Development of a core outcome set for congenital pulmonary airway malformations: study protocol of an international Delphi survey.
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ABSTRACT: Introduction
A worldwide lack of consensus exists on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM) even though the incidence is increasing. Either a surgical resection is performed or a wait-and-see policy is employed, depending on the treating physician. Management is largely based on expert opinion and scientific evidence is scarce. Wide variations in outcome measures are seen between studies making comparison difficult thus highlighting the lack of universal consensus in outcome measures as well. We aim to define a core outcome set which will include the most important core outcome parameters for paediatric patients with an asymptomatic CPAM.Methods and analysis
This study will include a critical appraisal of the current literature followed by a three-stage Delphi process with two stakeholder groups. One surgical group including paediatric as well as thoracic surgeons, and a non-surgeon group including paediatric pulmonologists, intensive care and neonatal specialists. All participants will score outcome parameters according to their level of importance and the most important parameters will be determined by consensus.Ethics and dissemination
Electronic informed consent will be obtained from all participants. Ethical approval is not required. After the core outcome set has been defined, we intend to design an international randomised controlled trial: the COllaborative Neonatal NEtwork for the first CPAM Trial, which will be aimed at determining the optimal management of patients with asymptomatic CPAM.
SUBMITTER: Hermelijn S
PROVIDER: S-EPMC8047990 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:ObjectivesA core outcome set (COS) describes a minimum set of outcomes to be reported by all clinical trials of one healthcare condition. Delphi surveys are frequently used to achieve consensus on core outcomes. International input is important to achieve global COS uptake. We aimed to investigate participant representation in international Delphi surveys, with reference to the inclusion of patients and participants from low and middle income countries as stakeholders (LMICs).DesignSystematic review.Data sourcesEMBASE, Medline, Web of Science, COMET database and hand-searching.Eligibility criteriaProtocols and studies describing Delphi surveys used to develop an international COS for trial reporting, published between 1 January 2017 and 6 June 2019.Data extraction and synthesisDelphi participants were grouped as patients or healthcare professionals (HCPs). Participants were considered international if their country of origin was different to that of the first or senior author. Data extraction included participant numbers, country of origin, country income group and whether Delphi surveys were translated. We analysed the impact these factors had on outcome prioritisation.ResultsOf 90 included studies, 69% (n=62) were completed and 31% (n=28) were protocols. Studies recruited more HCPs than patients (median 60 (IQR 30-113) vs 30 (IQR 14-66) participants, respectively). A higher percentage of HCPs was international compared with patients (57% (IQR 37-78) vs 20% (IQR 0-68)). Only 31% (n=28) studies recruited participants from LMICs. Regarding recruitment from LMICs, patients were under-represented (16% studies; n=8) compared with HCPs (22%; n=28). Few (7%; n=6) studies translated Delphi surveys. Only 3% studies (n=3) analysed Delphi responses by geographical location; all found differences in outcome prioritisation.ConclusionsThere is a disproportionately lower inclusion of international patients, compared with HCPs, in COS-development Delphi surveys, particularly within LMICs. Future international Delphi surveys should consider exploring for geographical and income-based differences in outcome prioritisation.Prospero registration numberCRD42019138519.
| S-EPMC7684826 | biostudies-literature
Project description:IntroductionAppendicitis is a global disease affecting roughly 1 in every 12 people in the world, with the highest incidence between ages 10 and 19 years. To date, a wide variety of health outcomes have been reported in randomised controlled trials and meta-analyses evaluating treatments for appendicitis. This is especially the case in studies comparing non-operative treatment with operative treatment. A set of standard outcomes, to be reported in all future trials, is needed to allow for adequate comparison and interpretation of clinical trial results and to make data pooling possible. This protocol describes the development of such a global core outcome set (COS) to allow unified reporting of treatment interventions in children with acute uncomplicated appendicitis.Methods and analysisWe use current international standard methodology for the development and reporting of this COS. Its development consists of three phases: (1) an update of the most recent systematic review on outcomes reported in uncomplicated paediatric appendicitis research to identify additional outcomes, (2) a three-step global Delphi study to identify a set of core outcomes for which there is consensus between parents and (paediatric) surgeons and (3) an expert meeting to finalise the COS and its definitions. Children and young people will be involved through their parents during phase 2 and will be engaged directly using a customised face-to-face approach.Ethics and disseminationThe medical research ethics committee of the Academic Medical Center Amsterdam has approved the study. Each participating country/research group will ascertain ethics board approval. Electronic informed consent will be obtained from all participants. Results will be presented in peer-reviewed academic journals and at (international) conferences.Trial registration numberCOMET registration: 1119.
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Project description:PURPOSE:Cryptoglandular anal fistula is a disorder with an incidence of around 1 per 5,000 people per year in European countries. Many studies have been conducted to evaluate the effectiveness of interventions for anal fistula. However, there is considerable heterogeneity in the outcomes assessed and reported in these studies. This limits research quality and complicates evidence synthesis. A solution for heterogeneity in outcome reporting is the development of a Core Outcome Set (COS). This paper describes the protocol for the development of a European COS for Anal Fistula (AFCOS). METHODS:The first step will be a systematic review of the literature to identify potential outcomes that may be included in the COS. Patient interviews will be conducted in The United Kingdom and The Netherlands to ensure that both clinician-important and patient-important outcomes are captured. The outcomes will be categorized using the COMET taxonomy and taken forward to a Delphi consensus exercise. In up to three web-based Delphi surveys the outcomes will be prioritized by patients, clinicians (surgeons, gastroenterologists, and radiologists), and (clinical) researchers. The responses will be summarized and reported anonymously in subsequent round(s) facilitating convergence to a consensus opinion. The final COS will be decided during a face-to-face consensus meeting with patients, clinicians, and (clinical) researchers. DISCUSSION:This study protocol describes the development of a European COS for anal fistula to improve research quality, evidence synthesis, and patient care.
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Project description:PurposeOver the last decade, many studies were performed regarding treatment options for hemorrhoidal disease. Randomised controlled trials (RCTs) should have well-defined primary and secondary outcomes. However, the reported outcome measures are numerous and diverse. The heterogeneity of outcome definition in clinical trials limits transparency and paves the way for bias. The development of a core outcome set (COS) helps minimizing this problem. A COS is an agreed minimum set of outcomes that should be measured and reported in all clinical trials of a specific disease. The aim of this project is to generate a COS regarding the outcome of treatment after hemorrhoidal disease.MethodsA Delphi study will be performed by an international steering group healthcare professionals and patients with the intention to create a standard outcome set for future clinical trials for the treatment of hemorrhoidal disease. First, a literature review will be conducted to establish which outcomes are used in clinical trials for hemorrhoidal disease. Secondly, both healthcare professionals and patients will participate in several consecutive rounds of online questionnaires and a face-to-face meeting to refine the content of the COS.DiscussionDevelopment of a COS for hemorrhoidal disease defines a minimum outcome-reporting standard and will improve the quality of research in the future.
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Project description:To investigate the differential expression of genes in whole transcripts of congenital pulmonary airway malformation (CPAM) using second-generation sequencing (also known as next-generation sequencing, NGS) technology. Children with CPAM were strictly screened after setting the criteria, and grouped by taking CPAM parietal tissue and CPAM lesion tissue respectively, and RNA-Seq libraries were established separately using second-generation sequencing technology, followed by differential expression analysis and GO (gene ontology) functional enrichment analysis, KEGG (Kyoto encyclopedia of genes and genomes, a database) pathway analysis and GSEA (Gene Set Enrichment Analysis) analysis. Five cases were screened from 36 children with CPAM, and high-throughput sequencing was performed to obtain 10 whole transcripts of samples with acceptable sequence quality and balanced gene coverage. One aberrantly expressed sample (3b) was found by analysis of principal components, which was excluded and then subjected to differential expression analysis, and 860 up-regulated genes and 203 down-regulated genes. GO functional enrichment analysis of differentially expressed genes demonstrates the functional class and cellular localization of target genes. The whole transcript of CPAM shows obvious gene up and down-regulation, differentially expressed genes are located in specific cells and belong to different functional categories, and NGS can provide an effective means to study the transcriptional regulation of CPAM from the overall transcriptional level.
| S-EPMC9705386 | biostudies-literature
Project description:Shared decision-making about clinical care options in end-stage kidney disease is limited by inconsistencies in the reporting of outcomes and the omission of patient-important outcomes in trials. Here we generated a consensus-based prioritized list of outcomes to be reported during trials in peritoneal dialysis (PD). In an international, online, three-round Delphi survey, patients/caregivers and health professionals rated the importance of outcomes using a 9-point Likert scale (with 7-9 indicating critical importance) and provided comments. Using a Best-Worst Scale (BWS), the relative importance of outcomes was estimated. Comments were analyzed thematically. In total, 873 participants (207 patients/caregivers and 666 health professionals) from 68 countries completed round one, 629 completed round two and 530 completed round three. The top outcomes were PD-related infection, membrane function, peritoneal dialysis failure, cardiovascular disease, death, catheter complications, and the ability to do usual activities. Compared with health professionals, patients/caregivers gave higher priority to six outcomes: blood pressure (mean difference, 0.4), fatigue (0.3), membrane function (0.3), impact on family/friends (0.1), peritoneal thickening (0.1) and usual activities (0.1). Four themes were identified that underpinned the reasons for ratings: contributing to treatment longevity, preserving quality of life, escalating morbidity, and irrelevant and futile information and treatment. Patients/caregivers and health professionals gave highest priority to clinical outcomes. In contrast to health professionals, patients/caregivers gave higher priority to lifestyle-related outcomes including the impact on family/friends and usual activities. Thus, prioritization will inform a core outcome set to improve the consistency and relevance of outcomes for trials in PD.
| S-EPMC6961705 | biostudies-literature
Project description:BackgroundSingle-sided deafness (SSD) describes the presence of a unilateral severe to profound sensorineural hearing loss. SSD disrupts spatial hearing and understanding speech in background noise. It has functional, psychological and social consequences. Potential options for rehabilitation include hearing aids and auditory implants. Benefits and harms of these interventions are documented inconsistently in the literature, using a variety of outcomes ranging from tests of speech perception to quality of life questionnaires. It is therefore difficult to compare interventions when rehabilitating SSD. The Core Rehabilitation Outcome Set for Single Sided Deafness (CROSSSD) study is an international initiative that aims to develop a minimum set of core outcomes for use in future trials of SSD interventions.Methods/designThe CROSSSD study adopts an international two-round online modified Delphi survey followed by a stakeholder consensus meeting to identify a patient-centred core outcome domain set for SSD based on what is considered critical and important for assessing whether an intervention for SSD has worked.DiscussionThe resulting core outcome domain set will act as a minimum standard for reporting in future clinical trials and could have further applications in guiding the use of outcome measures in clinical practice. Standardisation will facilitate comparison of research findings.
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Project description:BackgroundThere is limited evidence on the best available treatment options for capillary malformations (CMs), mainly due to the absence of uniform outcome measures in trials on therapies. A core outcome set (COS) enables standard reporting of trial outcomes, which facilitates comparison of treatment results.ObjectivesTo develop a core outcome domain set (CDS), as part of a core outcome set (COS), for clinical research on CMs.MethodsSixty-seven potentially relevant outcome subdomains were recognized based on the literature, focus group sessions, and input from the COSCAM working group. These outcome subdomains were presented in an online Delphi study to CM experts (medical specialists and authors of relevant literature) and (parents of) patients with CM (international patient associations). During three e-Delphi study rounds, the participants repeatedly scored the importance of these outcome subdomains on a seven-point Likert scale. Participants could also propose other relevant outcome subdomains. Consensus was defined as ≥ 80% agreement as to the importance of an outcome subdomain among both stakeholder groups. The CDS was finalized during an online consensus meeting.ResultsIn total 269 participants from 45 countries participated in the first e-Delphi study round. Of these, 106 were CM experts from 32 countries, made up predominantly of dermatologists (59%) and plastic surgeons (18%). Moreover, 163 (parents of) patients with CM from 28 countries participated, of whom 58% had Sturge-Weber syndrome. During the two subsequent e-Delphi study rounds, 189 and 148 participants participated, respectively. After the entire consensus process, consensus was reached on 11 outcome subdomains: colour/redness, thickness, noticeability, distortion of anatomical structures, glaucoma, overall health-related quality of life, emotional functioning, social functioning, tolerability of intervention, patient satisfaction with treatment results, and recurrence.ConclusionsWe recommend the CDS to be used as a minimum reporting standard in all future trials of CM therapy. Our next step will be to select suitable outcome measurement instruments to score the core outcome subdomains. What is already known about this topic? Besides physical and functional sequelae, capillary malformations (CMs) often cause emotional and social burden. The lack of uniform outcome measures obstructs proper evaluation and comparison of treatment strategies. As a result, there is limited evidence on the best available treatment options. The development of a core outcome set (COS) may improve standardized reporting of trial outcomes. What does this study add? A core outcome domain set (CDS), as part of a COS, was developed for clinical research on CMs. International consensus was reached on the recommended core outcome subdomains to be measured in CM trials: colour/redness, thickness, noticeability, distortion of anatomical structures, glaucoma, overall health-related quality of life, emotional functioning, social functioning, tolerability of intervention, patient satisfaction with treatment results, and recurrence. This CDS enables the next step in the development of a COS, namely to reach consensus on the core outcome measurement instruments to score the core outcome subdomains. What are the clinical implications of this work? The obtained CDS will facilitate standardized reporting of treatment outcomes, thereby enabling proper comparison of treatment results. This comparison is likely to provide more reliable information for patients about the best available treatment options.
| S-EPMC9796083 | biostudies-literature
Project description:BackgroundInternational stakeholder participation is important in the development of core outcome sets (COS). Stakeholders from varying regions may value health outcomes differently. Here, we explore how region, health income and participant characteristics influence prioritisation of outcomes during development of a COS for gastric cancer surgery trials (the GASTROS study).Methods952 participants from 55 countries participating in a Delphi survey during COS development were eligible for inclusion. Recruits were grouped according to region (East or West), country income classification (high and low-to-middle income) and other characteristics (e.g. patients; age, sex, time since surgery, mode of treatment, surgical approach and healthcare professionals; clinical experience). Groups were compared with respect to how they categorised 56 outcomes identified as potentially important to include in the final COS ('consensus in', 'consensus out', 'no consensus'). Outcomes categorised as 'consensus in' or 'consensus out' by all 3 stakeholder groups would be automatically included in or excluded from the COS respectively.ResultsIn total, 13 outcomes were categorised 'consensus in' (disease-free survival, disease-specific survival, surgery-related death, recurrence of cancer, completeness of tumour removal, overall quality of life, nutritional effects, all-cause complications, intraoperative complications, anaesthetic complications, anastomotic complications, multiple organ failure, and bleeding), 13 'consensus out' and 31 'no consensus'. There was little variation in prioritisation of outcomes by stakeholders from Eastern or Western countries and high or low-to-middle income countries. There was little variation in outcome prioritisation within either health professional or patient groups.ConclusionOur study suggests that there is little variation in opinion within stakeholder groups when participant region and other characteristics are considered. This finding may help COS developers when designing their Delphi surveys and recruitment strategies. Further work across other clinical fields is needed before broad recommendations can be made.
| S-EPMC8719722 | biostudies-literature
Project description:BACKGROUND:There are numerous challenges in including patients in a core outcome set (COS) study, these can vary depending on the patient group. This study describes current efforts to include patients in the development of COS, with the aim of identifying areas for further improvement and study. METHODS:Using the COMET database, corresponding authors of COS projects registered or published from 1 January 2013 to 2 February 2017 were invited via a personalised email to participate in a short online survey. The survey and emails were constructed to maximise the response rate by following the academic literature on enhancing survey responses. Personalised reminder emails were sent to non-responders. This survey explored the frequency of patient input in COS studies, who was involved, what methods were used and whether or not the COS development was international. RESULTS:One hundred and ninety-two COS developers were sent the survey. Responses were collected from 21 February 2017 until 7 May 2017. One hundred and forty-six unique developers responded, yielding a 76% response rate and data in relation to 195 unique COSs (as some developers had worked on multiple COSs). Of focus here are their responses regarding 162 COSs at the published, completed or ongoing stages of development. Inclusion of patient participants was indicated in 87% (141/162) of COSs in the published completed or ongoing stages and over 94% (65/69) of ongoing COS projects. Nearly half (65/135) of COSs included patient participants from two or more countries and 22% (30/135) included patient participants from five or more countries. The Delphi survey was reported as being used singularly or in combination with other methods in 85% (119/140) of projects. Almost a quarter (16/65) of ongoing studies reported using a combination of qualitative interviews, Delphi survey and consensus meeting. CONCLUSIONS:These findings indicated that the Delphi survey is the most popular method of facilitating patient participation, while the combination of qualitative interviews, Delphi survey and consensus meetings is the most popular combination of methods. The increased inclusion of patient participants in the development of COSs is encouraging, as is the international approach to COS development that some developers are adopting.
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