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BMC Complementary Medicine and Therapies May 2024Pragmatic acupuncture trials (PATs) are a research tool for assessing the effectiveness of acupuncture treatments in a real-world setting. This study aimed to provide a...
BACKGROUND
Pragmatic acupuncture trials (PATs) are a research tool for assessing the effectiveness of acupuncture treatments in a real-world setting. This study aimed to provide a comprehensive methodological analysis of PATs using the PRECIS-2(PRagmatic Explanatory Continuum Indicator Summary-2) tool to determine their pragmatism.
METHODS
The MEDLINE, EMBASE, Cochrane Central Register for Controlled Trials, CINAHL, Allied and Complementary Medicine Database, China National Knowledge Infrastructure, VIP, WANFANG, Taiwan Periodical Literature Database, KoreaMed, KMbase, Research Information Service System, Oriental Medicine Advanced Searching Integrated System, CiNii and ClinicalTrials.gov were searched. The search included randomised controlled trials (RCTs) and protocols of RCTs that investigated all types of acupuncture and used self-declared pragmatic design. Two authors independently collected the basic information and characteristics of the studies and assessed their pragmatism using the nine PRECIS-2 domains and the additional domain of control.
RESULTS
A total of 93 studies were included. The means of eligibility, recruitment, organisation, primary outcome, primary analysis, and control domains were statistically larger than three and were shown to be pragmatic. The means of setting, flexibility:delivery, and follow-up domains were not greater than three and were shown to be non-pragmatic. For flexibility:adherence domain was inappropriate for assessment owing to insufficient information in the studies.
CONCLUSIONS
PATs were pragmatic in the domain of eligibility, recruitment, organisation, primary outcome, primary analysis, and control and were not pragmatic in the domain of setting, flexibility:delivery, and follow-up. Future PATs need to strengthen the pragmatism in the setting, flexibility:delivery, and follow-up domains and to describe the flexibility:adherence domain in more detail.
TRIAL REGISTRATION
CRD42021236975.
Topics: Humans; Acupuncture Therapy; Pragmatic Clinical Trials as Topic; Randomized Controlled Trials as Topic; Research Design
PubMed: 38702632
DOI: 10.1186/s12906-024-04473-7 -
JAMA Network Open Mar 2024Platform trials have become increasingly common, and evidence is needed to determine how this trial design is actually applied in current research practice.
IMPORTANCE
Platform trials have become increasingly common, and evidence is needed to determine how this trial design is actually applied in current research practice.
OBJECTIVE
To determine the characteristics, progression, and output of randomized platform trials.
EVIDENCE REVIEW
In this systematic review of randomized platform trials, Medline, Embase, Scopus, trial registries, gray literature, and preprint servers were searched, and citation tracking was performed in July 2022. Investigators were contacted in February 2023 to confirm data accuracy and to provide updated information on the status of platform trial arms. Randomized platform trials were eligible if they explicitly planned to add or drop arms. Data were extracted in duplicate from protocols, publications, websites, and registry entries. For each platform trial, design features such as the use of a common control arm, use of nonconcurrent control data, statistical framework, adjustment for multiplicity, and use of additional adaptive design features were collected. Progression and output of each platform trial were determined by the recruitment status of individual arms, the number of arms added or dropped, and the availability of results for each intervention arm.
FINDINGS
The search identified 127 randomized platform trials with a total of 823 arms; most trials were conducted in the field of oncology (57 [44.9%]) and COVID-19 (45 [35.4%]). After a more than twofold increase in the initiation of new platform trials at the beginning of the COVID-19 pandemic, the number of platform trials has since declined. Platform trial features were often not reported (not reported: nonconcurrent control, 61 of 127 [48.0%]; multiplicity adjustment for arms, 98 of 127 [77.2%]; statistical framework, 37 of 127 [29.1%]). Adaptive design features were only used by half the studies (63 of 127 [49.6%]). Results were available for 65.2% of closed arms (230 of 353). Premature closure of platform trial arms due to recruitment problems was infrequent (5 of 353 [1.4%]).
CONCLUSIONS AND RELEVANCE
This systematic review found that platform trials were initiated most frequently during the COVID-19 pandemic and declined thereafter. The reporting of platform features and the availability of results were insufficient. Premature arm closure for poor recruitment was rare.
Topics: Humans; Pandemics; COVID-19; Cognition; Data Accuracy; Medical Oncology
PubMed: 38506807
DOI: 10.1001/jamanetworkopen.2024.3109 -
Archives of Physiotherapy 2024Manual therapy is an often-utilized intervention for the management of knee osteoarthritis (OA). The interpretation of results presented by these trials can be affected... (Review)
Review
INTRODUCTION
Manual therapy is an often-utilized intervention for the management of knee osteoarthritis (OA). The interpretation of results presented by these trials can be affected by how well the study designs align applicability to real-world clinical settings.
AIM
To examine the existing body of clinical trials investigating manual therapy for knee OA to determine where they fall on the efficacy-effectiveness spectrum.
METHODS
This systematic review has been guided and informed by the Preferred Reporting Items for Systematic Review and Meta-Analysis (PRISMA) guidelines. Randomized controlled trials that investigated manual therapy treatments for adults with knee OA were retrieved via searches of multiple databases to identify trials published prior to April 2023. The Rating of Included Trials on the Efficacy-Effectiveness Spectrum (RITES) tool was used to objectively rate the efficacy-effectiveness nature of each trial design. The Cochrane Risk of Bias 2.0 assessment tool (RoB-2) was used to assess the risk of bias across five domains.
RESULTS
Of the 36 trials, a higher percentage of trials had a greater emphasis on efficacy within all four domains: participant characteristics (75.0%), trial setting (77.8%), flexibility of intervention (58.3%), and clinical relevance of experimental and comparison intervention (47.2%). In addition, 13.9% of the trials had low risk of bias, 41.7% had high risk of bias, and 44.4% had some concerns regarding bias.
CONCLUSIONS
While many trials support manual therapy as effective for the management of knee OA, a greater focus on study designs with an emphasis on effectiveness would improve the applicability and generalizability of future trials.
PubMed: 38444787
DOI: 10.33393/aop.2024.2916 -
Orphanet Journal of Rare Diseases Feb 2024Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disease caused by deficiency in arylsulfatase A (ASA) activity arising primarily from ASA...
BACKGROUND
Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disease caused by deficiency in arylsulfatase A (ASA) activity arising primarily from ASA gene (ARSA) variants. Late-infantile, juvenile and adult clinical subtypes are defined by symptom onset at ≤ 2.5, > 2.5 to < 16 and ≥ 16 years, respectively. Epidemiological data were sought to address knowledge gaps and to inform decisions regarding the clinical development of an investigational drug.
METHODS
To synthesize all available estimates of MLD incidence and birth prevalence worldwide and in selected countries, Ovid MEDLINE and Embase were searched systematically (March 11, 2022) using a population, intervention, comparator, outcome, time and setting framework, complemented by pragmatic searching to reduce publication bias. Where possible, results were stratified by clinical subtype. Data were extracted from non-interventional studies (clinical trials, non-clinical studies and case reports were excluded; reviews were used for snowballing only).
RESULTS
Of the 31 studies included, 14 reported birth prevalence (13 countries in Asia-Pacific, Europe, the Middle East, North America and South America), one reported prevalence and none reported incidence. Birth prevalence per 100,000 live births ranged from 0.16 (Japan) to 1.85 (Portugal). In the three European studies with estimates stratified by clinical subtypes, birth prevalence was highest for late-infantile cases (0.31-1.12 per 100,000 live births). The distribution of clinical subtypes reported in cases diagnosed over various time periods in 17 studies varied substantially, but late-infantile and juvenile MLD accounted for at least two-thirds of cases in most studies.
CONCLUSIONS
This review provides a foundation for further analysis of the regional epidemiology of MLD. Data gaps indicate the need for better global coverage, increased use of epidemiological measures (e.g. prevalence estimates) and more stratification of outcomes by clinical and genetic disease subtype.
Topics: Adult; Humans; Cerebroside-Sulfatase; Europe; Leukodystrophy, Metachromatic; Lysosomal Storage Diseases; Prevalence
PubMed: 38383398
DOI: 10.1186/s13023-024-03044-w -
Health Technology Assessment... Jan 2024Up to 30% of children have constipation at some stage in their life. Although often short-lived, in one-third of children it progresses to chronic functional...
BACKGROUND
Up to 30% of children have constipation at some stage in their life. Although often short-lived, in one-third of children it progresses to chronic functional constipation, potentially with overflow incontinence. Optimal management strategies remain unclear.
OBJECTIVE
To determine the most effective interventions, and combinations and sequences of interventions, for childhood chronic functional constipation, and understand how they can best be implemented.
METHODS
Key stakeholders, comprising two parents of children with chronic functional constipation, two adults who experienced childhood chronic functional constipation and four health professional/continence experts, contributed throughout the research. We conducted pragmatic mixed-method reviews. For all reviews, included studies focused on any interventions/strategies, delivered in any setting, to improve any outcomes in children (0-18 years) with a clinical diagnosis of chronic functional constipation (excluding studies of diagnosis/assessment) included. Dual reviewers applied inclusion criteria and assessed risk of bias. One reviewer extracted data, checked by a second reviewer. We systematically searched electronic databases (including Medical Literature Analysis and Retrieval System Online, Excerpta Medica Database, Cumulative Index to Nursing and Allied Health Literature) (January 2011 to March 2020) and grey literature, including studies (any design) reporting any intervention/strategy. Data were coded, tabulated and mapped. Research quality was not evaluated. For each different intervention, we included existing systematic reviews judged to be low risk of bias (using the Risk of Bias Assessment Tool for Systematic Reviews), updating any meta-analyses with new randomised controlled trials. Where there was no existing low risk of bias systematic reviews, we included randomised controlled trials and other primary studies. The risk of bias was judged using design-specific tools. Evidence was synthesised narratively, and a process of considered judgement was used to judge certainty in the evidence as high, moderate, low, very low or insufficient evidence. Included studies (any design, English-language) detailed intervention-related costs. Studies were categorised as cost-consequence, cost-effectiveness, cost-utility or cost-benefit, and reporting quality evaluated using the consensus health economic criteria checklist. Included studies reported data relating to implementation barriers or facilitators. Using a best-fit framework synthesis approach, factors were synthesised around the consolidated framework for implementation research domains.
RESULTS
Stakeholders prioritised outcomes, developed a model which informed evidence synthesis and identified evidence gaps.
SCOPING REVIEW
651 studies, including 190 randomised controlled trials and 236 primary studies, conservatively reported 48 interventions/intervention combinations.
EFFECTIVENESS SYSTEMATIC REVIEWS
studies explored service delivery models ( = 15); interventions delivered by families/carers ( = 32), wider children's workforce ( = 21), continence teams ( = 31) and specialist consultant-led teams ( = 42); complementary therapies ( = 15); and psychosocial interventions ( = 4). One intervention (probiotics) had moderate-quality evidence; all others had low to very-low-quality evidence. Thirty-one studies reported evidence relating to cost or resource use; data were insufficient to support generalisable conclusions. One hundred and six studies described implementation barriers and facilitators.
CONCLUSIONS
Management of childhood chronic functional constipation is complex. The available evidence remains limited, with small, poorly conducted and reported studies. Many evidence gaps were identified. Treatment recommendations within current clinical guidelines remain largely unchanged, but there is a need for research to move away from considering effectiveness of single interventions. Clinical care and future studies must consider the individual characteristics of children.
STUDY REGISTRATION
This study is registered as PROSPERO CRD42019159008.
FUNDING
This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 128470) and is published in full in ; Vol. 28, No. 5. See the NIHR Funding and Awards website for further award information.
Topics: Child; Adult; Humans; Systematic Reviews as Topic; Health Personnel; Constipation
PubMed: 38343084
DOI: 10.3310/PLTR9622 -
International Journal of Medical... Apr 2024Emergency department overcrowding could be improved by upstream telephone triage. Emergency telephone triage aims at managing and orientating adequately patients as... (Review)
Review
OBJECTIVES
Emergency department overcrowding could be improved by upstream telephone triage. Emergency telephone triage aims at managing and orientating adequately patients as early as possible and distributing limited supply of staff and materials. This complex task could be improved with the use of Clinical decision support systems (CDSS). The aim of this scoping review was to identify literature gaps for the future development and evaluation of CDSS for Emergency telephone triage.
MATERIALS AND METHODS
We present here a scoping review of CDSS designed for emergency telephone triage, and compared them in terms of functional characteristics, technical design, health care implementation and methodologies used for evaluation, following the PRISMA-ScR guidelines.
RESULTS
Regarding design, 19 CDSS were retrieved: 12 were knowledge based CDSS (decisional algorithms built according to guidelines or clinical expertise) and 7 were data driven (statistical, machine learning, or deep learning models). Most of them aimed at assisting nurses or non-medical staff by providing patient orientation and/or severity/priority assessment. Eleven were implemented in real life, and only three were connected to the Electronic Health Record. Regarding evaluation, CDSS were assessed through various aspects: intrinsic characteristics, impact on clinical practice or user apprehension. Only one pragmatic trial and one randomized controlled trial were conducted.
CONCLUSION
This review highlights the potential of a hybrid system, user tailored, flexible, connected to the electronic health record, which could work with oral, video and digital data; and the need to evaluate CDSS on intrinsic characteristics and impact on clinical practice, iteratively at each distinct stage of the IT lifecycle.
Topics: Humans; Triage; Decision Support Systems, Clinical; Delivery of Health Care; Emergency Service, Hospital; Telephone
PubMed: 38290244
DOI: 10.1016/j.ijmedinf.2024.105347 -
Critical Reviews in Oncology/hematology Mar 2024Exercise has been shown to play an important role in managing chemotherapy-related side effects, preserving skeletal muscle mass, and attenuating decline in...
BACKGROUND
Exercise has been shown to play an important role in managing chemotherapy-related side effects, preserving skeletal muscle mass, and attenuating decline in cardiorespiratory fitness associated with chemotherapy treatment, however, the feasibility of how these exercise programs are being delivered has yet to be synthesized. The objective of this review was to measure the rates of recruitment, adherence, and retention to exercise programs delivered for cancer patients during chemotherapy.
METHODS
Relevant studies were identified through a search of MEDLINE, Cochrane, EMBASE and CINAHL databases from January 2002 to July 2022 using keywords relating to exercise interventions during chemotherapy. Title and abstract screening, full text review, data extraction, and quality assessment were all performed independently by two reviewers.
RESULTS
A total of 36 studies were included in the review. The mean recruitment rate for the included studies was 62.39% (SD = 19.40; range 25.7-95%). Travel was the most common reason for declining recruitment in these trials. Adherence rates ranged from 17-109%, however the definition of adherence varied greatly between studies. Mean retention rates for the exercise groups was 84.1% (SD = 12.7; range 50-100%), with chemotherapy side effects being the most common reason why participants dropped out of these trials.
CONCLUSION
Multiple challenges exist for cancer patients during chemotherapy and careful consideration needs to be given when designing an exercise program for this population. Future research should include public and patient involvement to ensure exercise programs are pragmatic and patient centred.
Topics: Humans; Exercise Therapy; Neoplasms
PubMed: 38272152
DOI: 10.1016/j.critrevonc.2024.104272 -
PloS One 2023Children with neurodevelopmental disorders such as attention-deficit hyperactivity disorder (ADHD), autism, developmental language disorder (DLD), intellectual... (Meta-Analysis)
Meta-Analysis
RATIONALE
Children with neurodevelopmental disorders such as attention-deficit hyperactivity disorder (ADHD), autism, developmental language disorder (DLD), intellectual disability (ID), and social (pragmatic) communication disorder (SPCD) experience difficulties with social functioning due to differences in their social, emotional and cognitive skills. Previous systematic reviews have focussed on specific aspects of social functioning rather than broader peer functioning and friendships.
OBJECTIVE
To systematically review and methodologically appraise the quality and effectiveness of existing intervention studies that measured friendship outcomes for children with ADHD, autism, DLD, ID, and SPCD.
METHOD
Following PRISMA guidelines, we searched five electronic databases: CINAHL, Embase, Eric, PsycINFO, and PubMed. Two independent researchers screened all abstracts and disagreements were discussed with a third researcher to reach consensus. The methodological quality of studies was assessed using the Cochrane Risk of Bias Tool for Randomised Trials.
RESULTS
Twelve studies involving 15 interventions were included. Studies included 683 children with a neurodevelopmental disorder and 190 typically-developing children and diagnosed with either autism or ADHD. Within-group meta-analysis showed that the pooled intervention effects for friendship across all interventions were small to moderate (z = 2.761, p = 0.006, g = 0.485). The pooled intervention effect between intervention and comparison groups was not significant (z = 1.206, p = 0.400, g = 0.215).
CONCLUSION
Findings provide evidence that some individual interventions are effective in improving social functioning and fostering more meaningful friendships between children with neurodevelopmental disorders and their peers. Effective interventions involved educators, targeted child characteristics known to moderate peer functioning, actively involved peers, and incorporated techniques to facilitate positive peer perceptions and strategies to support peers. Future research should evaluate the effectiveness of friendship interventions for children with DLD, ID and SPCD, more comprehensively assess peer functioning, include child self-report measures of friendship, and longitudinally evaluate downstream effects on friendship.
Topics: Child; Humans; Friends; Attention Deficit Disorder with Hyperactivity; Peer Group; Social Adjustment; Neurodevelopmental Disorders
PubMed: 38096327
DOI: 10.1371/journal.pone.0295917 -
PloS One 2023Consideration for patients with visual impairment, from low vision to blindness, is an important part of building a barrier-free society. Some authors have elaborated... (Meta-Analysis)
Meta-Analysis
BACKGROUND
Consideration for patients with visual impairment, from low vision to blindness, is an important part of building a barrier-free society. Some authors have elaborated that visual impairment can indeed lead to delayed development in theory of mind, thereby causing pragmatic knowledge deficiency. Verifying whether those with eye conditions have pragmatic impairment is an essential way for their clinical evaluation, intervention and rehabilitation.
OBJECTIVE
We primarily carry out a meta-analysis of visual impairment from low vision to blindness and pragmatic impairment in people with low vision or blindness to verify visual impairment may cause pragmatic impairment.
DATA SOURCES
Electronic databases Pubmed, Medline, MesH, Psychinfo, Ovid, EBSCO and CNKI and the reference sections of previous reviews.
STUDY ELIGIBILITY CRITERIA
Studies were included when they built on primary data from clinical questionnaire surveys or field trials anywhere in the world, and when they reported impacts of visual impairment on social cognition, communication, skills, behavior and intelligence. In total, 25 original studies were included, in which 25735 people were evaluated.
RESULTS
Statistically, visual impairments and pragmatic impairment exist correlation due to the significant p value(p = 0.0005 < 0.05) in group and the subgroup sorted in the light of 18 years old (p < 0.0001 and p = 0.003 < 0.05). Psychologically, because people with visual impairment can not normally get non-verbal information, they can not get a complete pragmatic knowledge system. Pragmatic knowledge deficiency leads to abnormal in executive functions and development delay from the perspective of theory of mind, inducing pragmatic impairment. Therefore, visual impairment has an impact on pragmatic impairment.
CONCLUSION
The meta-analysis reveals robust evidence on the relationship of vision impairment and pragmatic impairment in children or adults. Such evidence may help to gradually improve the clinical evaluation, intervention and rehabilitation of these people.
Topics: Child; Humans; Adolescent; Vision, Low; Intelligence; Blindness
PubMed: 38064440
DOI: 10.1371/journal.pone.0294326 -
Exploration of Targeted Anti-tumor... 2023Coronavirus disease 2019 (COVID-19) became pandemic on 11th March 2020 and it deeply stressed the healthcare system. Cancer patients represent a vulnerable population,...
AIM
Coronavirus disease 2019 (COVID-19) became pandemic on 11th March 2020 and it deeply stressed the healthcare system. Cancer patients represent a vulnerable population, so many recommendations have been approved to ensure optimal management. Clinical research was notably impacted by COVID too. This review aims to analyze the challenges occurred during a pandemic for the management of enrolled patients (enrollment, use of telemedicine visits, study procedures) and for the clinical trials system (from feasibility to selection visit, site initiation visit, monitorings, use of e-signature, deviations and discontinuations).
METHODS
The studies included in the present review were selected from PubMed/Google Scholar/ScienceDirect databases.
RESULTS
During the first phase of pandemic many clinical trials were suspended in accrual and, as the pandemic progressed, recommendations were established to guarantee the safety and the continuity of care of enrolled patients. In addition, lot of new strategies was found during the pandemic to reduce the negative consequences on clinical trial performance and to guarantee new opportunities of care in the respect of good clinical practice (GCP) in a bad scenario.
CONCLUSIONS
Among all modifiers, investigators would prefer to maintain the positive ones such as pragmatic and simplified trial designs and protocols, reducing in-person visits when not necessary and to minimizing sponsor and contract research organizations (CROs) visits.
PubMed: 38023994
DOI: 10.37349/etat.2023.00183