Summary
This was a global study done by investigators at Johns Hopkins, Seoul National University in Korea, and the Karolinska Institute in Sweden. They wanted to better understand how people living with inflammatory muscle diseases experienced their condition and its impacts on their lives. Over the past decade, the value of the patient’s perspective of their diseases and outcomes has been increasingly appreciated. The investigators conducted a large international survey and identified that found that muscle symptoms, fatigue, interactions with the healthcare system, medication side effects, and pain were the things that patients with myositis cared most about.
Why was this study done?
Increasingly, it has been emphasized that clinical trials and patient care should include an assessment of the outcomes that matter most to patients. However, current outcome measures in inflammatory muscle diseases have focused on aspects of the disease that are measured by doctors. These include physical strength testing, imaging studies such as MRI scans, and blood levels of muscle enzymes. Although patient-reported outcomes are widely used in different disease areas in rheumatology and neurology, they have not been well studied in patients with inflammatory muscle diseases. This report publishes our progress to date in identifying the most important aspects of these diseases, as viewed from the patient perspective.
How was this study done?
Focus groups comprised of patients with inflammatory muscle diseases were conducted to identify a list of 26 candidate symptoms (also referred to as domains) that may be important. Then, the investigators conducted a large international survey of more than 600 myositis patients to identify the importance of these domains to a larger group of patients with inflammatory muscle disease. Patients participated from South Korea, Sweden, and the United States. Patients rated the importance of each domain.
What were the major findings?
Of the 26 domains, 19 (73%) were rated of high importance. The 5 top rated domainsof most importance to patients in thinking about their disease were muscle symptoms, fatigue, interactions with the healthcare system, medication side effects, and pain.
What is the impact of this work?
This study was a major step forward to identify what should be asked of patients with myositis concerning their symptoms. This information will be used to develop a patient-reported outcome measure for myositis that can be used for clinical research, clinical trials, and to monitor patients with inflammatory muscle diseases. The next steps will be to obtain input from other stakeholders including patients from other countries, caregivers, physicians, and regulatory agencies. The investigators aim to identify a smaller core set of symptoms that are most important to patients, to ensure they can be regularly and efficiently measured in myositis patients around the world.
This research was supported by:
The Johns Hopkins Rheumatic Disease 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; Methods Award SC14-1402-10818 from the Patient Centered Outcomes Research Institute (PCORI); Huayi and Siuling Zhang Discovery Fund; NuFactor and OptionCare; Swedish Rheumatism Association; Korea Health Technology R&D Project from the Republic of Korea Ministry of Health and Welfare (HI14C1277).
Link to original research article:
Advancing the Development of Patient-reported Outcomes for Adult Myositis at OMERACT 2016: An International Delphi Study. Park JK, Mecoli CA, Alexanderson H, Regardt M, Christopher-Stine L, Domínguez MC, de Groot I, Sarver C, Lundberg IE, Bingham CO Rd, Song YW. J Rheumatol. 2017 Aug 1. pii: jrheum.161252. doi: 10.3899/jrheum.161252.
