OMERACT 2018 Modified Patient-reported outcome domain core set in the life impact area for adult idiopathic inflammatory myopathies

Objective. To present and vote on a myositis modified patient-reported outcome core domain set in the life impact area at the Outcome Measures in Rheumatology (OMERACT) 2018. Methods. Based on results from international focus groups and Delphi surveys, a draft core set was developed. Results. Domains muscle symptoms, fatigue, level of physical activity, and pain reached ≥ 70% consensus and were mandatory to assess in all trials. Domains lung, joint, and skin symptoms were mandatory in specific circumstances. This core set was endorsed by > 85% at OMERACT 2018. Conclusion. We propose a life impact core set for patients with idiopathic inflammatory myopathies and will proceed with instrument selections

Perceptions of patients, caregivers, and healthcare providers of idiopathic inflammatory myopathies: An international OMERACT Study

Objective. Patient-reported outcome measures (PROM) that incorporate the patient perspective have not been well established in idiopathic inflammatory myopathies (IIM). As part of our goal to develop IIM-specific PROM, the Outcome Measures in Rheumatology (OMERACT) Myositis special interest group sought to determine which aspects of disease and its effects are important to patients and healthcare providers (HCP). Methods. Based on a prior qualitative content analysis of focus groups, an initial list of 24 candidate domains was constructed. We subsequently conducted an international survey to identify the importance of each of the 24 domains to be assessed in clinical research. Patients with IIM, their caregivers, and HCP treating IIM completed the survey. Results. In this survey, a total of 638 respondents completed the survey, consisting of 510 patients, 101 HCP, and 27 caregivers from 48 countries. Overall, patients were more likely to rank “fatigue,” “cognitive impact,” and “difficulty sleeping” higher compared with HCP, who ranked “joint symptoms,” “lung symptoms,” and “dysphagia” higher. Both patients and providers rated muscle symptoms as their top domain. In general, patients from different countries were in agreement on which domains were most important. One notable exception was that patients from Sweden and the Netherlands ranked lung symptoms significantly higher compared to other countries including the United States and Australia (mean weighted rankings of 2.86 and 2.04 vs 0.76 and 0.80, respectively; p < 0.0001). Conclusion. Substantial differences exist in how IIM is perceived by patients compared to HCP, with different domains prioritized. In contrast, patients’ ratings across the world were largely similar

A Draft Modified Core Domain Set for Patient-Reported Outcomes (PRO) in Patients with Idiopathic Inflammatory Myopathies (IIM): An Omeract Report

Background/Purpose: The OMERACT Myositis special interest group (SIG) represents clinicians, patients, and researchers from four continents. Focus groups were conducted including 61 patients on three countries resulting in a list of 26 domains (1). In collaboration with International Myositis Assessment Clinical Study Group (IMACS), our goal was to identify a set of core patient-reported outcomes (PRO) in regards to life impact important to assess in clinical trials and clinical practice in myositis. Methods: Patients with adult polymyositis, dermatomyositis, antisynthetase syndrome, or immune-mediated necrotizing myopathy (IMNM) in South Korea, Sweden and USA (N=638) responded to the first online modified Delphi in 2016. The second modified Delphi included patients (N=563), health care providers (HCP) (N=101), care givers (N=27) and regulatory agencies (n=xx) from multiple countries in 2017. A third modified Delphi was administered in 2018 including 410 patients, 109 HCP, 22 caregivers. Results: From this work, four domains were deemed mandatory to measure in all clinical trials for IIM: fatigue, pain, levels of physical activity, and muscle symptoms (Figure 1). Additional optional domains include skin symptoms, lung symptoms, and joint symptoms. Several other domains were deemed important to study with further research efforts including sleeping difficulty, cognitive distress, ability to work, and emotional distress. Conclusion: A draft set of core PRO has been developed through validated methods based on OMERACT guidelines. Fatigue, pain, levels of physical activity and muscle symptoms were included in the inner circle and should always be used in clinical trials in IIM. We next seek to develop corresponding instruments with each of these domains with future efforts

Assessing the content validity of patient-reported outcome measures in adult myositis: A report from the OMERACT myositis working group

Objective To investigate the content validity of several patient-reported outcome measures (PROMs) in patients with idiopathic inflammatory myopathies (IIM). Methods Seven individual PROM instruments were selected by the Outcome Measures in Rheumatology (OMERACT) Myositis Working Group relating to the following domains: pain, fatigue, physical function and physical activity. Twenty patients from the Johns Hopkins Myositis Center were selected for one-on-one face-to-face or phone interviews for cognitive interviewing of individual PROMs to assess comprehension and content validity. Additionally, patients were asked if they thought muscle symptoms, an area originally identified in qualitative studies, were encapsulated by the other four domains. Results The majority of patients (>70%) felt that each of the instruments was clear, easy to read and understand, and could be used for assessment of its domain. Two-thirds (66%) of patients felt that ‘muscle symptoms’ were captured by the other domains. Conclusions We provided evidence to support adequate content validity for several PROMs. Further research is needed to determine whether ‘muscle symptoms’ warrant a separate domain