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

Malin Regardt^1,2, Christopher A. Mecoli^3,4,5, Jin Kyun Park^{6,7,8,9,10,11,12,13,14}, Merrilee Needham¹⁵, Ingrid De Groot¹⁶, Catherine Sarver¹⁷, Ingrid E. Lundberg¹⁸, Beverly Shea¹⁹, Marianne De Visser²⁰, Yeong Wook Song¹³, Clifton O. Bingham III²¹, Lisa Christopher-Stine^22,23,24 and Helene Alexanderson²⁵, ¹Learning, Informatics,Management and Ethics, Karolinska institutet, Stockholm, Sweden, ²Function Area Occupational Therapy and Physical Therapy, Karolinska University Hospital, Stockholm, Sweden, ³Johns Hopkins University School of Medicine, Baltimore, MD, ⁴Internal Medicine/Rheumatology, Johns Hopkins University, Baltimore, MD, ⁵Rheumatology, Johns Hopkins University, Baltimore, MD, ⁶Department of Molecular Medicine and Biopharmaceutical Sciences, Graduate School of Convergence Science and Technology, Seoul National University, Seoul, Korea, Republic of (South), ⁷Division of Rheumatology, Seoul National University Hospital, Seoul, Korea, Republic of (South), ⁸Department of Molecular medicine and biopharmaceutical science, Seoul National University, seoul, Korea, Republic of (South), ⁹Division of Rheumatology, Department of Internal Medicine, Seoul National University Hospital, Seoul, Korea, Republic of (South), ¹⁰jinkyunpark@gmail.com, Division of Rheumatology, Seoul National University Hospital, Seoul, Korea, Republic of (South), ¹¹Division of Rheumatology, Department of Internal Medicine, Seoul National University College of Medicine, Seoul, Korea, Republic of (South), ¹²Division of Rheumatology, Department of Internal Medicine, Seoul National University, Seoul, Korea, Republic of (South), ¹³Department of Molecular Medicine and Biopharmaceutical Sciences, Graduate School of Convergence Science and Technology, and College of Medicine, Seoul National University, Seoul, Korea, Republic of (South), ¹⁴Department of Molecular Medicine and Biopharmaceutical Sciences, BK21 plus Graduate School of Convergence Science and Technology, and College of Medicine or College of Pharmacy, Seoul National University, Seoul, Korea, Republic of (South), ¹⁵Murdoch University, Perth, Australia, ¹⁶patient research partner, Rotterdam, Netherlands, ¹⁷patient research partner, maryland, MD, ¹⁸Department of Medicine, Division of Rheumatology, Karolinska Institutet, Stockholm, Sweden, ¹⁹Bruyere Research Institute, Ottawa, ON, Canada, ²⁰Department of Neurology, Academic Medical Centre, Amsterdam, Netherlands, ²¹Rheumatology, Johns Hopkins University School of Medicine, Baltimore, MD, ²²Ste 4100 Rm 409, Johns Hopkins University School of Medicine, Baltimore, MD, ²³Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, ²⁴Medicine and Neurology, Johns Hopkins University, Baltimore, MD, ²⁵Department of NVS, Division of Physical Therapy, Karolinska Institutet, Huddinge, Sweden

Meeting: 2018 ACR/ARHP Annual Meeting

Keywords: Idiopathic Inflammatory Myopathies (IIM), outcome measures and patient outcomes

Session Information

Date: Wednesday, October 24, 2018

Session Title: 6W007 ACR Abstract: Patient Outcomes, Preferences, & Attitudes II: PROs (2910–2915)

Session Type: ACR Concurrent Abstract Session

Session Time: 9:00AM-10:30AM

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.

Reference: Regardt M et al. Patients’ experience of myositis and further validation of a patient-reported outcome measure – establishing core domains and expanding patient input on clinical assessment in myositis. Report from OMERACT 12. J Rheumatol 2015;42:2492-5.

Figure 1: Draft Modified Core Domain Set for PROs in IIM Patients

Disclosure: M. Regardt, None; C. A. Mecoli, None; J. K. Park, None; M. Needham, None; I. De Groot, None; C. Sarver, None; I. E. Lundberg, Bristol-Myers Squibb, 2,AstraZeneca, 2,AstraZeneca, 5,UCB, Inc., 5,Corbus Pharmaceuticals, 5,Novartis, 1,Roche, 1; B. Shea, None; M. De Visser, None; Y. W. Song, Astellas Pharma, Inc., 9; C. O. Bingham III, None; L. Christopher-Stine, Inova Diagnostics, 7; H. Alexanderson, None.

To cite this abstract in AMA style:

Regardt M, Mecoli CA, Park JK, Needham M, De Groot I, Sarver C, Lundberg IE, Shea B, De Visser M, Song YW, Bingham III CO, Christopher-Stine L, Alexanderson H. A Draft Modified Core Domain Set for Patient-Reported Outcomes (PRO) in Patients with Idiopathic Inflammatory Myopathies (IIM): An Omeract Report [abstract]. Arthritis Rheumatol. 2018; 70 (suppl 10). https://acrabstracts.org/abstract/a-draft-modified-core-domain-set-for-patient-reported-outcomes-pro-in-patients-with-idiopathic-inflammatory-myopathies-iim-an-omeract-report/. Accessed April 8, 2020.

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