scholarly journals Development of Outcome Measures for Large-vessel Vasculitis for Use in Clinical Trials: Opportunities, Challenges, and Research Agenda

2011 ◽  
Vol 38 (7) ◽  
pp. 1471-1479 ◽  
Author(s):  
HANER DIRESKENELI ◽  
SIBEL Z. AYDIN ◽  
TANAZ A. KERMANI ◽  
ERIC L. MATTESON ◽  
MAARTEN BOERS ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Research Agenda ◽  
Trial Design ◽  
Large Vessel Vasculitis ◽  
The Other ◽  
Large Vessel ◽  
Therapeutic Trials ◽  
Core Set ◽  
Existing Data

Giant cell (GCA) and Takayasu’s arteritis (TAK) are 2 forms of large-vessel vasculitis (LVV) that involve the aorta and its major branches. GCA has a predilection for the cranial branches, while TAK tends to affect the extracranial branches. Both disorders may also cause nonspecific constitutional symptoms. Although some clinical features are more common in one or the other disorder and the ages of initial presentation differ substantially, there is enough clinical and histopathologic overlap between these disorders that some investigators suggest GCA and TAK may be 2 processes within the spectrum of a single disease. There have been few randomized therapeutic trials completed in GCA, and none in TAK. The lack of therapeutic trials in LVV is only partially explained by the rarity of these diseases. It is likely that the lack of well validated outcome measures for LVV and uncertainties regarding trial design contribute to the paucity of trials for these diseases. An initiative to develop a core set of outcome measures for use in clinical trials of LVV was launched by the international OMERACT Vasculitis Working Group in 2009 and subsequently endorsed by the OMERACT community at the OMERACT 10 meeting. Aims of this initiative include: (1) to review the literature and existing data related to outcome assessments in LVV; (2) to obtain the opinion of experts and patients on disease content; and (3) to formulate a research agenda to facilitate a more data-based approach to outcomes development.

scholarly journals Update on Outcome Measure Development in Large-vessel Vasculitis: Report from OMERACT 2018

2019 ◽  
Vol 46 (9) ◽  
pp. 1198-1201 ◽  
Author(s):  
Sibel Z. Aydin ◽  
Joanna C. Robson ◽  
Antoine G. Sreih ◽  
Catherine Hill ◽  
Fatma Alibaz-Oner ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Patient Reported Outcome ◽  
Large Vessel Vasculitis ◽  
Large Vessel ◽  
Disease States ◽  
Core Set ◽  
Patient Reported ◽  
Delphi Exercise ◽  
Patient Acceptable Symptom State ◽  
Selection Of

Objective.The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group seeks to develop validated outcome measures for use in trials for large-vessel vasculitis (LVV).Methods.An international Delphi exercise conducted among investigators identified items considered important to measure active disease. In parallel, qualitative research with patients was conducted, including interviews and focus groups.Results.Next steps prioritized by the group for LVV include (1) defining disease states (remission, flare, and patient-acceptable symptom state) and (2) selection of patient-reported outcome tools.Conclusion.The ultimate goal is to develop an OMERACT-endorsed core set of outcome measures for use in clinical trials of LVV.

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 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 Development of a Core Set of Outcome Measures for Large-vessel Vasculitis: Report from OMERACT 2016

2017 ◽  
Vol 44 (12) ◽  
pp. 1933-1937 ◽  
Author(s):  
Antoine G. Sreih ◽  
Fatma Alibaz-Oner ◽  
Tanaz A. Kermani ◽  
Sibel Z. Aydin ◽  
Peter F. Cronholm ◽  
...  
Keyword(s):  
Focus Groups ◽  
Outcome Measures ◽  
Large Vessel Vasculitis ◽  
Assessment Tools ◽  
Large Vessel ◽  
Disease Assessment ◽  
Patient Interviews ◽  
Core Set ◽  
Delphi Exercise ◽  
Disease Specific

Objective.Among the challenges in conducting clinical trials in large-vessel vasculitis (LVV), including both giant cell arteritis (GCA) and Takayasu arteritis (TA), is the lack of standardized and meaningful outcome measures. The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group initiated an international effort to develop and validate data-driven outcome tools for clinical investigation in LVV.Methods.An international Delphi exercise was completed to gather opinions from clinical experts on LVV-related domains considered important to measure in trials. Patient interviews and focus groups were completed to identify outcomes of importance to patients. The results of these activities were presented and discussed in a “Virtual Special Interest Group” using telephone- and Internet-based conferences, discussions through electronic mail, and an in-person session at the 2016 OMERACT meeting. A preliminary core set of domains common for all forms of LVV with disease-specific elements was proposed.Results.The majority of experts agree with using common outcome measures for GCA and TA, with the option of supplementation with disease-specific items. Following interviews and focus groups, pain, fatigue, and emotional effect emerged as health-related quality of life domains important to patients. Current disease assessment tools, including the Birmingham Vasculitis Activity Score, were found to be inadequate to assess disease activity in GCA and standardized assessment of imaging tests were felt crucial to study LVV, especially TA.Conclusion.Initial data from a clinician Delphi exercise and structured patient interviews have provided themes toward an OMERACT-endorsed core set of domains and outcome measures.

scholarly journals Developing a Core Set of Outcome Measures for Behçet Disease: Report from OMERACT 2016

2017 ◽  
Vol 44 (11) ◽  
pp. 1750-1753 ◽  
Author(s):  
Gulen Hatemi ◽  
Alexa Meara ◽  
Yesim Ozguler ◽  
Haner Direskeneli ◽  
Alfred Mahr ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Working Group ◽  
Organ Involvement ◽  
Future Research ◽  
Behçet Disease ◽  
Behcet Disease ◽  
Core Set ◽  
Patient Reported ◽  
Specific Organ

Objective.The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group has been working toward developing a data-driven core set of outcome measures for use in clinical trials of Behçet’s syndrome [Behçet disease (BD)]. This paper summarizes the group’s work through OMERACT 2016, discussions during the meeting, and the future research agenda.Methods.Qualitative patient interviews were conducted among 20 patients with BD who have different types of organ involvement. A 3-round Delphi among BD experts and patients was initiated to identify domains, subdomains, and outcomes to be assessed in clinical trials of BD. The results of these studies were discussed during OMERACT 2016 and next steps were planned.Results.Patients’ perspectives and priorities were identified through qualitative interviews that identified candidate domains and subdomains for inclusion in the Delphi and characterized some shortcomings of the currently used patient-reported outcomes in BD. The first round of the Delphi was completed and several domains or subdomains were endorsed by the experts and/or the patients. Because many more items were endorsed than would be feasible to assess during a clinical trial, rating and ranking of items by physicians and patients was planned as a next critical step. The challenges of assessing specific organ system involvement was also discussed.Conclusion.The OMERACT Behçet Syndrome Working Group research program will identify core domains for assessment in BD with the goal of developing a core set of outcome measures for use in all trials of BD with the option to incorporate additional outcomes for specific organ involvement.

scholarly journals Problems in Clinical Trial Design in Multiple Sclerosis

2002 ◽  
Vol 4 (3) ◽  
pp. 125-138 ◽  
Author(s):  
Jeffrey I. Greenstein
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Evidence Based ◽  
Controlled Clinical Trials ◽  
Cognitive Components ◽  
Basic Understanding ◽  
Clinical Measures

Historically, it has been difficult to demonstrate the effectiveness of treatments for multiple sclerosis (MS) because of the variability in the course of the disease, the lack of well-defined, reliable clinical measures, and the pervasiveness of poorly controlled clinical trials. Hence, to interpret the results of clinical trials in MS and make evidence-based decisions regarding treatment for their patients, neurologists should have a basic understanding of appropriate outcome measures and the necessary controls of a well-designed study. This paper reviews the controls required to test the efficacy of agents for the treatment of MS and offers examples of poorly controlled clinical trials to illustrate the problems in interpreting data without such controls. In addition, the outcome measures that should be used to assess the efficacy of treatments on the physical, inflammatory, and cognitive components of the disease are discussed. (Int J MS Care. 2002; 4: 125–131, 136–137)

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.

scholarly journals Reconciling Healthcare Professional and Patient Perspectives in the Development of Disease Activity and Response Criteria in Connective Tissue Disease–related Interstitial Lung Diseases

2014 ◽  
Vol 41 (4) ◽  
pp. 792-798 ◽  
Author(s):  
Lesley Ann Saketkoo ◽  
Shikha Mittoo ◽  
Sid Frankel ◽  
Daphne LeSage ◽  
Catherine Sarver ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Connective Tissue ◽  
Connective Tissue Disease ◽  
Outcome Measures ◽  
Working Group ◽  
Lung Diseases ◽  
Interstitial Lung Diseases ◽  
Patient Perspectives ◽  
High Morbidity ◽  
Core Set

Interstitial lung diseases (ILD), including those related to connective tissue disease (CTD), and idiopathic pulmonary fibrosis (IPF) carry high morbidity and mortality. Great efforts are under way to develop and investigate meaningful treatments in the context of clinical trials. However, efforts have been challenged by a lack of validated outcome measures and by inconsistent use of measures in clinical trials. Lack of consensus has fragmented effective use of strategies in CTD-ILD and IPF, with a history of resultant difficulties in obtaining agency approval of treatment interventions. Until recently, the patient perspective to determine domains and outcome measures in CTD-ILD and IPF had never been applied. Efforts described here demonstrate unequivocally the value and influence of patient involvement on core set development. Regarding CTD-ILD, this is the first OMERACT working group to directly address a manifestation/comorbidity of a rheumatic disease (ILD) as well as a disease not considered rheumatic (IPF). The OMERACT 11 proceedings of the CTD-ILD Working Group describe the forward and lateral process to include both the medical and patient perspectives in the urgently needed identification of a core set of preliminary domains and outcome measures in CTD-ILD and IPF.

scholarly journals Establishing a Core Domain Set to Measure Rheumatoid Arthritis Flares: Report of the OMERACT 11 RA Flare Workshop

2014 ◽  
Vol 41 (4) ◽  
pp. 799-809 ◽  
Author(s):  
Vivian P. Bykerk ◽  
Elisabeth Lie ◽  
Susan J. Bartlett ◽  
Rieke Alten ◽  
Annelies Boonen ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trials ◽  
Focus Groups ◽  
Research Agenda ◽  
Observational Studies ◽  
Core Domain ◽  
Domain Identification ◽  
Core Set ◽  
Patient Reported ◽  
Core Domain Set

Objective.The OMERACT Rheumatoid Arthritis (RA) Flare Group (FG) is developing a data-driven, patient-inclusive, consensus-based RA flare definition for use in clinical trials, longterm observational studies, and clinical practice. At OMERACT 11, we sought endorsement of a proposed core domain set to measure RA flare.Methods.Patient and healthcare professional (HCP) qualitative studies, focus groups, and literature review, followed by patient and HCP Delphi exercises including combined Delphi consensus at Outcome Measures in Rheumatology 10 (OMERACT 10), identified potential domains to measure flare. At OMERACT 11, breakout groups discussed key domains and instruments to measure them, and proposed a research agenda. Patients were active research partners in all focus groups and domain identification activities. Processes for domain selection and patient partner involvement were case studies for OMERACT Filter 2.0 methodology.Results.A pre-meeting combined Delphi exercise for defining flare identified 9 domains as important (> 70% consensus from patients or HCP). Four new patient-reported domains beyond those included in the RA disease activity core set were proposed for inclusion (fatigue, participation, stiffness, and self-management). The RA FG developed preliminary flare questions (PFQ) to measure domains. In combined plenary voting sessions, OMERACT 11 attendees endorsed the proposed RA core set to measure flare with ≥ 78% consensus and the addition of 3 additional domains to the research agenda for OMERACT 12.Conclusion.At OMERACT 11, a core domain set to measure RA flare was ratified and endorsed by attendees. Domain validation aligning with Filter 2.0 is ongoing in new randomized controlled clinical trials and longitudinal observational studies using existing and new instruments including a set of PFQ.

A core outcome set for clinical trials in acute diarrhoea

2014 ◽  
Vol 100 (4) ◽  
pp. 359-363 ◽  
Author(s):  
Jacek Karas ◽  
Shai Ashkenazi ◽  
Alfredo Guarino ◽  
Andrea Lo Vecchio ◽  
Raanan Shamir ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Adverse Effects ◽  
Outcome Measures ◽  
Core Outcome Set ◽  
Acute Diarrhoea ◽  
Core Outcome ◽  
Two Phase ◽  
Outcome Set ◽  
Core Set ◽  
Study Heterogeneity

ObjectiveCore outcome sets are the baseline for what should be measured in clinical research and, thus, should serve as a guide for what should be collected and reported. The Consensus Group on Outcome Measures Made in Pediatric Enteral Nutrition Clinical Trials, established in 2012, agreed that consensus on a core set of outcomes with agreed-upon definitions that should be measured and reported in clinical trials was needed. To achieve this goal, six working groups (WGs) were setup, including WG on acute diarrhoea, whose main goal was to develop a core outcome set for trials in acute diarrhoea.MethodsThe first step identified how published outcomes related to acute diarrhoea were reported. The second focused on the methodology for determining which outcomes to measure in clinical trials. The third employed a two-phase questionnaire study using the Delphi technique to define clinically important outcomes to clinicians and parents.ResultsFor therapeutic studies, the five most important outcome measures were diarrhoea duration, degree of dehydration, need for hospitalisation (or duration of hospitalisation for inpatients), the proportion of patients recovered by 48 h and adverse effects. The prophylactic core outcome set included prevention of diarrhoea, prevention of dehydration, prevention of hospitalisation and adverse effects.ConclusionsThe outcome sets for therapy and prevention can be recommended for use in future trials of patients with gastroenteritis. Their envisioned goal is to decrease study heterogeneity and to ease the comparability of studies. WG's next step is to determine how to measure the outcomes included in the core set.

Sign in / Sign up

Export Citation Format

Share Document