Cross-Cultural Adaptation and Psychometric Validation of the Dutch Version of the Core Outcome Measures Index for the Neck in Patients Undergoing Surgery for Degenerative Disease of the Cervical Spine

Objective: To perform the psychometric validation of the Dutch version of the Core Outcome Measures Index (COMI) for the neck.Methods: A total of 178 patients, who had an indication for surgery due to degenerative cervical spinal disease, were enrolled in the study. They filled in a baseline booklet containing the Dutch version of the COMI-neck, Likert-scales for neck and arm/shoulder pain, the Neck Disability Index (NDI), the EuroQol-5 dimensions (EQ-5D) and the 12-item Short Form health survey (SF-12). Aside from analyzing construct validity using the Spearman correlation test, test-retest reliability, and responsiveness at 3 months were assessed using the intraclass correlation coefficient (ICC) and the receiver-operating characteristic (ROC) curve, respectively.Results: The COMI-neck showed good acceptability with missing data ranging from 0% to 4.5% and some floor/ceiling effects for 3 of the domains at baseline. The COMI-summary score showed good to very good correlation with the EQ5D (ρ = -0.43), the physical component summary of the SF-12 (ρ = -0.47) and the NDI (ρ = 0.73). Individual domains showed correlations of -0.28 to 0.85 with the reference questionnaires. Test-retest reliability analysis showed an ICC of 0.91 with a minimal detectable change of 1.7. Responsiveness analysis of the COMI-neck showed an area under 0.79 under the ROC-curve. The standardized response mean for a good outcome was 1.24 and for a poor outcome 0.37.Conclusion: The current study shows that the Dutch version of the COMI-neck is a valid, reliable and responsive Patient-Reported Outcome Measure, among patients undergoing surgery for degenerative cervical spinal disorders.

Automated Generation of Core Outcome Measures for Clinical Trials

Common outcome sets are vital for ensuring usability of clinical trial results and enabling inter-study comparisons. The task of identifying clinical outcomes for a particular field is cumbersome and time-consuming. The aim of this work was to develop an automated pipeline for identifying common outcomes by analyzing outcomes from relevant trials reported at ClinicalTrials.gov and to assess the pipeline accuracy. We validated the output of our pipeline by comparing the outcomes it identified for acute coronary syndromes and coronary artery disease with the set of outcomes recommended for these conditions by a panel of experts in a widely cited report. We found that our pipeline identified the same or similar outcomes for 100% of the outcomes recommended in the experts’ report. The coverage of the pipeline’s results dropped only slightly (to 21 out of 23 outcome domains, 91%) when we restricted the pipeline to trials posted before the publication of the report, indicating a great potential for this pipeline to be used in aiding and informing the future development of core outcome measures in clinical trials.

Novel method to select meaningful outcomes for evaluation in clinical trials

BackgroundA standardised framework for selecting outcomes for evaluation in trials has been proposed by the Core Outcome Measures in Effectiveness Trials working group. However, this method does not specify how to ensure that the outcomes that are selected are causally related to the disease and the health intervention being studied. Causal network diagrams may help researchers identify outcomes that are both clinically meaningful and likely to be causally dependent on the intervention, and endpoints that are, in turn, causally dependent on those outcomes. We aimed to (1) develop a generalisable method for selecting outcomes and endpoints in trials and (2) apply this method to select outcomes for evaluation in a trial investigating treatment strategies for pulmonary exacerbations of cystic fibrosis (CF).MethodsWe conducted a series of online surveys and workshops among people affected by CF. We used a modified Delphi approach to develop a consensus list of important outcomes. A workshop involving domain experts elicited how these outcomes were causally related to the underlying pathophysiological processes. Meaningful outcomes were prioritised based on the extent to which each outcome captured separate rather than common aspects of the underlying pathophysiological process.ResultsThe 10 prioritised outcomes were: breathing difficulty/pain, sputum production/clearance, fatigue, appetite, pain (not related to breathing), motivation/demoralisation, fevers/night sweats, treatment burden, inability to meet personal goals and avoidance of gastrointestinal symptoms.ConclusionsThis proposed method for selecting meaningful outcomes for evaluation in clinical trials may improve the value of research as a basis for clinical decisions.

Study protocol to develop a core outcome set for thyroid dysfunction to bridge the unmet needs of patient-centred care

IntroductionThyroid dysfunctions (TD) are common medical conditions affecting all global populations. Improved healthcare leading to increasing survival rates and delayed diagnosis rendered significant burden of the disease in the increasing number of patients with TD with comorbid illnesses. Therefore, reducing the burden of TD and improving the quality of care are crucial. Existing poor-quality data that guide evidence-based decisions only provide a fragmented picture of clinical care. The different outcomes across studies assessing the effectiveness of treatments impede our ability to synthesise results for determining the most efficient treatments. This project aims to produce a core outcome set (COS), which embeds the multiple complex dimensions of routine clinical care for the effectiveness studies and clinical care of adult patients with TD.Methods and analysisThis mixed-method project has two phases. In phase 1, we will identify a list of patient-reported and clinical outcomes through qualitative research and systematic reviews. In phase 2, we will categorise the identified outcomes using the Core Outcome Measures in Effectiveness Trials taxonomy of core domains and the International Classification of Functioning, Disability and Health. We will develop questionnaires from the list of outcomes identified from each domain for the two-round online Delphi exercise, aiming to reach a consensus on the COS. The Delphi process will include patients, carers, researchers and healthcare participants. We will hold an online consensus meeting involving representatives of all key stakeholders to establish the final COS.Ethics and disseminationThe study has been reviewed and approved by the Ethics Committee for Research Involving Human Subjects, Universiti Putra Malaysia and the Research Ethics Committee, National University of Malaysia. This proposed COS in TD will improve the value of data, facilitate high-quality evidence synthesis and evidence-based decision-making. Furthermore, we will present the results to participants, in peer-reviewed academic journals and conferences.Registration detailsCore Outcome Measures in Effectiveness Trials (COMET) Initiative database registration: http://www.comet-initiative.org/studies/details/1371

Study protocol: a core outcome set for perinatal interventions for congenital diaphragmatic hernia

Background Congenital diaphragmatic hernia (CDH) is, depending of the severity, a birth defect associated with significant mortality and morbidity. Prenatal screening by ultrasound may detect this condition and comprehensive assessment of severity is possible, allowing for in utero referral to an experienced centre for planned delivery. In an effort to improve outcomes, prenatal interventions to stimulate lung development were proposed. Along the same lines, new postnatal management strategies are being developed. In order to enable proper comparison of novel perinatal interventions as well as outcomes, a set of uniform and relevant outcome measures is required. Core outcome sets (COS) are agreed, clearly defined sets of outcomes to be measured in a standardised manner and reported consistently. Herein we aim to describe the methodology we will use to define a COS for perinatal and neonatal outcomes of foetuses and newborns with congenital diaphragmatic hernia and to draft a dissemination and implementation plan. Methods We will use the methodology described in the Core Outcome Measures in Effectiveness Trials (COMET) Initiative Handbook. An international steering group will be created to guide the development of the COS. We are systematically reviewing the literature to identify all potential relevant pre- and neonatal outcomes previously used in studies on perinatal interventions for CDH. We will build a consensus on these core outcomes in a stakeholder group using the Delphi method. After completion, a stakeholder meeting will decide on a final COS, using a modified Nominal Group Technique. Thereafter, we will review potential definitions and measurements of these outcomes, and again a consensus meeting will be organised, to finalise the COS before dissemination. Discussion We have started a procedure to develop a COS for studies on perinatal interventions for congenital diaphragmatic hernia, with the purpose of improving the quality of research, guide clinical practice and improve patient care and eventual use in future clinical trials, systematic reviews and clinical practice guidelines. Trial registration We prospectively registered this study in the International Prospective Register of Systematic Reviews (PROSPERO) (registration number: CRD42019124399) and The Core Outcome Measures in Effectiveness Trials (COMET) Initiative (registration number:1296).