Background: In this single-center, nonrandomized, prospective study, 11 children with severe genetic hypercholesterolemia, without previous cardiovascular disease events, were treated with low-density lipoprotein apheresis (LDLa).
Study design and methods: LDLa was given every 1 or 2 weeks for 2 to 17 years. Clinical cardiovascular events and coronary revascularization, as well as aortic and coronary angiographic findings and ejection fractions, were serially evaluated for 2 to 17 years.
Results: Total cholesterol (TC) and LDL cholesterol levels at baseline were 826.1 +/- 183.3 and 767.8 +/- 181.9 mg/dL, respectively. After LDLa, these levels decreased to 122.6 +/- 24.4 and 79.1 +/- 20.7 mg/dL, respectively (both differences, p < or = 0.001). There were no cardiac deaths, and 6 children were free from any coronary lesions. Nonfatal myocardial infarction was not observed, and coronary revascularization was not required in any patient. Regression of coronary stenosis in children with existing angiographically established lesions after treatment with LDLa was prospectively demonstrated. The statistical analysis applicable to a scoring model (overall atherogenic index [OAI]) highlighted a significant relation between values of 0 to 4 years relevant to the score (p < or = 0.018) and a weaker significant statistic for the value of OAI between 0 and 2 years (p < or = 0.03). The OAI score at baseline was significantly related to the basal values of TC (p = 0.015), LDL cholesterol (p = 0.015), and triglycerides (p = 0.01), but not of high-density lipoprotein cholesterol (p = 0.075) as demonstrated by the logistic regression analysis (Cox and Snell pseudo-R(2) of 0.67).
Conclusion: LDLa interrupted the development of new aortic and coronary lesions in the native arteries and prevented cardiac events and the need for coronary revascularization in children without previous cardiovascular disease events.