Key result
Evinacumab cuts pre-apheresis LDL-C ~67% in an infant with HoFH.
Why the study?
The efficacy of the ANGPTL3 antibody evinacumab as an adjunct to lipid apheresis in infants with homozygous familial hypercholesterolemia was investigated.
Does evinacumab reduce LDL cholesterol levels and the required frequency of lipid apheresis in an infant with homozygous familial hypercholesterolemia?
Case Report (n=1)
Does evinacumab reduce LDL cholesterol levels and the required frequency of lipid apheresis in an infant with homozygous familial hypercholesterolemia?
Evinacumab as an adjunct therapy can significantly lower LDL cholesterol and reduce the frequency of lipid apheresis required in infants with homozygous familial hypercholesterolemia.
Answers are grounded in this paper and cite their sources.
May support reduced apheresis frequency in infants with HoFH; hypothesis-generating and requires prospective validation.
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.
No takes yet. Share an insight, caveat, or question.
Stirnkorb et al. (2025) conducted a case report in Homozygous familial hypercholesterolemia (HoFH) (n=1). Evinacumab vs. Pre-treatment (lipid apheresis alone) was evaluated on Reduction in LDL cholesterol. Four-weekly infusion of evinacumab resulted in a 67% reduction in pre-apheresis LDL cholesterol in an infant with HoFH, allowing apheresis frequency to be reduced to once a week.
Synapse has enriched 10 related papers. Consider them for comparative context: