Abstract
BackgroundChildhood-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.MethodsIn 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.ResultsThe 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.ConclusionThis 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.
Highlights
Childhood-onset systemic erythematosus lupus is characterized by more severe disease, widespread organ involvement and higher mortality compared to adult-onset Systemic lupus erythematosus (SLE)
The Childhood Arthritis and Rheumatology Research Alliance (CARRA) membership response rate was highest with 174/403 (43%) of respondents completing the survey, and 150/174 (86%) of those respondents identified as members of the CARRA SLE Committee
Future work may inform the development of specific requests for applications needed to advance novel research in childhood lupus. This multidisciplinary survey effort represents a first research prioritization excerciseeffort among rheumatologists, dermatologists and nephrologists aimed at identifying key research opportunities and improving outcomes for Childhood onset systemic lupus erythematosus (cSLE)
Summary
Childhood-onset systemic erythematosus lupus (cSLE) is characterized by more severe disease, widespread organ involvement and higher mortality compared to adult-onset SLE. Few commonly used SLE medications have been studied in children, and important knowledge gaps exist concerning epidemiology, genetics, pathophysiology and optimal treatments for cSLE. Childhood-onset systemic erythematosus lupus (cSLE), defined as onset of SLE at age < 18 years, affects an estimated 6000 US children and adolescents [1, 2]. Up to 80% of children with cSLE develop lupus nephritis (LN), resulting in higher mortality rates in childhood versus adult-onset SLE [3]. While many aspects of adult and childhood SLE pathophysiology are likely similar, important differences exist concerning genetic predisposition, environmental triggers, pharmacokinetics and management of concerns specific to the pediatric population such as neurodevelopment, growth, puberty, mental health and educational development [5]. Our understanding of cSLE is limited by key gaps in knowledge surrounding these factors, which influence natural history as well as the identification of phenotypic and molecular heterogeneity impacting treatment choices, clinical outcomes and medication toxicity
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