Objective. To improve the quality and participation in pediatric rheumatology research, patient-prioritized studies should be emphasized. We collaborated with United States-based pediatric rheumatology advocacy organizations to survey patients and caregivers of children with rheumatic diseases to identify what research topics were most important to them. Methods. We conducted Web-based surveys and focus groups (FG) of patients and caregivers of children with juvenile myositis (JM), juvenile arthritis (JA), and childhood-onset systemic lupus erythematosus (cSLE). Surveys were emailed to listservs and posted to social media sites of JM, JA, and cSLE patient advocacy organizations. An initial survey asked open-ended questions about patient/caregiver research preferences. Responses were further characterized through FG. A final ranking survey asked respondents to rank from a list of research themes the 7 most important to them. Results. There were 365 JM respondents, 44 JA respondents, and 32 cSLE respondents to the final ranking survey. The top research priority for JM was finding new treatments, and for JA and cSLE, the priority was understanding genetic/environmental etiology. The 3 prioritized research themes common across all disease groups were medication side effects, disease flare, and disease etiology. Conclusion. Patient-centered research prioritization is recognized as valuable in conducting high-quality research, yet there is a paucity of data describing patient/family preferences, especially in pediatrics. We used multimodal methodologies to assess current patient/caregiver research priorities to help frame the agenda for the pediatric rheumatology research community. Patients and caregivers from all surveyed disease groups prioritized the study of medication side effects, disease flares, and disease etiology.
Bibliographical noteFunding Information:
The lead investigator, CKC, was supported by Childhood Arthritis and Rheumatology Research Alliance (CARRA) to conduct this study. The Research Committee of PARTNERS (Patients, Advocates and Rheumatology Teams Network for Research and Service) was funded by a PCORI (Patient-Centered Outcomes Research Institute) Patient Powered Research Network (PPRN-1306-04601) grant (Co-PIs Del Gaizo, Schanberg). 1C.K. Correll, MD, MPH, Assistant Professor, University of Minnesota Masonic Children’s Hospital, Department of Pediatrics, Division of Rheumatology, Minneapolis, Minnesota; 2M. Dave, MBA, President, Cure JM Foundation, Encinitas, California; 3A.F. Paul, MA, MBA, Project Manager, Anderson Center for Health Systems Excellence, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio; 4V. Del Gaizo, Director of Strategic Partnerships & Patient Engagement, CARRA, Milwaukee, Wisconsin; 5S. Schrandt, JD, Director of Patient Engagement, Arthritis Foundation, Atlanta, Georgia; 6R.S. Partovi, MPH, Lupus Foundation of America, Washington, DC; 7E.M. Morgan, MD, MSCE, Associate Professor, Division of Rheumatology, Cincinnati Children’s Hospital, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA. Address correspondence to Dr. C.K. Correll, Pediatric Rheumatology, University of Minnesota, 2450 Riverside Ave S., East Bldg Rm 668, Minneapolis, MN 55410, USA. Email: firstname.lastname@example.org.
The study was developed in collaboration with Patients, Advocates and Rheumatology Teams Network for Research and Service (PARTNERS)9. PARTNERS is supported by Patient-Centered Outcomes Research Institute (PCORI), a US government-sponsored institution aimed at promoting patient-centered outcomes research10. PARTNERS brings together 5 leading institutions in rheumatology including Cure JM, AF, LFA, Childhood Arthritis and Rheumatology Research Alliance (CARRA), and Pediatric Rheumatology Care and Outcomes Improvement Network (PR-COIN) to help conduct research on childhood rheumatic diseases with the patient’s and parent’s voice at the center9. A PARTNERS Research Committee, with representation from each affiliated organization, parents, and rheumatology researchers, worked with the lead investigator (CKC), who developed survey questions and focus group (FG) guides with input and feedback from the committee. Cure JM, AF, and LFA are all pediatric rheumatology organizations in the US. CARRA and PR-COIN are mainly US organizations, though they do include a few international centers. The approach was modified from published methodology11,12. The surveys were programmed using Research Electronic Data Capture (REDCap)13 and Qualtrics software (Qualtrics). Step 1: Open-ended survey. A link to an electronic survey (created using REDCap) was emailed to members of Cure JM, AF, and LFA patient/ family listservs and posted on their respective social media sites. The survey links were sent in November 2016, January 2017, and March 2017 for Cure JM, AF, and LFA, respectively. Parents, patients 13 years of age or older and other caregivers were invited to participate. The first question of the survey asked whether the respondent was 13 years or older and if the respondent answered “no” to this question, the survey closed. The survey link included 3 open-ended questions to assess what concerns they found most important. These open-ended questions were as follows: (1) What concerns do you have about your/your child’s health and well-being? What keeps you up at night? (2) Which specific questions or problems do you wish you/your child’s doctors could fix? (3) In addition to finding a cure, what specific areas would you like research to focus on in the next 5 years?
The authors acknowledge CARRA, and the ongoing Arthritis Foundation financial support of CARRA. The authors also acknowledge PARTNERS, Cure JM, Lupus Foundation of America, and all participants in the surveys and focus groups.
- Patient preference
PubMed: MeSH publication types
- Journal Article
- Research Support, Non-U.S. Gov't