There is a strong need for multidisciplinary and specific research on childhood-onset systemic lupus erythematosus (SLE) to help better understand the condition and guide treatment, a study by the Childhood Arthritis and Rheumatology Research Alliance (CARRA) and Lupus Foundation of America (LFA) found.
Among the areas that require higher priority are lupus nephritis, or kidney swelling and irritation, and neuropsychiatric disease — when lupus affects the nervous system. Also needing higher priority are clinical trials, biomarkers, and skin disease that does not respond to treatment (refractory).
The study, “Research priorities in childhood-onset lupus: results of a multidisciplinary prioritization exercise,” appeared in the journal Pediatric Rheumatology.
Pediatric lupus, or childhood-onset SLE, differs from the disease in adults in some important aspects — specifically genetic predisposition, environmental triggers, and how the treatments behave inside a patient’s body. However, most efforts to fight lupus do not differentiate between children and adults.
Most clinical trials only include adults, or a small number of adolescents. In most cases, treatment for children is based exclusively on results obtained in adults — which may or may not be the same for children. Therefore, there is a need for specific research addressing pediatric lupus.
The LFA and CARRA surveyed 256 specialists, including pediatric rheumatologists, dermatologists, and nephrologists, or kidney specialists, with experience treating the disease in an effort to establish the priorities that should guide research in pediatric lupus.
“This is the first published research prioritization effort among experts in childhood lupus, which highlights the lack of evidence that exists specific to this disease. There are important differences between adults and children, and significant knowledge gaps limit our understanding of the best treatments and long term outcomes in children with lupus,” Aimee Hersh, chair of the CARRA Systemic Lupus Erythematosus Committee, said in a press release.
The study found that treating children with lupus requires a multidisciplinary approach. Most respondents said that rheumatologists should collaborate with other specialists, such as nephrologists (92%), dermatologists (73%), and mental health specialists (68%).
The respondents identified lupus nephritis — when lupus affects the kidneys — as the top research priority. Up to 80% of children with lupus develop nephritis and the current treatments are complex, long-lasting, and cause adverse side effects.
“It has been well established that lupus nephritis is highly prevalent in cSLE [childhood systemic lupus erythematosus], 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,” the researchers said.
The second research priority was neuropsychiatric diseases. Lupus affects the nervous system in around 65% of children, 85% of which occurs in the first two years after diagnosis. This affects the cognitive development of children and adolescents, diminishing their quality of life.
Research on neuropsychiatric lupus has been carried out almost exclusively on adults — and even in that population, there are many gaps of knowledge.
“This study underscores the urgency for childhood lupus research. We are committed to investing in this area, and our partnership with CARRA as leaders in this field will bolster our progress as we work together to elevate childhood lupus research,” said Stevan Gibson, president, and CEO at LFA.
The most urgent needs across the two top research priorities were understanding the long-term outcomes, determining the best treatments, identifying biomarkers of diseases progression, and discovery and development of new treatments.
“This multidisciplinary approach to identifying research priorities emphasizes the need for collaboration for childhood lupus care and research. The results from this exercise will help set a research agenda moving forward,” said Hersh.
“The findings from the study offer a roadmap to guide future research endeavors so that the scope and devastation of lupus in children align with received funding,” Gibson concluded.