Dataset Information

Development of a core outcome set for myelodysplastic syndromes - a Delphi study from the EUMDS Registry Group.

ABSTRACT: Treatment options for myelodysplastic syndromes (MDS) vary widely, depending on the natural disease course and patient-related factors. Comparison of treatment effectiveness is challenging as different endpoints have been included in clinical trials and outcome reporting. Our goal was to develop the first MDS core outcome set (MDS-COS) defining a minimum set of outcomes that should be reported in future clinical studies. We performed a comprehensive systematic literature review among MDS studies to extract patient- and/or clinically relevant outcomes. Clinical experts from the European LeukemiaNet MDS (EUMDS) identified 26 potential MDS core outcomes and participated in a three-round Delphi survey. After the first survey (56 experts), 15 outcomes met the inclusion criteria and one additional outcome was included. The second round (38 experts) resulted in six included outcomes. In the third round, a final check on plausibility and practicality of the six included outcomes and their definitions was performed. The final MDS-COS includes: health-related quality of life, treatment-related mortality, overall survival, performance status, safety, and haematological improvement. This newly developed MDS-COS represents the first minimum set of outcomes aiming to enhance comparability across future MDS studies and facilitate a better understanding of treatment effectiveness.

SUBMITTER: Rochau U

PROVIDER: S-EPMC8221029 | biostudies-literature | 2020 Nov

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Development of a core outcome set for myelodysplastic syndromes - a Delphi study from the EUMDS Registry Group.

Rochau Ursula U Stojkov Igor I Conrads-Frank Annette A Borba Helena H HH Koinig Karin A KA Arvandi Marjan M van Marrewijk Corine C Garelius Hege H Germing Ulrich U Symeonidis Argiris A Sanz Guillermo F GF Fenaux Pierre P de Witte Theo T Efficace Fabio F Siebert Uwe U Stauder Reinhard R

British journal of haematology 20200514 3

Treatment options for myelodysplastic syndromes (MDS) vary widely, depending on the natural disease course and patient-related factors. Comparison of treatment effectiveness is challenging as different endpoints have been included in clinical trials and outcome reporting. Our goal was to develop the first MDS core outcome set (MDS-COS) defining a minimum set of outcomes that should be reported in future clinical studies. We performed a comprehensive systematic literature review among MDS studies ...[more]

PMID: 32410281

Similar Datasets

Project description:BackgroundA Core Outcome Set (COS) is an agreed list of outcomes that are measured and reported in all clinical trials for a particular health condition. An 'e-Delphi' is an increasingly popular method for developing a COS whereby stakeholders are consulted via a multi-round online survey to reach agreement regarding the most important outcomes. Many COS studies seek diverse, international input that includes professionals and healthcare users. However, the recruitment and retention of participants can be deterred by various factors (e.g. language barriers and iterative, time-consuming rounds). This report evaluates the effectiveness of recruitment and retention methods used in the Core Outcome Measures in Tinnitus International Delphi (COMiT'ID) study using participant feedback from healthcare users, healthcare practitioners, researchers, commercial representatives and funders.MethodsA range of methods were applied to recruit participants to the study and maintain engagement over the three rounds. Feedback on recruitment and retention methods was collected using a twenty-item online questionnaire, with free text comments.ResultsA personalised email invitation was the most frequent recruitment route, and 719 professionals and healthcare users consented to take part. Retention of each stakeholder group ranged from 76 to 91% completing all three e-Delphi rounds. Feedback was given by 379 respondents. A majority of respondents were satisfied with the study methods that were implemented to promote retention. Over 55% indicated that their overall experience closely matched their expectations at the start of the study, and over 90% felt that their contribution was appreciated.ConclusionsThis report highlights study methods that worked well with respect to recruitment and retention, and those that did not. Findings provide a unique contribution to the growing evidence base of good practice in COS development by demonstrating the relative effectiveness of recruitment and retention methods for an e-Delphi survey.Trial registrationThis project was registered (November 2014) in the database of the Core Outcome Measures in Effectiveness Trials (COMET) initiative. The protocol is published in Trials (doi:10.1186/s13063-017-2123-0).

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.

Project description:Facial aging is a concern for many patients. Wrinkles, loss of volume, and discoloration are common physical manifestations of aging skin. Genetic heritage, prior ultraviolet light exposure, and Fitzpatrick skin type may be associated with the rate and type of facial aging. Although many clinical trials assess the correlates of skin aging, there is heterogeneity in the outcomes assessed, which limits the quality of evaluation and comparison of treatment modalities. To address the inconsistency in outcomes, in this project we will develop a core set of outcomes that are to be evaluated in all clinical trials relevant to facial aging.A long list of measureable outcomes will be created from four sources: (1) systematic medical literature review, (2) patient interviews, (3) other published sources, and (4) stakeholder involvement. Two rounds of Delphi processes with homogeneous groups of physicians and patients will be performed to prioritize and condense the list. At a consensus meeting attended by physicians, patients, and stakeholders, outcomes will be further condensed on the basis of participant scores. By the end of the meeting, members will vote and decide on a final recommended set of core outcomes. Subsequent to this, specific measures will be selected or created to assess these outcomes.The aim of this study is to develop a core outcome set and relevant measures for clinical trials relevant to facial aging. We hope to improve the reliability and consistency of outcome reporting of skin aging, thereby enabling improved evaluation of treatment efficacy and patient satisfaction.Core Outcome Measures in Effectiveness Trials (COMET) Initiative, accessible at http://www.comet-initiative.org/studies/details/737 . Core Outcomes Set Initiative, (CSG-COUSIN) accessible at https://www.uniklinikum-dresden.de/de/das-klinikum/universitaetscentren/zegv/cousin/meet-the-teams/project-groups/core-outcome-set-for-the-appearance-of-facial-aging . Protocol version date is 28 July 2016.

Project description:BackgroundTension-type headache (TTH) is the most common type of headache and the second most common health-related complaint among children and adults. Traditional Chinese medicine (TCM) offers unique therapeutic benefits in treating TTH. However, the lack of standardized evidence-such as inconsistencies in outcome selection and reporting in clinical studies, a lack of consensus on outcomes and measures, high risks of selective reporting bias, and missing data-has limited the development of robust evidence supporting the efficacy of TCM in treating TTH. Therefore, establishing a core outcome set (COS) is crucial for standardizing TCM clinical studies for TTH, thereby enhancing the quality and comparability of research findings.ObjectiveThis study aims to develop a COS for future clinical studies on the treatment of TTH with TCM.MethodsThe COS will be developed through the following 3 stages. First, systematic reviews and semistructured interviews will be conducted to identify potential essential outcomes, which will be evaluated by the steering committee to finalize a preliminary list of outcomes. Data will be processed using thematic analysis to ensure comprehensive coverage of relevant outcomes. Second, a 2-round Delphi survey will be conducted, inviting stakeholders, including health care experts and patients with tension-type headaches, to determine the importance of each outcome. Statistical analysis will be used to assess the level of consensus and prioritize outcomes based on predefined criteria. Third, a face-to-face consensus meeting will be held to finalize the COS and recommend measurement times for each outcome. Key outcomes will be interpreted based on their clinical relevance and feasibility of measurement, ensuring the COS is comprehensive and applicable in clinical settings.ResultsThe protocol has been registered in PROSPERO, with the review commencing on October 1, 2024, and anticipated results by November 15, 2024. The systematic reviews will be finalized, followed by the Delphi survey and consensus conference in late 2024 and early 2025. The COS findings will be reported per COS-STAR (Core Outcome Set-STAndards for Reporting) guidelines, published in an international journal, presented at conferences, and disseminated to participants for clinical application.ConclusionsThis study is necessary as developing a COS for future TCM clinical studies in the treatment of TTH can maximize the value of data from individual trials and provide high-quality research evidence.Trial registrationCore Outcome Measures in Effectiveness Trials Initiative 1473; https://tinyurl.com/3ts62s2p.International registered report identifier (irrid)PRR1-10.2196/63481.

Dataset Information

Development of a core outcome set for myelodysplastic syndromes - a Delphi study from the EUMDS Registry Group.

Publications

Development of a core outcome set for myelodysplastic syndromes - a Delphi study from the EUMDS Registry Group.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets