Project Details

Early Career

Status: Funded - Open

Optimizing Hydroxychloroquine to Reduce Disease Activity in Pediatric Lupus

Rachel Randell, MD

Summary

BACKGROUND: Provide one to two sentences explaining the problem. Systemic lupus erythematosus (lupus) is a chronic autoimmune disease that causes inflammation, organ damage and early death. Up to 20% of all cases of lupus are diagnosed during childhood, and younger patients suffer more severe disease and worse outcomes. GAP: Provide one to two sentences outline the gap your study addresses in the medical research. Hydroxychloroquine (HCQ) decreases inflammation and improves outcomes in adults with lupus. Despite its widespread use in children with lupus, no pediatric- specific dosing guidelines exist, putting children at risk of routine under-dosing, therapeutic failure, uncontrolled inflammation and poor outcomes. HYPOTHESIS: Provide one to two sentences stating the hypothesis of your study. Optimal HCQ dosing will result in simulated HCQ concentrations ≥500 ng/mL in 90% of children, resulting in suppression of inflammatory biomarkers and clinical disease activity. METHODS: Provide one to two sentences describing the study design and study participants. This is an observational, pharmacokinetic/pharmacodynamic study nested within an existing pediatric lupus clinical trial (iPERSONAL NCT04358302). RESULTS: Provide one to two sentences describing any publicly available results. If there are none to date, please write, “Pending”. Pending IMPACT: Provide one to two sentences describing how these findings may impact the health of children. The results of this study will support development of the first pediatric-specific dosing guidelines for HCQ in lupus, which can be immediately applied in clinical practice to optimize treatment, decrease inflammation, and improve outcomes.