Background/Purpose: The Patient-Centered Outcomes Research Institute (PCORI) aims to improve the quality and relevance of research by conducting research that is of highest priority to patients and to engage patients at all phases of research. As a project of the PCORI-supported Patient Powered Research Network, PARTNERS (Patients, Advocates and Rheumatology Teams Network for Research and Service), we surveyed parents of children with juvenile myositis (JM) to identify what research questions are most important to them.

Methods: This research prioritization exercise was conducted through web-based surveys, a focus group, and a Delphi process. In November, 2016 a survey link, comprised of 3 open-ended questions to assess what concerns patients/families found most important, was emailed to members of the Cure JM Foundation listserv and posted on Cure JM social media sites. Parents, patients ≥13 years old and family/friends were included. Common themes on research areas of interest were identified from the survey and further characterized through a focus group consisting of parents of children with JM at the annual Cure JM conference in February, 2017. A final survey was created based upon these themes and emailed to the Cure JM listserv and posted to its social media sites in April, 2017. Survey respondents were asked to rank the themes most important to them. Responses were weighted and the 6 most important themes were identified.

Results: There were 138 respondents to the initial survey (77% parents, 11% patients, 12% family/friends). Response rate could not be calculated because the number of potential respondents from social media sites is unknown. From this survey, 23 concerns were identified. The 3 most common concerns were long-term effects of medications, long-term effects of juvenile myositis, and triggers for disease flare. The 23 concerns were further characterized into 20 research priorities during a 9-member focus group. The final ranking survey asked respondents to rank the 7 most important priorities from the 20 themes. There were 365 survey respondents (75% parents, 15% patients, 10% family/friends). The following were ranked in order as the most important research priorities: a cure, new treatments with less side effects, triggers for disease flare, treatment side effects (such as steroids), standards to measure disease activity/remission, genetic/environmental causes of the disease. These research priorities were formally presented at the Global Conference on Myositis and the Childhood Arthritis and Rheumatology Research Alliance annual meetings to help frame the juvenile myositis research agenda.

Conclusion: Patient centered research prioritization is increasingly recognized as a valuable tool in conducting high-quality research, yet there is a lack of publication describing patient/family preferences, especially in pediatrics. Here, we demonstrate a successful program from which we assessed patient/family research priorities in order to inform a joint research agenda process for the juvenile myositis research community.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/eliciting-the-research-priorities-of-parents-and-children-with-juvenile-myositis/