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International Journal of Stroke :... Oct 2021Tremendous progress in acute stroke therapy has improved short-term outcome but part of this achievement may be lost in the long run. Concepts for a better long-term...
BACKGROUND
Tremendous progress in acute stroke therapy has improved short-term outcome but part of this achievement may be lost in the long run. Concepts for a better long-term management of stroke survivors are needed to address their unmet needs and to reduce the burden of post-stroke complications, residual deficits, and recurrent vascular events.
AIMS
This review summarizes current knowledge on post-hospital care and the scientific evidence supporting individual programs.
SUMMARY OF REVIEW
A systematic search of electronic databases according to PRISMA guidelines identified 10,374 articles, 77 of which met the inclusion criteria. One large randomised controlled trial on a multifaceted care program delivered by the multidisciplinary stroke team reduced recurrent vascular events and improved quality of life and functional outcome one year after the event, while a number of studies offer solutions for individual components of post-hospital disease management like patient education, counselling, and self-management or the management of post-stroke complications and residual deficits. A majority of studies, however, was small in size and limited by a short follow-up. Most initiatives with a narrow focus on risk factor control failed to lower the risk of recurrent events. The caregivers' central role in post-stroke patient management is broadly neglected in research.
CONCLUSIONS
Over the past years, first knowledge on how to best organize post-hospital care of stroke patients has emerged. Comprehensive and pragmatic programs operated by the multidisciplinary stroke team hold promise to reduce the long-term health burden of stroke. There is a clear need for further high-quality studies with both clinical endpoints and patient-reported outcomes to establish sustainable solutions in different settings and regions to improve life after stroke, a key priority of the Stroke Action Plan for Europe 2018-2030.
Topics: Caregivers; Europe; Humans; Quality of Life; Randomized Controlled Trials as Topic; Risk Factors; Stroke
PubMed: 33949269
DOI: 10.1177/17474930211016963 -
PloS One 2019Complementary and Alternative Medicines (CAM) are widely used by cancer patients, despite limited evidence of efficacy. Manipulative and body-based practices are some of... (Meta-Analysis)
Meta-Analysis
BACKGROUND
Complementary and Alternative Medicines (CAM) are widely used by cancer patients, despite limited evidence of efficacy. Manipulative and body-based practices are some of the most commonly used CAM. This systematic review evaluates their benefits in oncology.
METHOD
A systematic literature review was carried out with no restriction of language, time, cancer location or type. PubMed, CENTRAL, PsycArticle, PsychInfo, Psychology and Behavioral Sciences Collection and SOCindex were queried. Inclusion criteria were adult cancer patients and randomized controlled trials (RCT) assessing manipulative and body-based complementary practices on psychological and symptom outcomes. Effect size was calculated when applicable.
RESULTS
Of 1624 articles retrieved, 41 articles were included: massage (24), reflexology (11), acupressure (6). Overall, 25 studies showed positive and significant effects on symptom outcomes (versus 9 that did not), especially pain and fatigue. Mixed outcomes were found for quality of life (8 papers finding a significant effect vs. 10 which did not) and mood (14 papers vs. 13). In most studies, there was a high risk of bias with a mean Jadad score of 2, making interpretation of results difficult.
CONCLUSION
These results seem to indicate that manipulative CAM may be effective on symptom management in cancer. However, more robust methodologies are needed. The methodological requirements of randomized controlled trials are challenging, and more informative results may be provided by more pragmatic study design.
Topics: Combined Modality Therapy; Complementary Therapies; Humans; Medical Oncology; Neoplasms; Psychotherapy; Treatment Outcome
PubMed: 31622362
DOI: 10.1371/journal.pone.0223564 -
Registry-based randomised controlled trials: conduct, advantages and challenges-a systematic review.Trials Jun 2024Registry-based randomised controlled trials (rRCTs) have been described as pragmatic studies utilising patient data embedded in large-scale registries to facilitate key...
BACKGROUND
Registry-based randomised controlled trials (rRCTs) have been described as pragmatic studies utilising patient data embedded in large-scale registries to facilitate key clinical trial procedures including recruitment, randomisation and the collection of outcome data. Whilst the practice of utilising registries to support the conduct of randomised trials is increasing, the use of the registries within rRCTs is inconsistent. The purpose of this systematic review is to explore the conduct of rRCTs using a patient registry to facilitate trial recruitment and the collection of outcome data, and to discuss the advantages and challenges of rRCTs.
METHODS
A systematic search of the literature was conducted using five databases from inception to June 2020: PubMed, Embase (through Ovid), CINAHL, Scopus and the Cochrane Controlled Register of Trials (CENTRAL). The search strategy comprised of MESH terms and key words related to rRCTs. Study selection was performed independently by two reviewers. A risk of bias for each study was completed. A narrative synthesis was conducted.
RESULTS
A total 47,862 titles were screened and 24 rRCTs were included. Eleven rRCTs (45.8%) used more than one registry to facilitate trial conduct. Six rRCTs (25%) randomised participants via a specific randomisation module embedded within a registry. Recruitment ranged between 209 to 106,000 participants. Advantages of rRCTs are recruitment efficiency, shorter trial times, cost effectiveness, outcome data completeness, smaller carbon footprint, lower participant burden and the ability to conduct multiple trials from the same registry. Challenges are data collection/management, quality assurance issues and the timing of informed consent.
CONCLUSIONS
Optimising the design of rRCTs is dependent on the capabilities of the registry. New registries should be designed and existing registries reviewed to enable the conduct of rRCTs. At all times, data management and quality assurance of all registry data should be given key consideration. We suggest the inclusion of the term 'registry-based' in the title of all rRCT manuscripts and a clear simple breakdown of the registry-based conduct of the trial in the abstract to facilitate indexing in the major databases.
Topics: Humans; Patient Selection; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic; Registries; Research Design
PubMed: 38863017
DOI: 10.1186/s13063-024-08209-3 -
JAMA Network Open Jun 2021Randomized clinical trials (RCTs) provide the highest level of evidence to evaluate 2 or more surgical interventions. Surgical RCTs, however, face unique challenges in...
IMPORTANCE
Randomized clinical trials (RCTs) provide the highest level of evidence to evaluate 2 or more surgical interventions. Surgical RCTs, however, face unique challenges in design and implementation.
OBJECTIVE
To evaluate the design, conduct, and reporting of contemporary surgical RCTs.
EVIDENCE REVIEW
A literature search performed in the 2 journals with the highest impact factor in general medicine as well as 6 key surgical specialties was conducted to identify RCTs published between 2008 and 2020. All RCTs describing a surgical intervention in both experimental and control arms were included. The quality of included data was assessed by establishing an a priori protocol containing all the details to extract. Trial characteristics, fragility index, risk of bias (Cochrane Risk of Bias 2 Tool), pragmatism (Pragmatic Explanatory Continuum Indicator Summary 2 [PRECIS-2]), and reporting bias were assessed.
FINDINGS
A total of 388 trials were identified. Of them, 242 (62.4%) were registered; discrepancies with the published protocol were identified in 81 (33.5%). Most trials used superiority design (329 [84.8%]), and intention-to-treat as primary analysis (221 [56.9%]) and were designed to detect a large treatment effect (50.0%; interquartile range [IQR], 24.7%-63.3%). Only 123 trials (31.7%) used major clinical events as the primary outcome. Most trials (303 [78.1%]) did not control for surgeon experience; only 17 trials (4.4%) assessed the quality of the intervention. The median sample size was 122 patients (IQR, 70-245 patients). The median follow-up was 24 months (IQR, 12.0-32.0 months). Most trials (211 [54.4%]) had some concern of bias and 91 (23.5%) had high risk of bias. The mean (SD) PRECIS-2 score was 3.52 (0.65) and increased significantly over the study period. Most trials (212 [54.6%]) reported a neutral result; reporting bias was identified in 109 of 211 (51.7%). The median fragility index was 3.0 (IQR, 1.0-6.0). Multiplicity was detected in 175 trials (45.1%), and only 35 (20.0%) adjusted for multiple comparisons.
CONCLUSIONS AND RELEVANCE
In this systematic review, the size of contemporary surgical trials was small and the focus was on minor clinical events. Trial registration remained suboptimal and discrepancies with the published protocol and reporting bias were frequent. Few trials controlled for surgeon experience or assessed the quality of the intervention.
Topics: General Surgery; Humans; Randomized Controlled Trials as Topic; Time Factors
PubMed: 34190996
DOI: 10.1001/jamanetworkopen.2021.14494 -
Archivos de Bronconeumologia May 2024The effectiveness of home high flow nasal cannula (HFNC) for the treatment of chronic respiratory failure in patients with chronic respiratory diseases (CRDs) has not... (Review)
Review
INTRODUCTION
The effectiveness of home high flow nasal cannula (HFNC) for the treatment of chronic respiratory failure in patients with chronic respiratory diseases (CRDs) has not been summarized. We aimed to conduct a systematic review of the effectiveness, adherence, and safety of HFNC in the long-term treatment of patients with chronic respiratory diseases and respiratory failure.
METHODS
A systematic review was conducted. PubMed, Web of science, and SCOPUS were search up to August 2023. Long-term HFNC studies (≥4 weeks) reporting dyspnea; exacerbations, hospitalizations; peripheral oxygen saturation (SpO), comfort; patient experience, health-related quality of life or partial pressure of carbon dioxide (paCO) were included.
RESULTS
Thirteen articles (701 patients) based on 10 studies were selected: randomized control trials (n=3), randomized crossover trials (n=2), crossover (n=3) and retrospective (n=2) studies. COPD (n=6), bronchiectasis (n=2), COPD/bronchiectasis (n=1) and ILD (n=1) were the underlined CRDs. HFNC reduced exacerbations when compared to usual care/home respiratory therapies (n=6). Quality of life outcomes were also in favor of HFNC in patients with COPD and bronchiectasis (n=6). HFNC had significant effects on hospitalizations, paCO, and lung function. Adherence ranged from 5.2 to 8.6h/day (n=5). Three studies reported no events, 3 non-serious events and 2 no differences compared with other home respiratory therapies.
CONCLUSIONS
HFNC seems more effective than usual care or other home respiratory therapies in reducing exacerbations and improving quality of life in patients with COPD and bronchiectasis, while presenting good adherence and being safe. Its apparently superior effectiveness needs to be better studied in future real-world pragmatic trials.
PubMed: 38782632
DOI: 10.1016/j.arbres.2024.05.001 -
BMJ Global Health Jun 2021Routine health information system(s) (RHIS) facilitate the collection of health data at all levels of the health system allowing estimates of disease prevalence,...
BACKGROUND
Routine health information system(s) (RHIS) facilitate the collection of health data at all levels of the health system allowing estimates of disease prevalence, treatment and preventive intervention coverage, and risk factors to guide disease control strategies. This core health system pillar remains underdeveloped in many low-income and middle-income countries. Efforts to improve RHIS data coverage, quality and timeliness were launched over 10 years ago.
METHODS
A systematic review was performed across 12 databases and literature search engines for both peer-reviewed articles and grey literature reports on RHIS interventions. Studies were analysed in three stages: (1) categorisation of RHIS intervention components and processes; (2) comparison of intervention component effectiveness and (3) whether the post-intervention outcome improved above the WHO integrated disease surveillance response framework data quality standard of 80% or above.
RESULTS
5294 references were screened, resulting in 56 studies. Three key performance determinants-technical, organisational and behavioural-were proposed as critical to RHIS strengthening. Seventy-seven per cent [77%] of studies identified addressed all three determinants. The most frequently implemented intervention components were 'providing training' and 'using an electronic health management information systems'. Ninety-three per cent [93%] of pre-post or controlled trial studies showed improvements in one or more data quality outputs, but after applying a standard threshold of >80% post-intervention, this number reduced to 68%. There was an observed benefit of multi-component interventions that either conducted data quality training or that addressed improvement across multiple processes and determinants of RHIS.
CONCLUSION
Holistic data quality interventions that address multiple determinants should be continuously practised for strengthening RHIS. Studies with clearly defined and pragmatic outcomes are required for future RHIS improvement interventions. These should be accompanied by qualitative studies and cost analyses to understand which investments are needed to sustain high-quality RHIS in low-income and middle-income countries.
Topics: Delivery of Health Care; Developing Countries; Health Information Systems; Humans; Income; Poverty
PubMed: 34117009
DOI: 10.1136/bmjgh-2020-004223 -
Psychology & Health Jul 2022Reporting of the content and delivery characteristics of comparator interventions in published articles is often incomplete. This study examines the feasibility and...
OBJECTIVE
Reporting of the content and delivery characteristics of comparator interventions in published articles is often incomplete. This study examines the feasibility and validity of two methods for collecting additional information on comparator interventions from trial authors.
METHODS & MEASURES
In a systematic review of smoking cessation trials (IC-Smoke), all trial authors were asked to send unpublished comparator intervention materials and complete a specially-developed comparator intervention checklist. All published and additionally obtained information from authors were coded for behaviour change techniques (BCTs) and other characteristics (type of comparator, provider, provider training, delivery mode and treatment duration). To assess representativeness, we assessed the amount of additional information obtained from trial authors compared with the amount that was published. We examined known-group and convergent validity of comparator intervention data when using only published or also unpublished information.
RESULTS
Additional information were obtained from 91/136 (67%) of trial authors. Representativeness, known-group and convergent validity improved substantially based on the data collected by means of the comparator intervention checklist, but not by requesting authors to send any existing comparator materials.
CONCLUSIONS
Requesting authors for unpublished comparator intervention data, using specially-developed checklists and unpublished materials, substantially improves the quality of data available for systematic reviews.
PubMed: 35876093
DOI: 10.1080/08870446.2022.2081688 -
BMJ Open Apr 2022(1) To summarise the literature on the impact of paediatric weight management interventions on health outcomes in preschool age children with overweight or obesity and... (Meta-Analysis)
Meta-Analysis
OBJECTIVES
(1) To summarise the literature on the impact of paediatric weight management interventions on health outcomes in preschool age children with overweight or obesity and (2) to evaluate the completeness of intervention description and real-world applicability using validated tools.
DESIGN
Systematic review and meta-analysis.
DATA SOURCES
MEDLINE, Embase, CINAHL, Cochrane Library and PsychInfo were searched between 10 March 2015 and 21 November 2021.
ELIGIBILITY CRITERIA
Randomised controlled trials addressing weight management in preschool children (2-6 years) with overweight or obesity.
DATA EXTRACTION AND SYNTHESIS
Two reviewers independently extracted key information from each study and assessed risk of bias. Random-effects meta-analysis was performed where there was evidence for homogeneous effects. The certainty of evidence was assessed by the Grading of Recommendations Assessment, Development and Evaluation.
RESULTS
Of the 16 908 studies retrieved, 9 trials (1687 participants) met the inclusion criteria. These interventions used motivational interviewing (MI) or multicomponent educational interventions related to health behaviour approaches and were 6-12 months in duration. All studies contained some risk of bias. A difference was found in the intervention groups compared with controls for body mass index (BMI) z score (mean difference -0.10, 95% CI -0.12 to -0.09; eight trials, 1491 participants; p<0.001; I 68%), though there was substantial heterogeneity. There were no subgroup effects between studies using MI compared with studies using multicomponent interventions. The certainty of the evidence was considered low. The trials were reported in sufficient detail and were considered pragmatic.
CONCLUSIONS
Paediatric weight management interventions delivered to the parents of young children with obesity result in small declines in BMI z score. The results should be interpreted cautiously as they were inconsistent and the quality of the evidence was low.
PROSPERO REGISTRATION NUMBER
CRD42020166843.
Topics: Body Mass Index; Child; Child, Preschool; Humans; Obesity; Overweight; Parents; Quality of Life
PubMed: 35383062
DOI: 10.1136/bmjopen-2021-053523 -
The Cochrane Database of Systematic... Oct 2019Cardiovascular disease (CVD) is the leading cause of morbidity and mortality globally. Early intervention for those with high cardiovascular risk is crucial in improving... (Review)
Review
BACKGROUND
Cardiovascular disease (CVD) is the leading cause of morbidity and mortality globally. Early intervention for those with high cardiovascular risk is crucial in improving patient outcomes. Traditional prevention strategies for CVD have focused on conventional risk factors, such as overweight, dyslipidaemia, diabetes, and hypertension, which may reflect the potential for cardiovascular insult. Natriuretic peptides (NPs), including B-type natriuretic peptide (BNP) and N-terminal pro B-type natriuretic peptide (NT-proBNP), are well-established biomarkers for the detection and diagnostic evaluation of heart failure. They are of interest for CVD prevention because they are secreted by the heart as a protective response to cardiovascular stress, strain, and damage. Therefore, measuring NP levels in patients without heart failure may be valuable for risk stratification, to identify those at highest risk of CVD who would benefit most from intensive risk reduction measures.
OBJECTIVES
To assess the effects of natriuretic peptide (NP)-guided treatment for people with cardiovascular risk factors and without heart failure.
SEARCH METHODS
Searches of the following bibliographic databases were conducted up to 9 July 2019: CENTRAL, MEDLINE, Embase, and Web of Science. Three clinical trial registries were also searched in July 2019.
SELECTION CRITERIA
We included randomised controlled trials enrolling adults with one or more cardiovascular risk factors and without heart failure, which compared NP-based screening and subsequent NP-guided treatment versus standard care in all settings (i.e. community, hospital).
DATA COLLECTION AND ANALYSIS
Two review authors independently screened titles and abstracts and selected studies for inclusion, extracted data, and evaluated risk of bias. Risk ratios (RRs) were calculated for dichotomous data, and mean differences (MDs) with 95% confidence intervals (CIs) were calculated for continuous data. We contacted trial authors to obtain missing data and to verify crucial study characteristics. Using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, two review authors independently assessed the quality of the evidence and GRADE profiler (GRADEPRO) was used to import data from Review Manager to create a 'Summary of findings' table.
MAIN RESULTS
We included two randomised controlled trials (three reports) with 1674 participants, with mean age between 64.1 and 67.8 years. Follow-up ranged from 2 years to mean 4.3 years.For primary outcome measures, effect estimates from a single study showed uncertainty for the effect of NP-guided treatment on cardiovascular mortality in patients with cardiovascular risk factors and without heart failure (RR 0.33, 95% CI 0.04 to 3.17; 1 study; 300 participants; low-quality evidence). Pooled analysis demonstrated that in comparison to standard care, NP-guided treatment probably reduces the risk of cardiovascular hospitalisation (RR 0.52, 95% CI 0.40 to 0.68; 2 studies; 1674 participants; moderate-quality evidence). This corresponds to a risk of 163 per 1000 in the control group and 85 (95% CI 65 to 111) per 1000 in the NP-guided treatment group.When secondary outcome measures were evaluated, evidence from a pooled analysis showed uncertainty for the effect of NP-guided treatment on all-cause mortality (RR 0.90, 95% CI 0.60 to 1.35; 2 studies; 1354 participants; low-quality evidence). Pooled analysis indicates that NP-guided treatment probably reduces the risk of all-cause hospitalisation (RR 0.83, 95% CI 0.75 to 0.92; 2 studies; 1354 participants; moderate-quality evidence). This corresponds to a risk of 601 per 1000 in the control group and 499 (95% CI 457 to 553) per 1000 in the NP-guided treatment group. The effect estimate from a single study indicates that NP-guided treatment reduced the risk of ventricular dysfunction (RR 0.61, 95% CI 0.41 to 0.91; 1374 participants; high-quality evidence). The risk in this study's control group was 87 per 1000, compared with 53 (95% CI 36 to 79) per 1000 with NP-guided treatment. Results from the same study show that NP-guided treatment does not affect change in NP level at the end of follow-up, relative to standard care (MD -4.06 pg/mL, 95% CI -15.07 to 6.95; 1 study; 1374 participants; moderate-quality evidence).
AUTHORS' CONCLUSIONS
This review shows that NP-guided treatment is likely to reduce ventricular dysfunction and cardiovascular and all-cause hospitalisation for patients who have cardiovascular risk factors and who do not have heart failure. Effects on mortality and natriuretic peptide levels are less certain. Neither of the included studies were powered to evaluate mortality. Available evidence shows uncertainty regarding the effects of NP-guided treatment on both cardiovascular mortality and all-cause mortality; very low event numbers resulted in a high degree of imprecision in these effect estimates. Evidence also shows that NP-guided treatment may not affect NP level at the end of follow-up.As both trials included in our review were pragmatic studies, non-blinding of patients and practices may have biased results towards a finding of equivalence. Further studies with more adequately powered sample sizes and longer duration of follow-up are required to evaluate the effect of NP-guided treatment on mortality. As two trials are ongoing, one of which is a large multi-centre trial, it is hoped that future iterations of this review will benefit from larger sample sizes across a wider geographical area.
PubMed: 31613983
DOI: 10.1002/14651858.CD013015.pub2 -
World Neurosurgery May 2022Classic randomized controlled trials (RCTs) form the cornerstone for medical guidelines and protocols. However, in neurosurgery, RCTs are not always applicable to...
BACKGROUND
Classic randomized controlled trials (RCTs) form the cornerstone for medical guidelines and protocols. However, in neurosurgery, RCTs are not always applicable to everyday clinical practice. Pragmatic controlled trials aim to incorporate real-life data with the preservation of the methodologic quality. This study is a systematic literature review of all pediatric neurosurgical RCTs published between 2000 and 2020 and an analysis of their pragmatism.
METHODS
An electronic database search was performed in PubMed, EMBASE, and the Cochrane Library to identify all relevant trials. Pragmatism was evaluated retrospectively on 9 domains: eligibility, recruitment, setting, organization, flexibility (delivery and adherence), follow-up, primary outcome, and primary analysis.
RESULTS
Of the 1862 studies included, 15 met the inclusion criteria. On average, studies scored between equally pragmatic/explanatory and rather pragmatic (M = 3.59, standard deviation [SD] = 0.56). Lowest ratings were seen for setting (M = 2.80, SD = 1.66) and eligibility (M = 3.20, SD = 1.66). Highest scores of pragmatism were given to analysis (M = 4.67, SD = 0.82) and intervention organization (M = 4.60, SD = 1.06). There was no significant difference between studies based on number of patients included, main subject, or publication year.
CONCLUSIONS
Pediatric neurosurgical RCTs scored reasonably well on overall pragmatism. In the future, there will be a greater need for pragmatic controlled trials in pediatric neurosurgery to bridge the divide between real-life data and reliable methodological quality. There is an opportunity to develop further applications of pragmatism tailored to surgical interventions.
Topics: Child; Databases, Factual; Humans; Neurosurgery; Neurosurgical Procedures; PubMed
PubMed: 35505562
DOI: 10.1016/j.wneu.2021.09.124