Inherited retinal diseases (IRDs) are a broad range of diseases associated with abnormalities/degeneration of retinal cells. We aimed to identify the top 10 Australian research priorities for IRDs to ultimately facilitate more meaningful and potentially cost-effective research.

We conducted a James Lind Alliance priority setting partnership that involved two Australian-wide surveys and online workshops.

Australia-wide.

Individuals aged 16 years or older were eligible to participate if they had an IRD, were caregivers of an individual with an IRD or were health professionals providing care to this community.

In Survey 1, we gathered participants’ unanswered questions about IRDs. We grouped these into summary questions and undertook a literature review to verify if they were truly unanswered (ie, evidence uncertainties). In Survey 2, participants voted for the uncertainties that they considered a priority. Top-ranked uncertainties progressed for discussion and final prioritisation in two workshops.

In Survey 1, we collected 223 questions from 69 participants. We grouped these into 42 summary questions and confirmed 41 as evidence uncertainties. In Survey 2, 151 participants voted, with the 16 uncertainties progressing to final prioritisation. The top 10 priorities, set by the 24 workshop participants, represented (1) treatment/cure; (2) symptoms and disease progression; (3) psychosocial well-being and (4) health service delivery. The #1 priority was for treatment to prevent, slow down or stop vision loss, followed by the #2 priority to address the psychological impact of having an IRD.

The top 10 research priorities highlight the need for IRD research that takes a whole-person, systems approach. Collaborations to progress priorities will accelerate the translation of research into real-world benefits.

We evaluated the performance of risk models that incorporate ambulatory ECG data and clinical information for prediction of healthcare expenditures related to heart failure (HF) and stroke events in treated and untreated patients.

A retrospective cohort study of Medicare patients who underwent Zio XT ambulatory monitoring in the USA was conducted between 2014 and 2020.

14-day ambulatory ECG data and claims data were evaluated in the study sample which included 89 923 patients in the HF hospitalisation group, 75 870 in the new-onset HF group and 90 159 in the stroke hospitalisation group. Predictive models for new-onset HF, HF hospitalisation and stroke hospitalisation were generated using LASSO Cox regression with ambulatory ECG variables and components of the CHA₂DS₂-VASc. For each outcome, we scored patients using standardised linear predictors from three composite risk models, and we evaluated the association between risk score and total Medicare cost.

The following hazard ratios per one SD increase in the new risk score were observed for the model that included all CHA₂DS₂-VASc components and ECG variables: HF hospitalisation in treated 2.94, 95% CI 2.75 to 3.15; new-onset HF in treated 1.84, 95% CI 1.75 to 1.93; HF hospitalisation in untreated 3.51, 95% CI 3.23 to 3.82; and new-onset HF in untreated 1.92, 95% CI 1.85 to 2.00. Risk scores generated by the model were also predictive of Medicare cost in both treated and untreated patients, with patients in the high-risk category for all outcomes having the greatest Medicare costs during 1 year of follow-up.

Integrating arrhythmia data from ambulatory ECG monitoring into clinical risk models allows for better prediction of healthcare utilisation and cost in both treated and untreated patients at high risk for HF and stroke events.

Combined vascular endothelial growth factor/programmed death-ligand 1 blockade through atezolizumab/bevacizumab (A/B) is the current standard of care in advanced hepatocellular carcinoma (HCC). A/B substantially improved objective response rates compared with tyrosine kinase inhibitor sorafenib; however, a majority of patients will still not respond to A/B. Strong scientific rationale and emerging clinical data suggest that faecal microbiota transfer (FMT) may improve antitumour immune response on PD-(L)1 blockade. Early trials in melanoma with FMT and reinduction of immune checkpoint blockade (ICI) therapy in patients with anti-PD-1-refractory metastatic melanoma were reported in 2021 and demonstrated reinstatement of response to ICI therapy in many patients. Due to anatomical vicinity and the physiological relevance of the gut-liver axis, we hypothesise HCC to be a particularly attractive cancer entity to further assess a potential benefit of FMT in combination with ICI towards increased antitumour immunity. Additionally, HCC often occurs in patients with liver cirrhosis, where liver function is prognostically relevant. There is evidence that FMT may increase hepatic function and therefore could positively affect outcome in this patient population.

This prospective, multicentre, randomised, placebo-controlled, double-blind phase II clinical trial has been designed to assess immunogenicity and safety of FMT via INTESTIFIX 001 combined with A/B in advanced HCC in comparison to A/B with placebo. Primary endpoints are measured as tumour CD8+ T cell infiltration after 2 cycles of treatment with vancomycin, A/B+INTESTIFIX 001 in comparison to vancomycin-placebo, A/B+INTESTIFIX 001-placebo and safety of the therapeutic combination in advanced HCC. INTESTIFIX 001 is an encapsulated FMT preparation by healthy donors with a high alpha-diversity in their gut microbiome for oral administration, manufactured by the Cologne Microbiota Bank (CMB). Sample size was calculated to achieve a specific expected accuracy for the primary immunological endpoint. 48 subjects will be randomised to reach a goal of 42 usable measurements in the modified intention-to-treat set. Subjects will be randomised in a 2:1 ratio to A/B or placebo (28 A/B, 14 placebo).

The study was approved by ethics committee review and the German Federal Ministry of Drugs and Medical Devices. The trial is registered under EU CT no. 2023-506887-15-00. The outcome of the study will be disseminated via peer-reviewed publications and at international conferences.

NCT05690048.

Childhood cancer accounts for a significant proportion of global childhood mortality, especially in low-income and middle-income countries (LMICs). Unlike many adult malignancies, primary prevention of childhood cancers is not possible. Improving survival requires a two-pronged strategy: earlier diagnosis and effective treatment. Our study aims to establish the feasibility, clinical and implementation effectiveness of an adapted early warning signs and symptoms (EWSS) intervention in Cameroon and Kenya. It will equip healthcare workers, Ministry of Health (MOH) representatives and National Cancer Institute leaders with evidence-informed guidance on implementing context-adapted interventions to improve the early detection and referral of childhood cancers in these countries.

The study is a quasi-experimental, hybrid type 2 implementation effectiveness study based on a Ghanaian adaptation of the ‘Saint Siluan’ EWSS campaign. Our protocol proposes context-specific adaptation and evidence-based implementation of the EWSS intervention through iterative engagement with country-level implementation teams to train healthcare workers and improve referral pathways for earlier childhood cancer diagnoses in each study country. Training effectiveness will be measured through pretraining and post-training tests of knowledge and application, as well as training satisfaction surveys. Clinical effectiveness will be assessed by using a REDCap database to track the number of newly diagnosed childhood cancer cases in the study regions and counties, healthcare timelines and paths to diagnosis, and the stage and proportion of metastatic disease at diagnosis. Implementation effectiveness will be evaluated through interviews with senior and mid-level health system partners and clinicians, tracking fidelity to the implementation process as laid out in The Implementation Roadmap Workbook, and analysis of meeting minutes from monthly local implementation team meetings.

This study has received ethical approval from The Hospital for Sick Children (REB # 1000080092) and all participating sites. We have received National Ethical Clearance from the Cameroon Ethical Board (#1699) and Regional Administrative Authorizations from our piloting regions (Centre and West). We have also received ethical clearance from Kenyatta National Hospital (KNH) (ERB# KNH-ERC/RR/955) and our National Commission for Science, Technology and Innovation in Kenya licence from the counties we are piloting in Kenya. As clinical data will be collected from existing health registries and patient charts, patient consent will not be required; however, we will obtain consent from all members of the leadership implementation teams and operational implementation teams for their participation in the implementation meetings and from all individuals participating in the semistructured interviews. We will disseminate findings to build awareness and share findings among various target audiences: (1) key county and regional parties (eg, clinical societies, advocacy groups, country MOHs and regional bodies such as the East African Community, Economic Community of West African States); (2) international bodies such as the WHO; and (3) the academic community.

Urinary tract infections (UTIs) are highly prevalent and pose a significant burden among older adults. Accurate diagnosis in this population is challenging due to the high prevalence of pre-existing lower urinary tract symptoms, inability to express symptoms and asymptomatic bacteriuria. Current diagnostic tests are unreliable, often resulting in over- and underdiagnosis. A previous pilot study proposed a higher cut-off for pyuria and identified five promising biomarkers for the diagnosis of UTIs in older adults. The UTI-GOLD study aims to validate these five new biomarkers and the higher leucocyte cut-off as a diagnostic tool for UTIs in older people in a real-world setting.

Between August 2024 and December 2027, an observational multicentre diagnostic accuracy study is being conducted across primary, secondary and tertiary healthcare facilities in the Netherlands and the UK. Adults ≥65 years with a suspected UTI will be considered eligible. Patients with pre-existing decision-making incapacity or an indwelling catheter will be excluded. UTI will be defined according to an international consensus-based reference standard. Biomarkers will be measured by liquid chromatography-mass spectrometry (neutrophil gelatinase-associated lipocalin, tissue inhibitor of metalloproteinase 2 and CXC motif chemokine ligand 9) and enzyme-linked immunosorbent assay (interleukin 6 and azurocidin). Pyuria will be quantified by automated microscopy and/or flow cytometry. Diagnostic accuracy measures will be calculated using the receiver operating characteristic curves, and sensitivity, specificity, likelihood ratios and predictive values will be reported for optimal cut-offs.

The protocol was reviewed by the local Leiden University Medical Center research committee, who declared on 15 April 2024 that the medical research involving human subject act (Dutch abbreviation: WMO) does not apply to the current study (reference number nWMODIV2_2024025). The study also received approval from the NHS Research Ethics Committee in the UK (reference number 24/LO/0649).

The study findings will be published in a peer-reviewed journal, presented at academic congresses and shared with healthcare providers.

The study was registered at clinicaltrial.gov on the 24 September 2024 with registration number: NCT06610721.

Mental health problems are important causes of disability and economic costs worldwide. Randomised clinical trials examining the treatment of mental health disorders measure heterogeneous outcomes, causing difficulties in data synthesis, interpretation and translation into clinical practice. The aim of the Patient Important Outcomes in Psychiatry (PIO-Psych) Initiative is to develop an overarching, transdiagnostic research-based and consensus-based core outcome set for adult mental health disorders.

The development of the PIO-Psych transdiagnostic core outcome set will include three phases: (1) a systematic scoping review of the literature to develop the initial list of outcomes for the Delphi study; (2) a Delphi study in three rounds including people with lived experience of mental health disorders and their relatives, clinicians, researchers and others (administrators, mental healthcare policymakers, philosophers); (3) a hybrid consensus meeting to agree on the final overarching, transdiagnostic core outcome set and corresponding time points of assessment of each outcome.

Ethical approval is not applicable to this study according to the Research Ethics Committee of the Capital Region of Denmark, as it is not an interventional study. All data will be reported anonymously, and it will not be possible to identify study participants. Results will be disseminated via stakeholder and research networks and peer-reviewed publications.

The PIO-Psych Initiative was pre-registered with COMET (Core Outcome Measures for Effectiveness Trials) on 17 May 2024 (https://www.comet-initiative.org/Studies/Details/3125).

The first 3 months post partum is a critical transition period for women and families, which is characterised by physical, psychological and social adaptation. The first year post partum is also a critical phase for women’s health, given their physical recovery and adjustment to motherhood. The WHO 2023 recommendations highlight the importance of a postnatal care focus, including maternal health assessment, mental health support, nutritional interventions, postpartum contraceptive use and home care visits. This scoping review aims to map the evidence of the effectiveness of postpartum care on women’s health and quality of life in the Southeast Asia region.

This scoping review will be guided by the methodological principles developed by Arksey and O’Malley. This review will also consider observational studies, including cohort studies, case-control studies and cross-sectional studies. For inclusion, studies should be selected for eligibility based on the following criteria: articles investigating the uptake of postpartum women (women who have had childbirth up to 42 days after delivery) in Southeast Asian countries. The search will comprise peer-reviewed articles from 2013 to 2023 from the following electronic databases: PubMed/MEDLINE, ProQuest, EBSCO, Scopus, Web of Science and Google Scholar. We use both keywords in the title and/or abstract and subject headings as appropriate. We will follow the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) Protocols to develop the protocol and use the PRISMA flow diagram to present the results for scoping reviews.

A scoping review is a novel approach for examining the breadth of literature regarding the connection between the effectiveness of postpartum care and women’s health and quality of life and, as a secondary analysis, does not require ethics approval. The results of this review will be submitted for publication in a peer-reviewed journal and presented at relevant conferences.

This study aimed to explore what intervention specificities or attributes newly diagnosed individuals with multiple sclerosis (MS) find important and to explore possible reasons behind their evaluations.

A stepwise approach began with a systematic literature review to identify significant attributes. Patients with MS then assessed these attributes through an online survey, which included a ranking exercise and open-ended questions. Finally, the results were evaluated by the clinical team to select the most relevant factors for personalised care.

From June 2023 to December 2023, all consecutive patients referred to the MS Center of Careggi University Hospital were screened for inclusion. Following recruitment, cognitive and physical assessments were administered at the Don Gnocchi Centre. All participants were interviewed by an experienced neuropsychologist.

Participants were enrolled in the RELIABLE clinical trial, which included a ranking exercise and open-ended question. In the ranking exercise, patients prioritised levels of treatment attributes: treatment effects, methods of intervention, type of monitoring, monitoring, mode and mental support. The open-ended questions addressed the reasons behind the level rankings.

Participants’ rankings revealed the most important levels of each attribute. The highest-ranked method of intervention was disease-modifying treatment, which received 164 points. For mental support, individual psychotherapy was deemed most important with 149 points. Preservation of cognitive function, a key treatment effect, received 144 points. Clinical check-ups were the top type of monitoring with 129 points. Lastly, the hybrid mode of monitoring (half remote/half in-person) was ranked with 77 points. Open-ended responses provided insights into the reasons behind these preferences, emphasising the importance of maintaining mobility, cognitive function and emotional well-being. The clinical team evaluated these findings, confirming that the selected attributes were both clinically relevant and aligned with patient priorities. This evaluation process ensured that the treatment specificities chosen for individualised care were comprehensive and reflective of patient needs.

By identifying and prioritising key treatment attributes, this research highlights the multifaceted nature of MS management and emphasises the importance of aligning treatment options with patient preferences. Addressing these factors through further quantitative preference assessments is essential for preventative MS care, improving patient outcomes and promoting a more patient-centred approach to treatment.

This longitudinal study aimed to document shifts in specialty preferences, career pathways and intended practice locations among medical students following the implementation of structured career initiatives during the 2023–2024 academic year.

A longitudinal observational survey study.

A private, not-for-profit institution, VinUniversity in Hanoi, Vietnam during the 2023–2024 academic year.

All year 2, year 3 and year 4 medical students (n=144 eligible), of whom 105 (73%) completed both baseline and follow-up surveys.

Structured career counselling initiatives introduced at the start of the academic year, including academic mentoring, clinical mentoring, hands-on clinical exposure in year 4 and multiple career counselling activities.

The primary outcome was change in specialty preference over time, measured by students’ self-reported first-choice specialty at baseline and follow-up. Secondary outcomes included shifts in factors influencing career decisions (eg, personal interest, income and family expectations), intended practice location (domestic or international) and preferred career pathways (residency, Specialist Level I, master’s degree or direct workforce entry).

Personal interest remained the strongest influence on specialty choice from baseline to follow-up (mean scores 4.27 vs 4.36 on a 5-point scale). A notable decrease occurred in the importance of income (3.82 to 3.22; p

Noticeable shifts in specialty preferences and career pathways were observed after a series of career initiatives were implemented. Although these trends coincided with the new programmes, further qualitative research is needed to elucidate how and why these career initiatives may have influenced decision-making. Informed by these findings, medical educators can refine interventions to support students’ evolving preferences and ultimately strengthen healthcare workforce distribution.