Research priorities in childhood-onset lupus: results of a multidisciplinary prioritization exercise

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.

Principles of pediatric lupus nephritis in a prospective contemporary multi-center cohort

“…16 LN has recently been named a research priority by both CARRA and the Lupus Foundation of America. 17 Since CARRA currently has a robust active cohort of cSLE patients, our aim was to identify traditional principles related to the diagnosis, treatment, and surveillance of LN in childhood and determine if they remain valid in this contemporary multi-center cohort.…”

“There’s so much to be done”: a qualitative study to elucidate research priorities in childhood-onset systemic lupus erythematosus

“…While there have been advances in understanding disease mechanisms and treatment of cSLE in the last decade, significant needs remain, underscoring the necessity of a focused research agenda for allocation of available resources. Expanding on the findings from the ALPHA study5 6 and CARRA-LFA survey regarding cSLE research priorities,7 we elucidated approaches to address cSLE research priorities, exploring both barriers and opportunities to advancing cSLE research. Multidisciplinary experts in our study identified research priorities represented by five domains: (1) expanding disease knowledge; (2) investigator collaboration; (3) partnering with patients and families; (4) improving care to optimise research; and (5) overcoming investigator barriers.…”

“…To expand on the NIAMS-identified objectives and ALPHA study results, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) and the Lupus Foundation of America (LFA) partnered to identify key knowledge gaps and strategies for advancing cSLE research. An initial objective of this collaboration was to prioritise cSLE research domains by surveying rheumatologists, nephrologists and dermatologists with expertise in cSLE 7. The previously published survey study identified the highest prioritised domains as SLE nephritis, clinical trials, biomarkers, neuropsychiatric disease and refractory skin disease 7…”

Reproductive health needs of adolescent and young adult women with pediatric rheumatic diseases

“…Some participants with mild juvenile arthritis even questioned their ability to have children at all. These issues highlight a critical disconnect between patients’ and clinicians’ priorities and knowledge: reproductive health is not yet routinely addressed as part of transitional care [ 20 ] and ranks low on clinicians’ research priorities [ 21 ]. Reasons why reproductive health inquiry and counseling has not become standard in pediatric subspecialty care is likely multifactorial and is a topic of ongoing research.…”