Nephritis, clinical trials top list of childhood lupus research priorities
Nephritis, clinical trials and biomarkers rank highest among research priorities related to childhood-onset systematic lupus erythematosus, according to a survey of rheumatologists, dermatologists and nephrologists published in Pediatric Rheumatology.
“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,” Stacy P. Ardoin, MD, of Nationwide Children’s Hospital, and colleagues wrote.
The researchers added that “our understanding of [childhood-onset] SLE 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.”
To address these gaps in knowledge, the Lupus Foundation of America (LFA) and the Childhood Arthritis and Rheumatology Research Alliance (CARRA) partnered to develop and distribute surveys to rheumatologists, dermatologists and nephrologists. The survey itself was developed by a working group that included members of both the LFA and CARRA, as well as the Midwestern Pediatric Nephrology Consortium (MWPNC) and the Pediatric Dermatology Research Association (PeDRA), based on their expertise and a literature review.
The online survey was distributed via Qualtrics to 752 members of CARRA, the MWPNC and the PeDRA. It included 17 items, and asked respondents to categorize research domains as either high, medium or low priority. In addition, for domains categorized as high priority, respondents were asked to rank research topics within that category. A total of 256 individuals responded to the survey, of whom 55% were pediatric rheumatologists, 24% were nephrologists, 7% were dermatologists, 2% were adult and pediatric rheumatologists, 9% were fellows and 2% identified as “other.”
According to the researchers, nephritis was the highest ranked priority for childhood-onset SLE among the participants, followed by clinical trials, biomarkers, neuropsychiatric disease, quality of life, cardiovascular outcomes, transition to adult care, mental care and adult outcomes. Respondents in all groups ranked nephritis, clinical trials and biomarkers among their top five priorities. In addition, all groups agreed on specific areas of focus within each research priority. These included determining best treatments, biomarkers and pathophysiology, drug discovery and novel treatments, understanding long term outcomes, and refining provider reported quality measures.
“These results confirm critical, unmet needs in [childhood-onset] SLE,” Ardoin and colleagues wrote. “It has been well established that lupus nephritis is highly prevalent in [childhood-onset] SLE, and despite clear advances in the improvement of patient and renal survival over the last several decades, clinicians and investigators across specialties agree further research needs to be carried out, particularly around treatments and outcomes. Treatment for [childhood-onset] SLE, and particularly [lupus nephritis], is extrapolated from adult clinical trials data, and optimal drug dosing, duration of therapy and outcomes measurement may be distinct for pediatric patients.” – by Jason Laday
Disclosure: The researchers report no relevant financial disclosures.