with a distributed...

Abstract Background Periodontal accelerated osteogenic orthodontics (PAOO) is a widely-used clinical procedure that combines selective alveolar corticotomy, particulate bone grafting, and the application of orthodontic forces. Different modifications of PAOO such as collagen-membrane coverage can better benefit patients from preventing displacement of grafts. Due to its stability, collagen-membrane coverage gradually gained popularity and became a widely-used procedure in traditional PAOO technique. Objectives To quantitatively investigate the radiographic changes of alveolar bone, periodontal soft tissue changes...

Abstract Background Neuropathic pain is still a challenge for clinical treatment as a result of the comprehensive pathogenesis. Although emerging evidence demonstrates the pivotal role of glial cells in regulating neuropathic pain, the role of Schwann cells and their underlying mechanisms still need to be uncovered. Pannexin 1 (Panx 1), an important membrane channel for the release of ATP and inflammatory cytokines, as well as its activation in central glial cells, contributes to pain development....

Abstract Background In mammals, oocytes display compromised quality after experiencing a process of postovulatory aging. However, the mechanisms underlying are not yet fully understood. Here, we portrayed a protein expression profile of fresh and aging metaphase II (MII) mouse oocytes by means of four-dimensional label-free quantification mass spectrometry (4D-LFQ). Results The analysis of 4D-LFQ data illustrated that there were seventy-six differentially expressed proteins (DEPs) between two groups of MII stage oocytes. Fifty-three DEPs were up-regulated...

Abstract Background For the design and analysis of clinical trials with time-to-event outcomes, the Cox proportional hazards model and the logrank test have been the cornerstone methods for many decades. Increasingly, the key assumption of proportionality—or time-fixed effects—that underpins these methods has been called into question. The availability of novel therapies with new mechanisms of action and clinical trials of longer duration mean that non-proportional hazards are now more frequently encountered. Methods We compared several...

The study describes the development and preliminary psychometric validation of the Suicidal Ideation Attributes Scale-Modified (SIDAS-M), a five-item assessment of suicidal ideation for use with autistic adults. Participants (n = 102 autistic adults; 58% women, 34% men, 8% nonbinary; Mage = 41.75, SD = 12.89) completed an online survey including the SIDAS-M at Time 1 and participated in follow-up interviews at Time 2 (~111 days later). A single factor solution provided good to excellent fit...

The evolution of surface damages in laser direct cleaning of nanoparticulate contaminations on silicon was theoretically and experimentally investigated. Nanobumps with a volcano-like shape were found in near infrared laser cleaning of polystyrene latex nanoparticles on Si wafers. According to the finite-difference time-domain simulation and high-resolution surface characterization, unusual particle-induced optical filed enhancement in the vicinity at the interface between Si and nanoparticles is mainly responsible for the generation of volcano-like nanobumps. This work is...

China is a multi-ethnic country, and the prevalence of Helicobacter pylori (H pylori) infection may be diverse among ethnics. This meta-analysis was conducted to compare the prevalence of H pylori infection between Tibetans and Han ethnics. Ten studies that reported the prevalence of H pylori infection between Tibetans and Hans in China were eligible. The pooled prevalence of H pylori infection was 62.2% versus 55.3% among Tibetans and Hans, respectively. Tibetans had a higher risk...

Abstract Background The Accelerating Innovation for Mothers (AIM) project established a database of candidate medicines in research and development (R&D) between 2000 and 2021 for five pregnancy-related conditions, including pre-eclampsia. In parallel, we published target product profiles (TPPs) that describe optimal characteristics of medicines for use in preventing/treating pre-eclampsia. The study objective was to use systematic double screening and extraction to identify all candidate medicines being investigated for pre-eclampsia prevention/treatment and rank their potential based...

Abstract Background Various stakeholders are calling for increased availability of data and code from cancer research. However, it is unclear how commonly these products are shared, and what factors are associated with sharing. Our objective was to evaluate how frequently oncology researchers make data and code available and explore factors associated with sharing. Methods A cross-sectional analysis of a random sample of 306 cancer-related articles indexed in PubMed in 2019 which studied research subjects with...

Abstract Background In 2019, WHO prioritized updating recommendations relating to three labour induction topics: labour induction at or beyond term, mechanical methods for labour induction, and outpatient labour induction. As part of this process, we aimed to review the evidence addressing factors beyond clinical effectiveness (values, human rights and sociocultural acceptability, health equity, and economic and feasibility considerations) to inform WHO Guideline Development Group decision-making using the WHO-INTEGRATE evidence-to-decision framework, and to reflect on how...