Rapid Resolution of Life-Threatening Hypertriglyceridemia after Evinacumab Administration in a Pediatric HSCT Recipient: A Case Report

Evinacumab, a human monoclonal antibody against angiopoietin-like protein 3 (ANGPTL3), has recently been approved by the U.S. Food and Drug Administration as an add-on therapy for homozygous familial hypercholesterolemia (HoFH) in patients of 12 years older. Its role triglyceride-lowering drug is also emerging literature. However, it not this indication yet, neither adult nor pediatric population. We describe case 10-year-old boy who underwent allogeneic hematopoietic stem cell transplant acute lymphoblastic leukemia complicated chronic graft-versus-host disease (GVHD) presented life-threatening refractory hypertriglyceridemia due to concomitant use ruxolitinib sirolimus. After failure insulin treatment technical impossibility performing lipid apheresis, child evinacumab treatment, obtaining dramatic rapid reduction triglyceride cholesterol levels. This first report patient younger than Europe receiving treat severe hypertriglyceridemia. The with proteins inhibitors effective, safe, well-tolerated our patient, suggesting that may be used population when other therapeutic strategies are ineffective or contraindicated.