Progress in the treatment of pediatric homozygous familial hypercholesterolemia
Homozygous familial hypercholesterolemia(HoFH)is a rare,life-threatening disease.The main manifestation of the disease is a substantial elevation in low-density lipoprotein cholesterol levels,which can cause atherosclerotic vascular lesions including coronary arteries,beginning in the first ten years of life.HoFH is still a rare disease in pediatric clinical practice,and the diagnosis is usually postponed and the treatment is insufficient.A mutation in the low-density lipoprotein receptor(LDLR)gene is the most common cause of the illness.The disease requires early,intensive and individualized medication and multimodal treatment to improve the prognosis.This article focuses on the treatment progress of HoFH.