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PURPOSE: The aim of this randomized, placebo-controlled, two-stage, phase II/III trial was to determine the efficacy of an oral cannabis extract in adults with refractory nausea and/or vomiting during moderately or highly emetogenic, intravenous chemotherapy despite guideline-consistent antiemetic prophylaxis. Here, we report results of the prespecified combined analysis including the initial phase II and subsequent phase III components. PATIENTS AND METHODS: Study treatment consisted of oral capsules containing either tetrahydrocannabinol 2.5 mg plus cannabidiol 2.5 mg capsules (THC:CBD) or matching placebo, taken three times a day from days -1 to 5, in addition to guideline-consistent antiemetics. The primary measure of effect was the difference in the proportions of participants with no vomiting or retching and no use of rescue medications (a complete response) during hours 0-120 after the first cycle of chemotherapy on study (cycle A). RESULTS: We recruited 147 evaluable of a planned 250 participants from 2016 to 2022. Background antiemetic prophylaxis included a corticosteroid and 5-hydroxytryptamine antagonist in 97%, a neurokinin-1 antagonist in 80%, and olanzapine in 10%. THC:CBD compared with placebo improved the complete response rate from 8% to 24% (absolute difference 16%, 95% CI, 4 to 28, P = .01), with similar effects for absence of significant nausea, use of rescue medications, daily vomits, and the nausea scale on the Functional Living Index-Emesis quality-of-life questionnaire. More frequent bothersome adverse events of special interest included sedation (18% v 7%), dizziness (10% v 0%), and transient anxiety (4% v 1%). There were no serious adverse events attributed to THC:CBD. CONCLUSION: THC:CBD is an effective adjunct for chemotherapy-induced nausea and vomiting despite standard antiemetic prophylaxis, but was associated with additional adverse events. Drug availability, cultural attitudes, legal status, and preferences may affect implementation. Future analyses will evaluate the cost-effectiveness of THC:CBD.
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OBJECTIVES: This study aims to evaluate the efficacy and cost-effectiveness of sonothrombolysis delivered pre and post primary percutaneous coronary intervention (pPCI) on infarct size assessed by cardiac MRI, in patients presenting with STEMI, when compared against sham procedure. BACKGROUND: More than a half of patients with successful pPCI have significant microvascular obstruction and residual infarction. Sonothrombolysis is a therapeutic use of ultrasound with contrast enhancement that may improve microcirculation and infarct size. The benefits and real time physiological effects of sonothrombolysis in a multicentre setting are unclear. METHODS: The REDUCE (Restoring microvascular circulation with diagnostic ultrasound and contrast agent) trial is a prospective, multicentre, patient and outcome blinded, sham-controlled trial. Patients presenting with STEMI will be randomized to one of 2 treatment arms, to receive either sonothrombolysis treatment or sham echocardiography before and after pPCI. This tailored design is based on preliminary pilot data from our centre, showing that sonothrombolysis can be safely delivered, without prolonging door to balloon time. Our primary endpoint will be infarct size assessed on day 4±2 on Cardiac Magnetic Resonance (CMR). Patients will be followed up for 6 months post pPCI to assess secondary endpoints. Sample size calculations indicate we will need 150 patients recruited in total. CONCLUSIONS: This multicentre trial will test whether sonothrombolysis delivered pre and post primary PCI can improve patient outcomes and is cost-effective, when compared with sham ultrasound delivered with primary PCI. The results from this trial may provide evidence for the utilization of sonothrombolysis as an adjunct therapy to pPCI to improve cardiovascular outcomes in STEMI. ANZ Clinical Trial Registration number: ACTRN 12620000807954.
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Meios de Contraste , Microcirculação , Intervenção Coronária Percutânea , Infarto do Miocárdio com Supradesnível do Segmento ST , Humanos , Intervenção Coronária Percutânea/métodos , Microcirculação/fisiologia , Infarto do Miocárdio com Supradesnível do Segmento ST/terapia , Infarto do Miocárdio com Supradesnível do Segmento ST/diagnóstico por imagem , Estudos Prospectivos , Terapia por Ultrassom/métodos , Circulação Coronária/fisiologia , Masculino , Feminino , Ecocardiografia/métodos , Análise Custo-BenefícioRESUMO
BACKGROUND: The disparity between the demand for and supply of kidney transplants has resulted in prolonged waiting times for patients with kidney failure. A potential approach to address this shortage is to consider kidneys from donors with a history of common cancers, such as breast, prostate, and colorectal cancers. METHODS: We used a patient-level Markov model to evaluate the outcomes of accepting kidneys from deceased donors with a perceived history of breast, prostate, or colorectal cancer characterized by minimal to intermediate transmission risk. Data from the Australian transplant registry were used in this analysis. The study compared the costs and quality-adjusted life years (QALYs) from the perspective of the Australian healthcare system between the proposed practice of accepting these donors and the conservative practice of declining them. The model simulated outcomes for 1500 individuals waitlisted for a deceased donor kidney transplant for a 25-y horizon. RESULTS: Under the proposed practice, when an additional 15 donors with minimal to intermediate cancer transmission risk were accepted, QALY gains ranged from 7.32 to 20.12. This translates to an approximate increase of 7 to 20 additional years of perfect health. The shift in practice also led to substantial cost savings, ranging between $1.06 and $2.3 million. CONCLUSIONS: The proposed practice of accepting kidneys from deceased donors with a history of common cancers with minimal to intermediate transmission risk offers a promising solution to bridge the gap between demand and supply. This approach likely results in QALY gains for recipients and significant cost savings for the health system.
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Análise Custo-Benefício , Transplante de Rim , Cadeias de Markov , Anos de Vida Ajustados por Qualidade de Vida , Doadores de Tecidos , Humanos , Transplante de Rim/economia , Masculino , Feminino , Doadores de Tecidos/provisão & distribuição , Austrália , Pessoa de Meia-Idade , Neoplasias da Mama/cirurgia , Neoplasias da Mama/economia , Neoplasias da Próstata/cirurgia , Neoplasias da Próstata/economia , Neoplasias Colorretais/cirurgia , Neoplasias Colorretais/economia , Adulto , Sistema de Registros , Seleção do Doador/economia , Fatores de Risco , Listas de Espera , Modelos Econômicos , Fatores de TempoRESUMO
OBJECTIVES: The environmental impacts of healthcare are important factors that should be considered during health technology assessments. This study aims to summarize the evidence that exists about methods to include environmental impacts in health economic evaluations and health technology assessments. METHODS: We identified records for screening using an existing scoping review and a systematic search of academic databases and gray literature up to September 2023. We screened the identified records for eligibility and extracted data using a narrative synthesis approach. The review was conducted following the JBI Manual for Evidence Synthesis and reported according to the Preferred Reporting Items for Systematic Reviews and Meta Analyses Extension for Scoping Reviews checklist. RESULTS: We identified 2898 records and assessed the full text of 114, of which 54 were included in this review. Ten methods were identified to include environmental impacts in health economic evaluations and health technology assessments. Methods included converting environmental impacts to dollars or disability-adjusted life years and including them in a cost-effectiveness, cost-utility, or cost-benefit analysis, calculating an incremental carbon footprint effectiveness ratio or incremental carbon footprint cost ratio, incorporating impacts as one criteria of a multi-criteria decision analysis, and freely considering impacts during health technology assessment deliberation processes. CONCLUSIONS: Methods to include environmental impacts in health economic evaluations and health technology assessments exist but have not been tested for widespread use by health technology assessment agencies. Further research and implementation work is needed to determine which method can best aid decision makers to choose low environmental impact healthcare interventions.
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Análise Custo-Benefício , Meio Ambiente , Avaliação da Tecnologia Biomédica , Avaliação da Tecnologia Biomédica/economia , Humanos , Análise Custo-Benefício/métodos , Pegada de Carbono/economia , Anos de Vida Ajustados por Qualidade de VidaRESUMO
BACKGROUND/AIMS: The demand for simplified data collection within trials to increase efficiency and reduce costs has led to broader interest in repurposing routinely collected administrative data for use in clinical trials research. The aim of this scoping review is to describe how and why administrative data have been used in Australian randomised controlled trial conduct and analyses, specifically the advantages and limitations of their use as well as barriers and enablers to accessing administrative data for use alongside randomised controlled trials. METHODS: Databases were searched to November 2022. Randomised controlled trials were included if they accessed one or more Australian administrative data sets, where some or all trial participants were enrolled in Australia, and where the article was published between January 2000 and November 2022. Titles and abstracts were independently screened by two reviewers, and the full texts of selected studies were assessed against the eligibility criteria by two independent reviewers. Data were extracted from included articles by two reviewers using a data extraction tool. RESULTS: Forty-one articles from 36 randomised controlled trials were included. Trial characteristics, including the sample size, disease area, population, and intervention, were varied; however, randomised controlled trials most commonly linked to government reimbursed claims data sets, hospital admissions data sets and birth/death registries, and the most common reason for linkage was to ascertain disease outcomes or survival status, and to track health service use. The majority of randomised controlled trials were able to achieve linkage in over 90% of trial participants; however, consent and participant withdrawals were common limitations to participant linkage. Reported advantages were the reliability and accuracy of the data, the ease of long term follow-up, and the use of established data linkage units. Common reported limitations were locating participants who had moved outside the jurisdictional area, missing data where consent was not provided, and unavailability of certain healthcare data. CONCLUSIONS: As linked administrative data are not intended for research purposes, detailed knowledge of the data sets is required by researchers, and the time delay in receiving the data is viewed as a barrier to its use. The lack of access to primary care data sets is viewed as a barrier to administrative data use; however, work to expand the number of healthcare data sets that can be linked has made it easier for researchers to access and use these data, which may have implications on how randomised controlled trials will be run in future.
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Ensaios Clínicos Controlados Aleatórios como Assunto , Humanos , Ensaios Clínicos Controlados Aleatórios como Assunto/métodos , Ensaios Clínicos Controlados Aleatórios como Assunto/estatística & dados numéricos , Austrália , Bases de Dados Factuais , Coleta de Dados/métodosRESUMO
OBJECTIVES: The EQ-5D-5L is a commonly used health-related quality of life instrument for evaluating interventions in patients receiving dialysis; however, the minimal important difference (MID) that constitutes a meaningful treatment effect for this population has not been established. This study aims to estimate the MID for the EQ-5D-5L utility index in dialysis patients. METHODS: 6-monthly EQ-5D-5L measurements were collected from adult dialysis patients between April 2017 and November 2020 at a renal network in Sydney, Australia. EQ-VAS and Integrated Palliative care Outcome Scale Renal symptom burden scores were collected simultaneously and used as anchors. MID estimates for the EQ-5D-5L utility index were derived using anchor-based and distribution-based methods. RESULTS: A total of 352 patients with ≥1 EQ-5D-5L observation were included, constituting 1127 observations. Mean EQ-5D-5L utility index at baseline was 0.719 (SD ± 0.267), and mean EQ-5D-5L utility decreased over time by -0.017 per year (95% CI -0.029 to -0.006, P = .004). Using cross-sectional anchor-based methods, MID estimates ranged from 0.073 to 0.107. Using longitudinal anchor-based methods, MID for improvement and deterioration ranged from 0.046 to 0.079 and -0.111 to -0.048, respectively. Using receiver operating characteristic curves, MID for improvement and deterioration ranged from 0.037 to 0.122 and -0.074 to -0.063, respectively. MID estimates from distribution-based methods were consistent with anchor-based estimates. CONCLUSIONS: Anchor-based and distribution-based approaches provided EQ-5D-5L utility index MID estimates ranging from 0.034 to 0.134. These estimates can inform the target difference or "effect size" for clinical trial design among dialysis populations.
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Qualidade de Vida , Diálise Renal , Adulto , Humanos , Estudos Transversais , Inquéritos e Questionários , PsicometriaRESUMO
BACKGROUND: Patient safety is threatened when early signs of clinical deterioration are missed or not acted upon. This research began as a clinical-academic partnership established around a shared concern of nursing physical assessment practices on general wards and delayed recognition of clinical deterioration. The outcome was the development of a complex intervention facilitated at the ward level for proactive nursing surveillance. METHODS: The evidence-based nursing core assessment (ENCORE) trial was a pragmatic cluster-randomised controlled trial. We hypothesised that ward intervention would reduce the incidence of patient rescue events (medical emergency team activations) and serious adverse events. We randomised 29 general wards in a 1:2 allocation, across 5 Australian hospitals to intervention (nâ¯=â¯10) and usual care wards (nâ¯=â¯19). Skilled facilitation over 12â¯months enabled practitioner-led, ward-level practice change for proactive nursing surveillance. The primary outcome was the rate of medical emergency team activations and secondary outcomes were unplanned intensive care unit admissions, on-ward resuscitations, and unexpected deaths. Outcomes were prospectively collected for 6â¯months following the initial 6â¯months of implementation. Analysis was at the patient level using generalised linear mixed models to account for clustering by ward. RESULTS: We analysed 29,385 patient admissions to intervention (nâ¯=â¯11,792) and control (nâ¯=â¯17,593) wards. Adjusted models for overall effects suggested the intervention increased the rate of medical emergency team activations (adjusted incidence rate ratio 1.314; 95â¯% confidence interval 0.975, 1.773), although the confidence interval was compatible with a marginal decrease to a substantial increase in rate. Confidence intervals for secondary outcomes included a range of plausible effects from benefit to harm. However, considerable heterogeneity was observed in intervention effects by patient comorbidity. Among patients with few comorbid conditions in the intervention arm there was a lower medical emergency team activation rate and decreased odds of unexpected death. Among patients with multimorbidity in the intervention arm there were higher rates of medical emergency team activation and intensive care unit admissions. CONCLUSION: Trial outcomes have refined our assumptions about the impact of the ENCORE intervention. The intervention appears to have protective effects for patients with low complexity where frontline teams can respond locally. It also appears to have redistributed medical emergency team activations and unplanned intensive care unit admissions, mobilising higher rates of rescue for patients with multimorbidity. TRIAL REGISTRATION NUMBER: ACTRN12618001903279 (Date of registration: 22/11/2018; First participant recruited: 01/02/2019).
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Deterioração Clínica , Humanos , Austrália , Enfermagem Baseada em Evidências , Hospitalização , HospitaisRESUMO
Background: Care gaps remain in modern health care despite the availability of robust, evidence-based medications. Although sodium-glucose cotransporter-2 (SGLT2) inhibitors have demonstrated profound benefits in improving both cardiovascular and kidney outcomes in patients, the uptake of these medications remain suboptimal, and the causes have not been systematically explored. Objective: The purpose of this study was to use the Consolidated Framework for Implementation Research (CFIR) to describe the barriers and facilitators faced by clinicians in British Columbia, Canada, when prescribing an SGLT2 inhibitor. To achieve this, we conducted semistructured interviews using the CFIR with practicing family physicians, nephrologists, endocrinologists, and cardiologists in British Columbia. Design: Semistructured interviews. Setting: British Columbia, Canada. Participants: Actively practicing family physicians, nephrologists, endocrinologists, and cardiologists in British Columbia. Methods: Twenty-one clinicians were interviewed using questions derived from the CFIR. The audio recordings were transcribed verbatim, and each transcription was individually analyzed in duplicate using thematic analysis. The analysis focused on identifying barriers and facilitators to using SGLT2 inhibitors in clinical practice and coded using the CFIR constructs. Once the transcriptions were coded, overarching themes were created. Results: Five overarching themes were identified to the barriers and facilitators to using SGLT2 inhibitors: current perceptions and beliefs, clinician factors, patient factors, medication factors, and health care system factors. The current perceptions and beliefs were that SGLT2 inhibitors are efficacious and have distinct advantages over other agents but are underutilized in British Columbia. Clinician factors included varying levels of knowledge of and comfort in prescribing SGLT2 inhibitors, and patient factors included intolerable adverse events and additional pill burden, but many were enthusiastic about potential benefits. Multiple SGLT2 inhibitor related adverse events like mycotic infections and euglycemic diabetic ketoacidosis and the difficulty in obtaining reimbursement for these medications were also identified as a barrier to prescribing these medications. Facilitators for the use of SGLT2 inhibitors included consensus among colleagues, influential leaders, and peers in support of their use, and endorsement by national guidelines. Limitations: The experience from the clinicians regarding costs and the reimbursement process is limited to British Columbia as each province has its own procedures. There may be responder bias as clinicians were approached through purposive sampling. Conclusion: This study highlights different themes to the barriers and facilitators of using SGLT2 inhibitors in British Columbia. The identification of these barriers provides a specific target for improvement, and the facilitators can be leveraged for the increased use of SGLT2 inhibitors. Efforts to address and optimize these barriers and facilitators in a systematic approach may lead to an increase in the use of these efficacious medications.
Contexte: Des lacunes subsistent dans les soins de santé modernes, malgré l'existence de médicaments éprouvés et fondés sur des données probantes. Les inhibiteurs du cotransporteur de sodium-glucose de type 2 (SGLT2) ont démontré d'importants effets dans l'amélioration des résultats cardiovasculaires et rénaux des patients, mais l'utilization de ces médicaments demeure sous-optimale et les raisons qui expliquent cette situation n'ont pas été systématiquement explorées. Objectif: Utiliser le Consolidated Framework for Implementation Research (CFIR) pour décrire les obstacles et les éléments facilitateurs rencontrés par les cliniciens de la Colombie-Britannique (Canada) lorsqu'ils prescrivent un inhibiteur du SGLT2. Pour ce faire, nous avons mené des entretiens semi-structurés au moyen du CFIR auprès de médecins de famille, de néphrologues, de cardiologues et d'endocrinologues exerçant en Colombie-Britannique. Conception: Entretiens semi-structurés. Cadre: Colombie-Britannique (Canada). Participants: Médecins de famille, cardiologues, endocrinologues et néphrologues exerçant en Colombie-Britannique. Méthodologie: Les questions dérivées du CFIR ont été posées à vingt-et-un cliniciens. Les enregistrements audio ont été transcrits verbatim et chaque transcription a été analysée individuellement en double en utilisant l'analyze thématique. L'analyze s'est concentrée sur l'identification des obstacles et des facilitateurs à l'utilization des inhibiteurs du SGLT2 dans la pratique clinique et sur le codage selon les concepts du CFIR. Une fois les transcriptions codées, des thèmes généraux ont été créés. Résultats: Cinq thèmes généraux ont été identifiés pour les obstacles et les facilitateurs à l'utilization des inhibiteurs du SGLT2: les perceptions et les croyances actuelles, les facteurs liés aux cliniciens, les facteurs liés aux patients, les facteurs liés aux médicaments et les facteurs liés au système de santé. Les perceptions et croyances actuelles étaient que les inhibiteurs du SGLT2 sont efficaces, qu'ils présentent des avantages distincts des autres agents, mais qu'ils sont sous-utilisés en Colombie-Britannique. Les facteurs liés aux cliniciens incluaient des niveaux variables de connaissance et de confort vis-à-vis la prescription d'inhibiteurs du SGLT2. Les facteurs liés aux patients incluaient les événements indésirables intolérables et la charge médicamenteuse supplémentaire, mais plusieurs répondants voyaient positivement les bienfaits potentiels. Les nombreux événements indésirables liés aux inhibiteurs du SGLT2, notamment les infections mycosiques et l'acidocétose diabétique euglycémique, et la difficulté à obtenir le remboursement de ces médicaments ont également été cités comme raisons limitant la prescription de ces médicaments. Le consensus parmi les collègues, les leaders influents et les pairs en faveur des inhibiteurs du SGLT2 et l'inclusion de ces médicaments dans les lignes directrices nationales figuraient parmi les facilitateurs. Limites: Les expériences rapportées par les cliniciens en ce qui concerne les coûts et le processus de remboursement se limitent à la Colombie-Britannique, car chaque province a ses propres procédures. L'étude comporte un possible biais de réponse puisque les cliniciens ont été approchés par échantillonnage dirigé. Conclusion: Cette étude met en évidence différents thèmes concernant les obstacles et les facilitateurs à l'utilization des inhibiteurs du SGLT2 en Colombie-Britannique. L'identification de ces obstacles fournit une cible précise pour l'amélioration, alors que les facilitateurs peuvent être mis à profit pour accroître l'utilization des inhibiteurs de SGLT2. Les efforts déployés pour aborder et optimiser ces obstacles et ces facilitateurs dans le cadre d'une approche systématique pourraient mener à une augmentation de l'utilization de ces médicaments efficaces.
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OBJECTIVE: To compare cost-effectiveness of oral sildenafil citrate, administered after onset of labor, with standard care to health system funders in the UK and Australia. METHODS: We conducted a modeled cost-effectiveness analysis, measuring costs and quality adjusted life years (QALYs), using a decision-analytic model covering onset of labor to 1 month post-birth. The relative risk of emergency cesarean section and operative vaginal birth was taken from a Phase 2 placebo controlled double blinded randomized control trial. RESULTS: Both options of care resulted in the same QALYs gained over the model time period (0.08). Sildenafil citrate was cost-saving compared with standard care, saving £92 per birth in the UK (AU$303 per birth in Australia). Sensitivity analyses did not identify any areas of uncertainty that stopped sildenafil citrate being cost saving compared with standard care. Threshold analysis revealed that sildenafil citrate would be cost saving up to a per birth drug or administration cost of £152.32 in the UK (AU$333.61 in Australia). CONCLUSION: Oral sildenafil citrate may be cost saving compared with standard care; however, the effects on neonatal outcomes still need to be demonstrated in large randomized trials.
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Cesárea , Análise de Custo-Efetividade , Feminino , Humanos , Recém-Nascido , Gravidez , Análise Custo-Benefício , Cuidado Pré-Natal , Citrato de Sildenafila/uso terapêutico , Reino Unido , Ensaios Clínicos Fase II como Assunto , Ensaios Clínicos Controlados Aleatórios como Assunto , Método Duplo-CegoRESUMO
The VALID BP project was initiated to increase the availability of validated blood pressure measuring devices (BPMDs). The goal is to eliminate non validated BPMDs and minimise over- and underdiagnosis of hypertension caused by inaccurate readings. This study was undertaken to assess the potential return on investment in the VALID BP project. The Framework to Assess the Impact of Translational Health Research was applied to the VALID BP project. This paper focuses on the implementation of the cost benefit analysis aspect of this framework to monetise past research investment and model future research costs, implementation costs, and benefits. Analysis was based on reasoned assumptions about potential impacts from availability and use of validated BPMDs (assuming an end goal of 100% validated BPMDs available in Australia by 2028) and improved skills leading to more accurate BP measurement. After 5 years, with 20% attribution of benefits, there is a potential $1.14-$1.30 return for every dollar spent if the proportion of validated BPMDs and staff trained in proper BP measurement technique increased from 20% to 60%. After eight years (2020-2028) and assuming universal validation and training coverage, the returns would be between $2.70 and $3.20 per dollar spent (not including cost of side effects of unnecessary medication or downstream patient impacts from unmanaged hypertension). This modelled economic analysis indicates there will be positive downstream economic benefits if the availability of validated BPMDs is increased. The findings support ongoing efforts toward a universal regulatory framework for BPMDs and can be considered within more detailed future economic analyses.
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Determinação da Pressão Arterial , Hipertensão , Humanos , Pressão Sanguínea/fisiologia , Esfigmomanômetros , AustráliaRESUMO
ASPiRATION is a national prospective observational cohort study assessing the feasibility, clinical and economic value of up-front tissue-based comprehensive genomic profiling (CGP) to identify actionable genomic alterations in participants with newly diagnosed metastatic non-squamous non-small-cell lung cancer in Australia. This study will enrol 1000 participants with tumor available for CGP and standard of care molecular testing (EGFR/ALK/ROS1). Participants with actionable variants may receive novel targeted treatments through ASPiRATION-specific substudies, other trials/programs. Clinical outcome data will be collected for a minimum of 2 years. Study outcomes are descriptive, including the ability of CGP to identify additional actionable variants, leading to personalized treatment recommendations, and will describe the feasibility, efficiency, cost and utility of implementation of CGP nationally.
Lung cancer is the most common cause of cancer death in Australia and worldwide. This disease often happens due to alterations in specific genes that allow cancer cells to develop and spread. Scientists have designed targeted drugs that are better at attacking cancer cells that have specific 'actionable' gene alterations and have less effect on other cells in the body. The result is often more benefit from treatment and fewer side effects than other standard treatments (chemotherapy or immunotherapy). The targeted drugs are well established as the best initial treatments for some gene alterations, but more research is needed to know if this is true for some of the less common or recently identified gene alterations, and where the targeted drugs are very new. Comprehensive genomic profiling is a new way of testing lung cancer cells for all the gene alterations (the well-known ones as well as the rare ones) in a single test. It is expected that this test will find many more of these gene alterations, which will allow more people to have safer and more effective targeted treatments leading to potentially better outcomes, and will allow some people to join clinical trials testing newer targeted treatments. The ASPiRATION study will help work out whether comprehensive genomic profiling is better than the current way of testing for gene alterations in Australia, and if it is feasible to use in all people diagnosed with advanced lung cancer in Australia. Clinical Trial Registration: ACTRN12621000221853 (ANZCTR).
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Carcinoma Pulmonar de Células não Pequenas , Neoplasias Pulmonares , Humanos , Neoplasias Pulmonares/patologia , Carcinoma Pulmonar de Células não Pequenas/tratamento farmacológico , Estudos Prospectivos , Proteínas Tirosina Quinases/genética , Mutação , Austrália , Proteínas Proto-Oncogênicas/genética , Genômica , Estudos Observacionais como AssuntoRESUMO
BACKGROUND: Refractory Out of Hospital Cardiac Arrest (r-OHCA) is common and the benefit versus harm of intra-arrest transport of patients to hospital is not clear. OBJECTIVE: To assess the rate of survival to hospital discharge in adult patients with r-OHCA, initial rhythm pulseless ventricular tachycardia (VT)/ventricular fibrillation (VF) or Pulseless Electrical Activity (PEA) treated with 1 of 2 locally accepted standards of care:1 expedited transport from scene; or2 ongoing advanced life support (ALS) resuscitation on-scene. HYPOTHESIS: We hypothesize that expedited transport from scene in r-OHCA improves survival with favorable neurological status/outcome. METHODS/DESIGN: Phase III, multi-center, partially blinded, prospective, intention-to-treat, safety and efficacy clinical trial with contemporaneous registry of patient ineligible for the clinical trial. Eligible patients for inclusion are adults with witnessed r-OHCA; estimated age 18 to 70, assumed medical cause with immediate bystander cardiopulmonary resuscitation (CPR); initial rhythm of VF/pulseless VT, or PEA; no return of spontaneous circulation following 3 shocks and/or 15 minutes of professional on-scene resuscitation; with mechanical CPR available. Two hundred patients will be randomized in a 1:1 ratio to either expedited transport from scene or ongoing ALS at the scene of cardiac arrest. SETTING: Two urban regions in NSW Australia. OUTCOMES: Primary: survival to hospital discharge with cerebral performance category (CPC) 1 or 2. Secondary: safety, survival, prognostic factors, use of ECMO supported CPR and functional assessment at hospital discharge and 4 weeks and 6 months, quality of life, healthcare use and cost-effectiveness. CONCLUSIONS: The EVIDENCE trial will determine the potential risks and benefits of an expedited transport from scene of cardiac arrest.
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Reanimação Cardiopulmonar , Serviços Médicos de Emergência , Parada Cardíaca Extra-Hospitalar , Taquicardia Ventricular , Adolescente , Adulto , Idoso , Humanos , Pessoa de Meia-Idade , Adulto Jovem , Parada Cardíaca Extra-Hospitalar/terapia , Estudos Prospectivos , Qualidade de VidaRESUMO
We determined weights for a multi-criteria tool for assessing the relative merits of clinical-trial research proposals, and investigated whether the weights vary across relevant stakeholder groups. A cross-sectional, adaptive discrete choice experiment using 1000minds online software was administered to consumers, researchers and funders affiliated with the Australian Clinical Trials Alliance (ACTA). We identified weights for four criteria-Appropriateness, Significance, Relevance, Feasibility-and their levels, representing their relative importance, so that research proposals can be scored between 0% (nil or very low merit) and 100% (very high merit). From 220 complete survey responses, the most important criterion was Appropriateness (adjusted for differences between stakeholder groups, mean weight 28.9%) and the least important was Feasibility (adjusted mean weight 19.5%). Consumers tended to weight Relevance more highly (2.7% points difference) and Feasibility less highly (3.1% points difference) than researchers. The research or grant writing experience of researchers or consumers was not associated with the weights. A multi-criteria tool for evaluating research proposals that reflects stakeholders' preferences was created. The tool can be used to assess the relative merits of clinical trial research proposals and rank them, to help identify the best proposals for funding.
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Pesquisa sobre Serviços de Saúde , Projetos de Pesquisa , Estudos Transversais , Austrália , Inquéritos e Questionários , Prioridades em SaúdeRESUMO
INTRODUCTION: The COVID-19 pandemic caused rapid implementation of telehealth for melanoma follow-up care in Australia. This study explores Australian melanoma patients and clinicians' level of satisfaction with telehealth. METHODS: A cross-sectional study was conducted across three specialist melanoma centres in Sydney, Australia. Melanoma patients (all stages) and clinicians completed mixed methods surveys seeking socio-demographic and clinical information and questionnaires to assess satisfaction with telehealth. Additionally, patients completed measures of quality of life, fear of cancer recurrence and trust in their oncologist. Patients and clinicians provided open-ended responses to qualitative questions about their perceptions of telehealth. RESULTS: One hundred and fifteen patients and 13 clinicians responded to surveys. Telephone was used by 109 (95%) patients and 11 (85%) clinicians. Fifty-seven (50%) patients and nine (69%) clinicians preferred face-to-face consultations, 38 (33%) patients and 3 (23%) clinicians preferred a combination of face-to-face and telehealth consultations. Five (4%) patients and nil clinicians preferred telehealth consultations. Patients diagnosed with early-stage melanoma, using telehealth for the first time, who have lower trust in their oncologist, and having higher care delivery, communication and supportive care concerns were likely to report lower satisfaction with telehealth. Open-ended responses were consistent between patients and clinicians, who reported safety, convenience and improved access to care as major benefits, while identifying personal, interpersonal, clinical and system-related disadvantages. DISCUSSION: While telehealth has been widely implemented during COVID-19, the benefits identified by patients and clinicians may extend past the pandemic. Telehealth may be considered for use in conjunction with face-to-face consultations to provide melanoma follow-up care.
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COVID-19 , Melanoma , Telemedicina , Humanos , Satisfação do Paciente , COVID-19/epidemiologia , Pandemias , Estudos Transversais , Seguimentos , Melanoma/epidemiologia , Melanoma/terapia , Qualidade de Vida , Austrália/epidemiologia , Encaminhamento e Consulta , Satisfação PessoalRESUMO
BACKGROUND: Patients with kidney failure on hemodialysis (HD) experience considerable symptom burden and poor health-related quality of life (HRQoL). There is limited use of patient reported outcome measures (PROMs) in facility HD units to direct immediate care, with response rates in other studies between 36 to 70%. The aim of this pilot study was to evaluate feasibility of electronic PROMs (e-PROMs) in HD participants, with feedback 3-monthly to the participants' treating team, for severe or worsening symptoms as identified by the Integrated Palliative Outcome Scale (IPOS-Renal), with linkage to the Australian and New Zealand Dialysis and Transplant (ANZDATA) registry, compared with usual care. METHODS: This is a registry-based cluster-randomized controlled pilot trial involving all adults receiving HD in 4 satellite units in Australia over a 6-month period. HD units were cluster randomized 1:1 to the control (HRQoL data collection only) or intervention arm (symptom monitoring with feedback to treating team every 3 months). Feasibility was assessed by participant response rate (percentage of eligible HD participants, including new incident participants, who completed the questionnaire at each time point); retention rate (percentage of participants who completed the baseline questionnaire and all subsequent measures); and completion time. HRQoL and symptom burden scores are described. RESULTS: There were 226 unique participants who completed the e-PROMs (mean age 62 years, 69% males, 78% White-European, median dialysis vintage 1.62 years). At 6 months, response rate and retention rate for the intervention arm were 54% and 68%, respectively, and 89% and 97% in the control arm. Median time to complete IPOS-Renal was 6.6 min (5.3, 10.1) at 3 months, and when combined with the outcome measure (EQ-5D-5L), the median time was 9.4 min (6.9, 13.6) at 6 months. CONCLUSIONS: Electronic symptom monitoring among HD participants with feedback to clinicians is feasible. Variations in response and retention rates could be potentially explained by the lengthier questionnaire, and higher frequency of data collection time points for participants in the intervention arm. A definitive national RCT is underway. TRIAL REGISTRATION: ACTRN12618001976279 (07/12/2018).
Assuntos
Qualidade de Vida , Diálise Renal , Masculino , Humanos , Adulto , Pessoa de Meia-Idade , Feminino , Projetos Piloto , Retroalimentação , Estudos de Viabilidade , Austrália/epidemiologia , Sistema de RegistrosRESUMO
BACKGROUND AND HYPOTHESIS: People on the kidney waitlist are less informed about potential suspensions. Disparities may exist among those who are suspended and who return to the waitlist. We evaluated the patient journey after entering the waitlist, including suspensions and outcomes, and factors associated with these transitions. METHODS: We included all incident patients waitlist for their first transplant from deceased donors in Australia, 2006-19. We described all clinical transitions after entering the waitlist. We predicted the restricted mean survival time (unadjusted and adjusted) until first transplant by number of prior suspensions. We evaluated factors associated with transitions using flexible survival models and clinical endpoints using Cox models. RESULTS: Of 8 466 patients waitlisted and followed over 45 757.4 person-years (median:4.8years), 6 741(80%) were transplanted, 381(5%) died waiting and 1 344(16%) were still waiting. 3 127(37%) people were suspended at least once. Predicted mean time from waitlist to transplant was 3.0 years(95%CI:2.8-3.2) when suspended versus 1.9 years(95%CI:1.8-1.9) when never suspended. Prior suspension increased likeliness of further suspensions 4.2-fold(95%CI:3.8-4.6) and returning to waitlist by 50%(95%CI:36-65%) but decreased likeliness of transplantation by 29%(95%CI:62-82%). Death risk while waiting was 12-fold(95%CI:8.0-18.3) increased when currently suspended. Australian non-Indigenous males were 13% (HR:1.13,95%CI:1.04-1.23) and Asian males 23% (HR:1.23,95%CI:1.06-1.42,) more likely to return to the waitlist compared to females of the same ethnicity. CONCLUSION: The waitlist journey was not straightforward. Suspension was common, impacted chance of transplantation and meant waiting an average one year longer until transplant. We have provided estimates for, and factors associated with, suspension, re-listing and outcomes after waitlisting to support more informed discussions. This evidence is critical to further understand drivers of inequitable access to transplantation.
RESUMO
BACKGROUND: The prevalence of neonatal abstinence syndrome is increasing, but the number and quality of clinical practice guidelines available are unknown. This systematic review aimed to identify, appraise and evaluate clinical practice guidelines for neonatal abstinence syndrome. METHODS: A systematic search of databases and the grey literature was conducted between 1 June and 1 July 2022. Full-text guidelines published by national or state-wide institutions were included. The recommendations from each guideline were extracted. The AGREE-II instrument was used to assess guideline quality. Sufficient-quality scores were defined as >60 and good-quality scores were >80 for each domain of AGREE-II. RESULTS: A total of 1703 records were identified, and 22 guidelines from the United States, Australia, Canada and the United Kingdom, published between 2012 to 2021, were included. The quality scores were low, with median scores of 37/100 for stakeholder involvement, 33/100 for methodology, 34/100 for applicability and 0 for editorial independence. Scope and purpose scored 72/100, and presentation scored 85/100. Sixteen (73%) guidelines did not meet the cut-offs for clinical use. CONCLUSION: Many guidelines were of insufficient quality to guide clinical practice for neonatal abstinence syndrome. This emphasises the need for high-quality studies to inform clinical practice guidelines, improve care and reduce the risk of poor outcomes in these high-risk infants.
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INTRODUCTION: Three-dimensional (3D) total body photography may improve early detection of melanoma and facilitate surveillance, leading to better prognosis and lower healthcare costs. The Australian Centre of Excellence in Melanoma Imaging and Diagnosis (ACEMID) cohort study will assess long-term outcomes from delivery of a precision strategy of monitoring skin lesions using skin surface imaging technology embedded into health services across Australia. METHODS AND ANALYSIS: A prospective cohort study will enrol 15 000 participants aged 18 years and above, across 15 Australian sites. Participants will attend study visits according to their melanoma risk category: very high risk, high risk or low/average risk, every 6, 12 and 24 months, respectively, over 3 years. Participants will undergo 3D total body photography and dermoscopy imaging at study visits. A baseline questionnaire will be administered to collect sociodemographic, phenotypic, quality of life and sun behaviour data. A follow-up questionnaire will be administered every 12 months to obtain changes in sun behaviour and quality of life. A saliva sample will be collected at the baseline visit from a subsample. ETHICS AND DISSEMINATION: The ACEMID cohort study was approved by the Metro South Health Human Research Ethics Committee (approval number: HREC/2019/QMS/57206) and the University of Queensland Human Research Ethics Committee (approval number: 2019003077). The findings will be reported through peer-reviewed and lay publications and presentations at conferences. TRIAL REGISTRATION NUMBER: ACTRN12619001706167.