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Abstract
Introduction: Since the 1980s, the primary treatment of acute Kawasaki disease (KD) has been intravenous immunoglobulin and aspirin. However, 5 – 10% of children with acute KD will develop coronary artery abnormalities despite treatment within the first 10 days after fever onset. There is no approved adjunctive therapy to prevent progression of coronary artery damage in these patients.
Areas covered: The rationale and study design of a Phase I/IIa trial of atorvastatin in children with acute KD and coronary artery inflammation is presented. The studies of host genetics and KD pathogenesis leading up to this trial are reviewed.
Expert opinion: The repurposing of well-studied drugs used in the adult population is a cost-effective and efficient strategy to identify new therapies for pediatric diseases. Exploiting the anti-inflammatory, non-lipid-lowering effects of statins may open up new applications for this class of drugs for the pediatric age group.
Declaration of interest
The atorvastatin clinical trial is supported by an NIH grant awarded to AHT (R01HD081296). No writing assistance was utilized in the production of this manuscript. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.
Notes
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