scholarly journals Outcome Measures Used in Clinical Trials for Behçet Syndrome: A Systematic Review

2014 ◽  
Vol 41 (3) ◽  
pp. 599-612 ◽  
Author(s):  
Gulen Hatemi ◽  
Peter A. Merkel ◽  
Vedat Hamuryudan ◽  
Maarten Boers ◽  
Haner Direskeneli ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Management Strategy ◽  
Unmet Need ◽  
Disease Assessment ◽  
Behçet Syndrome ◽  
Behcet Syndrome ◽  
Delphi Exercise ◽  
Significant Impairment

Behçet syndrome (BS) is a multisystem vasculitis that is most active during young adulthood, causing serious disability and significant impairment in quality of life. Differences in the disease course, severity, and organ involvement between patients, depending on the age at presentation and sex, makes it impossible to determine a single management strategy. The diversity and variability in the outcome measures used in clinical trials in BS makes it difficult to compare the results or inform physicians about the best management strategy for individual patients. There is a large unmet need to determine or develop validated outcome measures for use in clinical trials in BS that are acceptable to researchers and regulatory agencies. We conducted a systematic review to describe the outcomes and outcome measures that have been used in clinical trials in BS. This review revealed the diversity and variability in the outcomes and outcome measures and the lack of standard definitions for most outcomes and rarity of validated outcome tools for disease assessment in BS. This systematic literature review will identify domains and candidate instruments for use in a Delphi exercise, the next step in the development of a core set of outcome measures that are properly validated and widely accepted by the collaboration of researchers from many different regions of the world and from different specialties, including rheumatology, ophthalmology, dermatology, gastroenterology, and neurology.

scholarly journals Current Status, Goals, and Research Agenda for Outcome Measures Development in Behçet Syndrome: Report from OMERACT 2014

2015 ◽  
Vol 42 (12) ◽  
pp. 2436-2441 ◽  
Author(s):  
Gulen Hatemi ◽  
Yesim Ozguler ◽  
Haner Direskeneli ◽  
Alfred Mahr ◽  
Ahmet Gul ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Working Group ◽  
Research Agenda ◽  
Composite Measure ◽  
Behçet Syndrome ◽  
Advantages And Disadvantages ◽  
Behcet Syndrome ◽  
Core Set ◽  
Organ Specific

Objective.There is an unmet need for reliable, validated, and widely accepted outcomes and outcome measures for use in clinical trials in Behçet syndrome (BS). Our report summarizes initial steps taken by the Outcome Measures in Rheumatology (OMERACT) vasculitis working group toward developing a core set of outcome measures for BS according to the OMERACT methodology, including the OMERACT Filter 2.0, and discussions during the first meeting of the BS working group held during OMERACT 12 (2014).Methods.During OMERACT 12, some of the important challenges in developing outcomes for BS were outlined and discussed, and a research agenda was drafted.Results.Among topics discussed were the advantages and disadvantages of a composite measure for BS that evaluates several organs/organ systems; bringing patients and physicians together for discussions about how to assess disease activity; use of organ-specific measures developed for other diseases; and the inclusion of generic, disease-specific, or organ-specific measures. The importance of incorporating patients’ perspectives, concerns, and ideas into outcome measure development was emphasized.Conclusion.The planned research agenda includes conducting a Delphi exercise among physicians from different specialties that are involved in the care of patients with BS and among patients with BS, with the aim of identifying candidate domains and subdomains to be assessed in randomized clinical trials of BS, and candidate items for a composite measure. The ultimate goal of the group is to develop a validated and widely accepted core set of outcomes and outcome measures for use in clinical trials in BS.

scholarly journals Early interventions for the prevention of post-traumatic stress symptoms in survivors of critical illness: protocol for a systematic review

BMJ Open ◽  
2017 ◽  
Vol 7 (9) ◽  
pp. e018270 ◽  
Author(s):  
Lindsey J Glaspey ◽  
Michael B Roberts ◽  
Anthony Mazzarelli ◽  
Stephen Trzeciak ◽  
Brian W Roberts
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Critical Illness ◽  
Outcome Measures ◽  
Traumatic Stress ◽  
Early Interventions ◽  
Ptsd Symptoms ◽  
Post Traumatic Stress ◽  
Registration Number ◽  
Post Traumatic

IntroductionPost-traumatic stress disorder (PTSD) is being increasingly reported among survivors of critical illness and injury. Previous work has demonstrated that PTSD reduces patient quality of life and ability to return to work, as well as increases healthcare costs. As such, identifying interventions aimed at preventing the development of critical illness-related PTSD could have an important public health impact. The objective of this systematic review is to collate the world’s literature on early interventions aimed at preventing PTSD among survivors of critical illness.Methods and analysisWe will perform a qualitative systematic review of human clinical trials of interventions aimed at preventing or reducing critical illness-related PTSD symptoms. We will methodically search CENTRAL, MEDLINE, Embase and CINAHL. We will also search websites containing details on clinical trials registration (National Library of Medicine’s ClinicalTrials.gov and the WHO’s International Clinical Trials Registry Platform), as well as screen reference lists of the articles we select for inclusion to identify additional studies for potential inclusion. Two authors will independently review all search results. After identification and inclusion of articles, we will use a standardised form for data extraction. We will use tables to describe the study type, populations, interventions tested and timing of interventions, outcome measures and effects of interventions on outcome measures compared with control groups. This review will be completed between 1 August 2017 and 31 August 2017.Ethics and disseminationThe proposed systematic review will not collect individual patient level data and does not require ethical approval. Results of this study will contribute to the understanding of critical illness-related PTSD and help prompt future research aimed at further developing interventions to prevent PTSD symptoms in survivors of critical illness.PROSPERO registration numberThis systematic review is registered in the PROSPERO international prospective register of systematic reviews (registration number CRD42017069672).

Gaps in patient-reported outcome measures in randomized clinical trials of cardiac catheter ablation: a systematic review

2020 ◽  
Vol 6 (4) ◽  
pp. 234-242
Author(s):  
Yang Chen ◽  
Myura Nagendran ◽  
Manuel Gomes ◽  
Peter V Wharton ◽  
Rosalind Raine ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Catheter Ablation ◽  
Outcome Measures ◽  
Comparative Effectiveness ◽  
Randomized Clinical Trials ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Cardiac Catheter ◽  
Patient Reported

Abstract The aim of this systematic review was to evaluate randomized clinical trials (RCTs) of cardiac catheter ablation (CCA) and to assess the prevalence, characteristics and reporting standards of clinically relevant patient-reported outcome measures (PROMs). Electronic database searches of Medline, Embase, CENTRAL, and the WHO Trial Registry were conducted in March 2019. The study protocol was registered on PROSPERO (CRD42019133086). Of 7125 records identified, 237 RCTs were included for analysis, representing 35 427 patients with a mean age of 59 years. Only 43 RCTs (18%) reported PROMs of which 27 included a generic PROM that measured health-related quality of life (HRQL) necessary to conduct comparative effectiveness research. There was notable under-representation of certain patient groups—only 31% were women and only 8% were of non-Caucasian ethnicity, in trials which reported such data. The reporting standard of PROMs was highly variable with 8–62% adherence against CONSORT PRO-specific items. In summary, PROMs play a crucial role in determining the clinical and cost-effectiveness of treatments which primarily offer symptomatic improvement, such as CCA. Their underuse significantly limits evaluation of the comparative effectiveness of treatments. Using CCA as an exemplar, there are additional issues of infrequent assessment, poor reporting and under-representation of many population groups. Greater use of PROMs, and specifically validated HRQL questionnaires, is paramount in giving patients a voice in studies, generating more meaningful comparisons between treatments and driving better patient-centred clinical and policy-level decision-making.

scholarly journals Update on Outcome Measure Development for Large Vessel Vasculitis: Report from OMERACT 12

2015 ◽  
Vol 42 (12) ◽  
pp. 2465-2469 ◽  
Author(s):  
Sibel Zehra Aydin ◽  
Haner Direskeneli ◽  
Antoine Sreih ◽  
Fatma Alibaz-Oner ◽  
Ahmet Gul ◽  
...  
Keyword(s):  
Disease Activity ◽  
Outcome Measures ◽  
Research Agenda ◽  
Task Force ◽  
Large Vessel Vasculitis ◽  
Large Vessel ◽  
Disease Assessment ◽  
Activity Assessment ◽  
Delphi Exercise ◽  
Disease Activity Assessment

Objective.The rarity of large vessel vasculitis (LVV) is a major factor limiting randomized controlled trials in LVV, resulting in treatment choices in these diseases that are guided mainly by observational studies and expert opinion. Further complicating trials in LVV is the absence of validated and meaningful outcome measures. The Outcome Measures in Rheumatology (OMERACT) vasculitis working group initiated the Large Vessel Vasculitis task force in 2009 to develop data-driven, validated outcome tools for clinical investigation in LVV. This report summarizes the progress that has been made on a disease activity assessment tool and patient-reported outcomes in LVV as well as the group’s research agenda.Methods.The OMERACT LVV task force brought an international group of investigators and patient research partners together to work collaboratively on developing outcome tools. The group initially focused on disease activity assessment tools in LVV. Following a systematic literature review, an international Delphi exercise was conducted to obtain expert opinion on principles and domains for disease assessment. The OMERACT vasculitis working group’s LVV task force is also conducting qualitative research with patients, including interviews, focus groups, and engaging patients as research partners, all to ensure that the approach to disease assessment includes measures of patients’ perspectives and that patients have input into the research agenda and process.Results.The preliminary results of both the Delphi exercise and the qualitative interviews were discussed at the OMERACT 12 (2014) meeting and the completion of the analyses will produce an initial set of domains and instruments to form the basis of next steps in the research agenda.Conclusion.The research agenda continues to evolve, with the ultimate goal of developing an OMERACT-endorsed core set of outcome measures for use in clinical trials of LVV.

scholarly journals Clinical outcomes and outcome measurement tools reported in randomised controlled trials of treatment for snakebite envenoming: A systematic review

2021 ◽  
Vol 15 (8) ◽  
pp. e0009589
Author(s):  
Michael Abouyannis ◽  
Dinesh Aggarwal ◽  
David G. Lalloo ◽  
Nicholas R. Casewell ◽  
Mainga Hamaluba ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Randomised Controlled Trials ◽  
Core Outcome Set ◽  
Controlled Trials ◽  
Study Protocols ◽  
Outcome Set ◽  
Secondary Analyses ◽  
Randomised Controlled

Background Snakebite is a priority neglected tropical disease and causes a range of complications that vary depending on the snake species. Randomised clinical trials have used varied outcome measures that do not allow results to be compared or combined. In accordance with the Core Outcomes Measurements in Effectiveness Trials (COMET) initiative, this systematic review aims to support the development of a globally relevant core outcome set for snakebite. Methods All randomised controlled trials, secondary analyses of randomised controlled trials and study protocols investigating the efficacy of therapeutics for human snakebite envenoming were eligible for inclusion. Study screening and data extraction were conducted in duplicate by two independent reviewers. All primary and secondary outcome measures were extracted and compiled, as were adverse event outcome measures. Similar outcome measures were grouped into domains. The study was prospectively registered with PROSPERO: CRD42020196160. Results This systematic review included 43 randomised controlled trials, two secondary analyses and 13 study protocols. A total of 382 outcome measures were extracted and, after duplicates were merged, there were 153 unique outcomes. The most frequently used outcome domain (‘venom antigenaemia’) was included in less than one third of the studies. The unique outcomes were classified into 60 outcome domains. Patient-centred outcomes were used in only three of the studies. Discussion Significant heterogeneity in outcome measures exists in snakebite clinical trials. Consensus is needed to select outcome measures that are valid, reliable, patient-centred and feasible. The results of this systematic review strongly support the development of a core outcome set for use in snakebite clinical trials.

scholarly journals Outcome measures utilized in clinical trials of interventions for post-cardiac arrest syndrome: A systematic review

Resuscitation ◽  
2009 ◽  
Vol 80 (6) ◽  
pp. 617-623 ◽  
Author(s):  
Stephen Trzeciak ◽  
Alan E. Jones ◽  
J. Hope Kilgannon ◽  
Brian M. Fuller ◽  
Brian W. Roberts ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Cardiac Arrest ◽  
Outcome Measures ◽  
Post Cardiac Arrest Syndrome

scholarly journals Fibromyalgia Syndrome Module at OMERACT 9: Domain Construct

2009 ◽  
Vol 36 (10) ◽  
pp. 2318-2329 ◽  
Author(s):  
PHILIP MEASE ◽  
LESLEY M. ARNOLD ◽  
ERNEST H. CHOY ◽  
DANIEL J. CLAUW ◽  
LESLIE J. CROFFORD ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Cognitive Dysfunction ◽  
Outcome Measures ◽  
Outcome Measure ◽  
Core Domain ◽  
Patient Focus Group ◽  
Cerebrospinal Fluid Biomarkers ◽  
Core Set ◽  
Delphi Exercise ◽  
Core Domain Set

The objective of the module was to (1) establish a core domain set for fibromyalgia (FM) assessment in clinical trials and practice, (2) review outcome measure performance characteristics, (3) discuss development of a responder index for assessment of FM in clinical trials, (4) review objective markers, (5) review the domain of cognitive dysfunction, and (6) establish a research agenda for outcomes research. Presentations at the module included: (1) Results of univariate and multivariate analysis of 10 FM clinical trials of 4 drugs, mapping key domains identified in previous patient focus group: Delphi exercises and a clinician/researcher Delphi exercise, and breakout discussions to vote on possible essential domains and reliable measures; (2) Updates regarding outcome measure status; (3) Update on objective markers to measure FM disease state; and (4) Review of the issue of cognitive dysfunction (dyscognition) in FM. Consensus was reached as follows: (1) Greater than 70% of OMERACT participants agreed that pain, tenderness, fatigue, patient global, multidimensional function and sleep disturbance domains should be measured in all FM clinical trials; dyscognition and depression should be measured in some trials; and stiffness, anxiety, functional imaging, and cerebrospinal fluid biomarkers were identified as domains of research interest. (2) FM domain outcome measures have generally proven to be reliable, discriminative, and feasible. More sophisticated and comprehensive measures are in development, as is a responder index for FM. (3) Increasing numbers of objective markers are being developed for FM assessment. (4) Cognitive dysfunction assessment by self-assessed and applied outcome measures is being developed. In conclusion, a multidimensional symptom core set is proposed for evaluation of FM in clinical trials. Research on improved measures of single domains and composite measures is ongoing.

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