Abstract
BACKGROUND: Childhood-onset systemic erythematosus lupus (cSLE) is characterized by more severe disease, widespread organ involvement and higher mortality compared to adult-onset SLE. However, cSLE is largely underfunded to carry out necessary research to advance the field. Few commonly used SLE medications have been studied in children, and important knowledge gaps exist concerning epidemiology, genetics, pathophysiology and optimal treatments for cSLE. METHODS: In order to assess highest cSLE research priority areas, the Lupus Foundation of America (LFA) and Childhood Arthritis and Rheumatology Research Alliance (CARRA) administered a cSLE research prioritization survey to pediatric rheumatologists, dermatologists and nephrologists with expertise in lupus. Members of LFA and CARRA's SLE Committee identified a list of cSLE research domains and developed a 17-item tiered, web-based survey asking respondents to categorize the research domains into high, medium, or low priority areas. For domains identified as high priority, respondents ranked research topics within that category. For example, for the domain of nephritis, respondents ranked importance of: epidemiology, biomarkers, long-term outcomes, quality improvement, etc. The survey was distributed to members of CARRA, Midwestern Pediatric Nephrology Consortium (MWPNC) and Pediatric Dermatology Research Alliance (PeDRA) Connective Tissue Disease group. RESULTS: The overall response rate was 256/752 (34%). The highest prioritized research domains were: nephritis, clinical trials, biomarkers, neuropsychiatric disease and refractory skin disease. Notably, nephritis, clinical trials and biomarkers were ranked in the top five by all groups. Within each research domain, all groups showed agreement in identifying the following as important focus areas: determining best treatments, biomarkers/pathophysiology, drug discovery/novel treatments, understanding long term outcomes, and refining provider reported quality measures. CONCLUSION: This survey identified the highest cSLE research priorities among leading rheumatology, dermatology and nephrology clinicians and investigators engaged in care of children with lupus. There is a strong need for multidisciplinary collaboration moving forward, which was indicated as highly important among stakeholders involved in the survey. These survey results should be used as a roadmap to guide funding and specific research programs in cSLE to address urgent, unmet needs among this population.