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Research in Gerontological Nursing May 2020Pragmatic trials occur within the complexity of real-world care delivery, and when effective, contribute to more rapid translation into practice because of their greater...
Pragmatic trials occur within the complexity of real-world care delivery, and when effective, contribute to more rapid translation into practice because of their greater generalizability. Research with older adults is complex when participants have chronic conditions and multiple comorbidities. Often pragmatic trials introduce a novel intervention and try to determine whether it offers a benefit beyond the usual or routine care provided. Researchers commonly focus attention on describing the intervention, yet the comparator condition of usual or routine care can be anything but standard, reducing the effect size of the intervention and introducing threats to the overall validity of the study. The current article describes clinical trial guidelines, then illustrates the complexity of characterizing usual care for interventions addressing type 2 diabetes. The authors provide recommendations for improving description of usual care and discuss implications for gerontological nursing research. [Research in Gerontological Nursing, 13(3), 125-129.].
Topics: Aged; Delivery of Health Care; Diabetes Mellitus, Type 2; Humans; Nursing Research; Randomized Controlled Trials as Topic
PubMed: 31834414
DOI: 10.3928/19404921-20191127-01 -
BMC Health Services Research Nov 2022Transition from paediatric to adult heath care services is a challenging time for many adolescents with chronic illnesses and may include deterioration in illness...
Study protocol: a pragmatic trial reviewing the effectiveness of the TransitionMate mobile application in supporting self-management and transition to adult healthcare services for young people with chronic illnesses.
BACKGROUND
Transition from paediatric to adult heath care services is a challenging time for many adolescents with chronic illnesses and may include deterioration in illness control as a consequence of inadequate self-management skills, poor understanding of their chronic illness and failure to engage with adult services. Successful transfer of health care requires the development of self-management skills and increased autonomy. Mobile technology has been proposed as a modality to assist this process. Evidence is limited and generally restricted to illness specific applications. The TransitionMate app (TMApp) is a generic (non-illness specific) mobile application designed to support young people with chronic illness in their transition from paediatric to adult health care services. The overall aim of the study is to assess the effectiveness of TMApp in improving engagement and retention of adolescents with chronic illness within adult healthcare services, as well as preventing the deterioration in illness control and unplanned hospitalisations.
METHODS
The TransitionMate trial is a dual centre, pragmatic, single arm, mixed methods cohort study conducted within two university teaching tertiary paediatric hospitals in Australia. Data collection points are planned at 0, 6, 12 and 18 months. Outcome indicators include: usage of TransitionMate, engagement with adult services, quantitative markers of illness control, and unplanned hospital admissions. Data are collected through telephone interviews with the participants, their primary healthcare providers, electronic medical records and de-identified mobile application analytics. The development of the application involved co-design with recently transitioned young people with a number of chronic illnesses as well as online user experience in younger adolescents.
DISCUSSION
The TransitionMate study is the first identified trial of a generic mobile application designed to support adolescents with chronic illnesses during the transition process. Results are expected to provide novel insights into the value of technological tools in the transition space, especially their effectiveness in improving both the transition process and clinical outcomes of adolescents with chronic illnesses. Furthermore, the approach of a pragmatic study design may help identify research methods better designed to overcome inherent challenges in research involving adolescents, transition of care and use of mobile application technology.
TRIAL REGISTRATION
Registered retrospectively as of 30/1/2020 with Australian New Zealand Clinical Trials Registry: ACTRN12620000074998 .
Topics: Adolescent; Humans; Australia; Chronic Disease; Cohort Studies; Mobile Applications; Self-Management; Pragmatic Clinical Trials as Topic; Multicenter Studies as Topic; Transition to Adult Care
PubMed: 36447255
DOI: 10.1186/s12913-022-08536-8 -
Contemporary Clinical Trials Feb 2023The GetReal Trial Tool is a decision support tool to assess the impact of design choices on generalizability of clinical trials to routine clinical practice, while...
BACKGROUND
The GetReal Trial Tool is a decision support tool to assess the impact of design choices on generalizability of clinical trials to routine clinical practice, while taking into account the risk of bias, precision, acceptability and operational feasibility. This study describes the validation of the GetReal Trial Tool.
METHODS
Twelve experts took part in the GetReal Trial tool validation using the protocols of 6 trials conducted with pragmatic elements. The tool entails 7 domains with a total of 43 questions. A pooled Kappa statistic (95% CI) using random effects model was estimated using Open Meta (analyst) software. The possible operational challenges were collated and discussed with the trialists that conducted the trials.
RESULTS
Agreement in the design choices made for the trial protocols was >50% for all the trials and all teams reached consensus during discussion. The pooled Kappa statistic (95% CI) was 0.236 (0.154-0.318). The GetReal Trial tool highlighted several operational challenges, of which almost half had been experienced previously by the trialists. Out of 25 additional operational challenges mentioned by the trialists, 76% were already highlighted by the tool. The tool was considered helpful to optimize trials right from the design stage.
CONCLUSION
The GetReal Trial Tool helps to scrutinize the choice of study design in the light of Real World Evidence generation. The tool identifies most of the operational challenges experienced by trialists to date. The tool serves the intended purpose of facilitating discussion and understanding more pragmatic design choices and their implications.
Topics: Humans; Research Design; Clinical Trials as Topic; Decision Support Techniques
PubMed: 36529438
DOI: 10.1016/j.cct.2022.107054 -
BMJ Open Sep 2022Living with diabetes can be burdensome and lead to serious emotional distress and impaired mental health. Acceptance and commitment therapy (ACT) can support people...
ACTonDiabetes: study protocol of a pragmatic randomised controlled trial for the evaluation of an acceptance and commitment-based internet-based and mobile-based intervention for adults living with type 1 or type 2 diabetes.
INTRODUCTION
Living with diabetes can be burdensome and lead to serious emotional distress and impaired mental health. Acceptance and commitment therapy (ACT) can support people facing the challenges of living with diabetes. This trial aims to evaluate the effectiveness and cost-effectiveness of the internet-based and mobile-based intervention (IMI) 'ACTonDiabetes' in reducing diabetes distress against enhanced treatment as usual (TAU+) following specialised diabetes care.
METHODS AND ANALYSIS
A two-armed pragmatic randomised controlled trial will be conducted to evaluate the guided IMI ACTonDiabetes against TAU+. A total of 210 adults with type 1 or type 2 diabetes and elevated diabetes distress (Problem Areas in Diabetes ≥40) will be recruited at a specialised diabetes centre. The intervention begins 2-4 weeks after hospital discharge and takes about 7-10 weeks to complete. Assessments are performed at baseline and 5 and 10 weeks as well as 6 and 12 months after randomisation. The primary outcome is diabetes distress at a 10-week follow-up (T2). Secondary outcomes are depression (Patient Health Questionnaire-8), psychological well-being (WHO-5), quality of life (Assessment of Quality of Life-8 Dimension), Diabetes-related Self-Management Questionnaire, diabetes acceptance (Acceptance and Action Diabetes Questionnaire) and negative treatment effects (Inventory for the Assessment of Negative Effects of Psychotherapy). All statistical analyses will be performed based on the intention-to-treat principle with additional per-protocol analyses. Changes in outcomes will be evaluated using the general linear model. A health-economic evaluation will be conducted from a societal perspective. Reasons for drop-out will be systematically investigated.
ETHICS AND DISSEMINATION
This clinical trial has been approved by the State Medical Chamber of Baden-Württemberg (file no. B-F-2019-010). Trial results will be submitted for publication in a peer-reviewed journal and presented at conferences.
TRIAL REGISTRATION NUMBER
DRKS00016738.
Topics: Adult; Humans; Acceptance and Commitment Therapy; Diabetes Mellitus, Type 2; Internet; Patient Health Questionnaire; Pragmatic Clinical Trials as Topic; Quality of Life; Diabetes Mellitus, Type 1
PubMed: 36109030
DOI: 10.1136/bmjopen-2021-059336 -
BMJ Open Jan 2024It is encouraged to estimate the effectiveness of components within the enhanced recovery after surgery (ERAS) protocol through patient-reported outcomes, alongside...
Effectiveness of general anaesthesia with remimazolam tosilate on intraoperative haemodynamics and postoperative recovery: study protocol for a randomised, positive-controlled, pragmatic clinical trial (GARTH trial).
INTRODUCTION
It is encouraged to estimate the effectiveness of components within the enhanced recovery after surgery (ERAS) protocol through patient-reported outcomes, alongside doctor-reported outcomes and length of hospital stay. At present, studies on the contributions of optimal anaesthetic drugs within the ERAS protocol to patient-reported and doctor-reported outcomes are limited. Therefore, this study aims to pragmatically evaluate the effectiveness and safety of general anaesthesia (GA) with remimazolam tosilate within the ERAS protocol on intraoperative haemodynamics and postoperative recovery in adults undergoing elective surgeries, compared with propofol.
METHODS AND ANALYSIS
This study is a single-centre, randomised, blinded, positive-controlled, pragmatic clinical trial. A total of 900 patients, aged ≥18 years old, scheduled for an elective surgical procedure under GA will be included. Patients will be randomised in a 1:1 ratio to the remimazolam group (the GA with remimazolam tosilate within the ERAS protocol group) or propofol group (the GA with propofol within the ERAS protocol group), stratified by general surgery, thoracic surgery and other surgeries (including urological surgery and otolaryngology surgery). The primary outcomes include the 24-hour postoperative quality of recovery-40 score and the rate of intraoperative hypotension. Secondary endpoints include the rate of sedative hypotension requiring treatment, the haemodynamic profiles, the 72-hour postoperative quality of recovery-40 score, the functional anaesthetic capability, adverse events and complications, quality of life within 3 months as well as economic health outcomes.
ETHICS AND DISSEMINATION
This study protocol has been approved by the ethics committee of Guangdong Provincial People's Hospital (KY-H-2022-005-03-08). Dissemination plans will be presented at scientific meetings and in scientific publications.
TRIAL REGISTRATION NUMBER
ChiCTR2200062520.
Topics: Adolescent; Adult; Humans; Anesthesia, General; Anesthetics; Hemodynamics; Hypotension; Postoperative Complications; Propofol; Quality of Life; Randomized Controlled Trials as Topic; Pragmatic Clinical Trials as Topic
PubMed: 38176870
DOI: 10.1136/bmjopen-2023-073024 -
PLoS Medicine Feb 2022Use of patient-reported outcomes (PROs) and patient and public engagement are critical ingredients of pragmatic trials, which are intended to be patient centered.... (Review)
Review
BACKGROUND
Use of patient-reported outcomes (PROs) and patient and public engagement are critical ingredients of pragmatic trials, which are intended to be patient centered. Engagement of patients and members of the public in selecting the primary trial outcome and determining the target difference can better ensure that the trial is designed to inform the decisions of those who ultimately stand to benefit. However, to the best of our knowledge, the use and reporting of PROs and patient and public engagement in pragmatic trials have not been described. The objectives of this study were to review a sample of pragmatic trials to describe (1) the prevalence of reporting patient and public engagement; (2) the prevalence and types of PROs used; (3) how its use varies across trial characteristics; and (4) how sample sizes and target differences are determined for trials with primary PROs.
METHODS AND FINDINGS
This was a methodological review of primary reports of pragmatic trials. We used a published electronic search filter in MEDLINE to identify pragmatic trials, published in English between January 1, 2014 and April 3, 2019; we identified the subset that were registered in ClinicalTrials.gov and explicitly labeled as pragmatic. Trial descriptors were downloaded from ClinicalTrials.gov; information about PROs and sample size calculations were extracted from the manuscript. Chi-squared, Cochran-Armitage, and Wilcoxon rank sum tests were used to examine associations between trial characteristics and use of PROs. Among 4,337 identified primary trial reports, 1,988 were registered in CT.gov, of which 415 were explicitly labeled as pragmatic. Use of patient and public engagement was identified in 39 (9.4%). PROs were measured in 235 (56.6%): 144 (34.7%) used PROs as primary outcomes and 91 (21.9%) as only secondary outcomes. Primary PROs were symptoms (64; 44%), health behaviors (36; 25.0%), quality of life (17; 11.8%), functional status (16; 11.1%), and patient experience (10; 6.9%). Trial characteristics with lower prevalence of use of PROs included being conducted exclusively in children or adults over age 65 years, cluster randomization, recruitment in low- and middle-income countries, and primary purpose of prevention; trials conducted in Europe had the highest prevalence of PROs. For the 144 trials with a primary PRO, 117 (81.3%) reported a sample size calculation for that outcome; of these, 71 (60.7%) justified the choice of target difference, most commonly, using estimates from pilot studies (31; 26.5%), standardized effect sizes (20; 17.1%), or evidence reviews (16; 13.7%); patient or stakeholder opinions were used to justify the target difference in 8 (6.8%). Limitations of this study are the need for trials to be registered in ClinicalTrials.gov, which may have reduced generalizability, and extracting information only from the primary trial report.
CONCLUSIONS
In this study, we observed that pragmatic trials rarely report patient and public engagement and do not commonly use PROs as primary outcomes. When provided, target differences are often not justified and rarely informed by patients and stakeholders. Research funders, scientific journals, and institutions should support trialists to incorporate patient engagement to fulfill the mandate of pragmatic trials to be patient centered.
Topics: Cross-Sectional Studies; Databases, Factual; Humans; Patient Participation; Patient Reported Outcome Measures; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic
PubMed: 35134080
DOI: 10.1371/journal.pmed.1003896 -
Drug Design, Development and Therapy 2023Although empagliflozin has been recommended for individuals with heart failure, its effects on heart failure with preserved ejection fraction (HFpEF) remain uncertain...
Effects of Empagliflozin on Gut Microbiota in Heart Failure with a Preserved Ejection Fraction: The Design of a Pragmatic Randomized, Open-Label Controlled Trial (EMPAGUM).
Although empagliflozin has been recommended for individuals with heart failure, its effects on heart failure with preserved ejection fraction (HFpEF) remain uncertain from a physiopathological standpoint. The metabolites produced by gut microbiota have been shown to have a crucial role in the development of heart failure. Sodium-glucose cotransporter-2 inhibitors (SGLT2) have been shown to change the make-up of the gut microbiota in rodent studies. There is mixed evidence from similar studies investigating whether or not SGLT2 can affect the microbiota in the human gut. This trial is a pragmatic, randomized, open-label controlled study with empagliflozin as an intervention. We will enroll 100 patients with HFpEF and randomly assign them to one of two groups to receive either empagliflozin or a placebo. Patients in the Empagliflozin group will be given 10 mg of the drug daily, while those in the Control group will not be given empagliflozin or any other SGLT2. The purpose of the trial is to validate the changes that occur in gut microbiota in patients with HFpEF who take empagliflozin and to investigate the function of gut microbiota and their metabolites in the process.
Topics: Humans; Gastrointestinal Microbiome; Heart Failure; Sodium-Glucose Transporter 2 Inhibitors; Stroke Volume; Pragmatic Clinical Trials as Topic
PubMed: 37223722
DOI: 10.2147/DDDT.S404479 -
European Journal of Rheumatology Jul 2022The aim of this qualitative research was to identify physician-perceived patient and clinic barriers to patient recruitment in a rheumatoid arthritis (RA) pragmatic...
OBJECTIVE
The aim of this qualitative research was to identify physician-perceived patient and clinic barriers to patient recruitment in a rheumatoid arthritis (RA) pragmatic trial of anti-tumor necrosis factor (TNF) biologic versus non-TNF biologic/Janus-Kinase inhibitor initiation after an inadequate response to methotrexate.
METHODS
Semistructured telephone interviews were conducted with 26 rheumatologists in March 2019. An exploratory thematic analysis approach was used to analyze the interview data.
RESULTS
Physician perceived patient barriers to the implementation of an RA pragmatic trial. This theme covers three subthemes: (1) patients' personal barriers, (2) patients' treatment-related factors, and (3) trial-related factors (eg, patient recruitment, side effects, mode of use, etc). Physicians perceived clinic barriers interfered with the pragmatic trial enrollment from the clinic or the healthcare system perspective. This theme covered four subthemes: (1) clinic-related factors, (2) patient-related factors, (3) research personnel, and (4) facilitators (positive factors of the clinic).
CONCLUSION
Our results from the inductive thematic analysis will help researchers understand the key patient and clinic/system factors/barriers that may influence pragmatic RA trial implementation. The themes suggest there are factors that can be modified (eg, coordinator effort needed, effective patient recruitment during clinic visits, provider engagement) and challenges to overcome (patient insurance status, busy clinic flow, and space issues including limited number of patient rooms). In summary, these themes provide a basis for our and other research teams to develop clinic-centered and patientcentered strategies to implement a pragmatic RA trial.
Topics: Arthritis, Rheumatoid; Biological Products; Humans; Physician-Patient Relations; Physicians; Pragmatic Clinical Trials as Topic; Qualitative Research
PubMed: 35156626
DOI: 10.5152/eujrheum.2022.21038 -
BMC Medical Research Methodology Aug 2019The paper opens with a brief history of two of the major intellectual components of the recent utilitarian turn in clinical research, namely 'pragmatic trials' and...
BACKGROUND
The paper opens with a brief history of two of the major intellectual components of the recent utilitarian turn in clinical research, namely 'pragmatic trials' and 'implementation science'. The two schools of thought developed independently and the paper scrutinises their mutual compatibilities and incompatibilities, asking: i) what do the leading advocates of pragmatic trials assume about the transfer of research findings to real-world practice and ii) what role pragmatic trials can and should play in the evaluation of implementation science strategies.
METHODS
The paper utilises 'explication de texte': i) providing a close reading of the inferential logics contained in major published expositions of the two paradigms, and ii) interrogating the conclusions of a pragmatic trial of an intervention providing guidelines on retinal screening aimed at family practitioners.
RESULTS
The paper is in two parts. Part 1 unearths some significant incommensurability - the pragmatic trial literature retains an antiquated view of knowledge transfer and is overly optimistic about the wide applicability the findings of pragmatic trials to 'real world' conditions. Part 2 of the paper outlines an empirical strategy to better penetrate the mechanisms of knowledge transfer and to tackle the issue of the generalisabilty of research findings in implementation science.
CONCLUSIONS
Pragmatism, classically, is about problem solving and the melding of perspectives. The core research requirement in implementation science is a fundamental shift from the narrow shoulders of pragmatic trials to a model of explanation building based upon a multi-case, multi-method body of evidence.
Topics: Biomedical Research; Evidence-Based Practice; Humans; Implementation Science; Pragmatic Clinical Trials as Topic; Reproducibility of Results; Research Design
PubMed: 31420024
DOI: 10.1186/s12874-019-0814-9 -
Journal of Clinical Epidemiology Dec 2022To review the pragmatism of published randomized trials of remdesivir and favipiravir based on the Pragmatic-Explanatory Continuum Indicator Summary (PRECIS-2) framework. (Review)
Review
How pragmatic are randomized trials of remdesivir and favipiravir for in-hospital treatment of COVID-19: a descriptive methodological review of trial design using the PRECIS-2 framework.
OBJECTIVES
To review the pragmatism of published randomized trials of remdesivir and favipiravir based on the Pragmatic-Explanatory Continuum Indicator Summary (PRECIS-2) framework.
STUDY DESIGN AND SETTING
Ten eligible trials were identified from an existing comprehensive living review and were evaluated across the nine PRECIS-2 domains by two independent reviewers.
RESULTS
All 10 trials had mostly pragmatic design characteristics. Four of the domains (i.e., recruitment, setting, organization, and primary analysis) were found to be pragmatic with most trials scoring four or five across the two interventions. In comparison scores for four other design domains (i.e., eligibility, follow-up, flexibility of delivery, and primary outcome) varied across the trials with some design choices being more explanatory.
CONCLUSION
In our descriptive review of randomized controlled trails for two drugs for patients infected with COVID-19 early in the pandemic, we found that most trials had more pragmatic than explanatory characteristics. Some design choices for some of the trials, however, were not consistent with the urgent goal of informing clinical decision making in an epidemic. PRECIS-2 should be used as a guide by trialists, to help them match their trial design choices to the intended purpose of their trial.
Topics: Humans; COVID-19 Drug Treatment; Hospitals; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic; Research Design
PubMed: 36265553
DOI: 10.1016/j.jclinepi.2022.10.013