Drug treatment of hypercholesterolemia in children is controversial. The use of natural products has some appeal. The active ingredient of garlic extract, allicin, has been shown to reduce cholesterol levels by 12% in adults. We sought to determine compliance, safety and effectiveness of allicin therapy (Kwai®, 0.6 mg tablet t.i.d.) in children with familial hypercholesterolemia (FH) in a randomized double blind placebo-controlled clinical trial. Sample size was based on an hypothesized 10% relative reduction in total cholesterol in the garlic vs. an identical placebo group; this was deemed the minimal clinically significant difference. Measurements at baseline and after 8 weeks of intervention included blood pressure, height and weight, fasting lipid profiles, lp(a), apolipoproteins B100 and AI, homocysteine, fibrinogen, complete blood count and serum chemistry. Compliance was assessed by returned medication counts. Thirty patients were equally randomized; mean age 14.0±2.3 years, 16 boys(M) and 14 girls(F). Mean lipid values (mmol/L) at baseline were total cholesterol 6.86±1.52, LDL 5.33±1.45, HDL 0.95±0.22 and triglycerides 1.26±0.52. There were no significant differences between groups at baseline, except for gender (garlic, 11M:4F vs. placebo, 5M:10F; p=.07), alkaline phosphatase(179±88 vs. 265±130 U/L; p=.044) and homocysteine(6.49±1.17 vs. 8.25±2.32 μmol/L; p=.02). Compliance (% expected drug taken) was 72±21% for garlic vs. 78±21% for placebo (p=.45). Only 1 patient on garlic developed unpleasant body odor, but concommitantly had been working at a garlic farm during the study. There were no significant differences between groups regarding side effects, blood pressure or growth. There were no significant differences between groups regarding change from baseline in lipid parameters, fibrinogen or homocysteine except for apolipoprotein AI (garlic, +0.06±0.10, vs. placebo,-0.03±0.12 g/L; p=.04). Relative change in total cholesterol was +0.6%(95% confidence interval, -5.8%, +6.9%, p=.86). In the garlic group, there was no significant relationship between dose actually taken (mean 0.023±0.008 mg/kg/day) and any lipid parameter. Conclusion: While safe, garlic extract has no significant effect on lipid parameters, fibrinogen, homocysteine or blood pressure in children with FH.
