Core Outcome Measures in Effectiveness Trials

Advancing the Development of Patient-reported Outcomes for Adult Myositis at OMERACT 2016: An International Delphi Study

General Information

Objective. To define a set of core patient-reported domains and respective instruments for use in idiopathic inflammatory myopathies (IIM). Previously, we reported a systematic literature review on patient-reported outcomes (PRO) in IIM followed by conducting international focus groups to elicit patient perspectives of myositis symptoms and effects.

Methods. Based on qualitative content analysis of focus groups, an initial list of 26 candidate domains was constructed. We subsequently conducted an international modified Delphi survey to identify the importance of each of the 26 domains. Participants were asked to rate each domain on a scale of 0–10 (0 = not important, 10 = very important).

Results. In this first round of the Delphi survey, 643 patients participated from the United States (n = 543), Sweden (n = 49), and South Korea (n = 51). Of the 26 domains, 19 (73%) were rated of high importance (= 7/10). The top 5 domains were muscle symptoms, fatigue, interactions with healthcare, medication side effects, and pain. During Outcome Measures in Rheumatology (OMERACT) 2016, we discussed the goal for ultimate reduction in the number of domains and the importance of considering representation of healthcare providers from other specialties, caregivers, representatives of pharmaceutical industries, and regulatory authorities in the next rounds of Delphi to represent broader perspectives on IIM.

Conclusion. Further prioritization and a reduction in the number of domains will be needed for the next Delphi. At the next biennial OMERACT meeting, we aim to present and seek voting on a Myositis Preliminary PRO Core Set to enable ultimate measure selection and development.

Jin Kyun Park, Christopher A. Mecoli, Helene Alexanderson, Malin Regardt, Lisa Christopher-Stine, María Casal-Domínguez, Ingrid de Groot, Catherine Sarver, Ingrid E. Lundberg, Clifton O. Bingham 3rd and Yeong Wook Song


The Journal of Rheumatology
1683 - 1687
Further Study Information

Funding source(s):
Portions of the work have been supported by the Rheumatic Diseases Research Core Center (P30-AR053503) Human Subjects Core from the US National Institutes of Arthritis and Musculoskeletal and Skin Diseases of the National Institutes of Health. Dr. Bingham is supported in part through a Methods Award SC14-1402-10818 from the Patient Centered Outcomes Research Institute. Dr. Christopher-Stine is supported through the Huayi and Siuling Zhang Discovery Fund. Portions of the work have been supported by NuFactor and OptionCare. Dr. Alexanderson and Dr. Regardt are supported by the Swedish Rheumatism Association. Dr. Song and Dr. Park are supported by a grant of the Korea Health technology R&D project through the Korea Health Industry Development Institute, funded by the Ministry of Health and Welfare, Republic of Korea (grant number: HI14C1277).

Health Area

Disease Category

Disease Name

Target Population

Age Range
18 - 100


Nature / type of Intervention


Delphi process
Focus group(s)

Stakeholders Involved


Study Type

COS Patient Reported Outcomes

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