132 boys aged 10-17 years with heterozygous familial hypercholesterolemia
were randomized to placebo vs. treatment with Lovastatin in a
48 week, double-blind, placebo-controlled study. In the first
24 weeks, Lovastatin was increased from 10mg/d to 20mg/d and 40mg/d
at 8 and 16 weeks respectively vs. placebo. During the second
24 weeks, Lovastatin was kept at 40mg/d vs. placebo.
Patients were monitored for lipid levels, hepatic enzymes, CK,
growth, sexual maturation, nutritional levels, thyroid, adrenal,
and pituitary function.
For efficacy analysis, patients needed to complete the 8 week
phases to be included in the summary statistics. For safety, analysis
was carried out on an intent-to-treat basis. Study was not powered
to detect significant difference in safety.
Lovastatin reduced LDL by 17-27%, Tot Chol by 13-21% at all dosages.
There was a minimal and non-significant increase in HDL.
Clinical and biochemical data on growth, hormones, and nutrition
indicate no significant difference between placebo and Lovastatin
over 48 weeks. (Further studies are required).