Journal of pediatric endocrinology & metabolism : JPEM · 2025
Evinacumab as an adjunct to lipid apheresis in an infant with homozygous familial hypercholesterolemia.
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Stirnkorb Christian, Ney Verena, Bergmann Carsten, Bald Martin
Аннотация
OBJECTIVES: Investigation of the efficacy of the angiopoietin-like protein 3 (ANGPTL3) antibody evinacumab in a four-year-old infant with homozygous familial hypercholesterolemia (HoFH) as an adjunct to lipid apheresis.
CASE PRESENTATION: A two-year-old boy was found to have xanthomas of the Achilles tendon and LDL cholesterol levels around 900 mg/dL. HoHF was subsequently confirmed by molecular genetics. At the age of three, lipid apheresis was started twice a week. At the age of four, a four-weekly infusion of evinacumab was started. This resulted in a 67 % reduction in LDL cholesterol before apheresis, allowing the frequency of apheresis to be reduced to once a week. The mean LDL concentration fell by a further 37 % despite the reduction in apheresis. With the addition of ezetimibe, the mean LDL concentration was reduced to below 115 mg/dL.
CONCLUSIONS: The administration of evinacumab can significantly lower the concentration of LDL cholesterol in infants and thus reduce the frequency of lipid apheresis.
CASE PRESENTATION: A two-year-old boy was found to have xanthomas of the Achilles tendon and LDL cholesterol levels around 900 mg/dL. HoHF was subsequently confirmed by molecular genetics. At the age of three, lipid apheresis was started twice a week. At the age of four, a four-weekly infusion of evinacumab was started. This resulted in a 67 % reduction in LDL cholesterol before apheresis, allowing the frequency of apheresis to be reduced to once a week. The mean LDL concentration fell by a further 37 % despite the reduction in apheresis. With the addition of ezetimibe, the mean LDL concentration was reduced to below 115 mg/dL.
CONCLUSIONS: The administration of evinacumab can significantly lower the concentration of LDL cholesterol in infants and thus reduce the frequency of lipid apheresis.