Efficacy and Safety of Pravastatin in Children and Adolescents with Heterozygous Familial Hypercholesterolemia: A Prospective Clinical Follow-Up Study

Abstract

Heterozygous familial hypercholesterolemia (HeFH) is associated with elevated cholesterol levels and early-onset atherosclerosis. We assessed the efficacy and safety for up to 2 yr of pravastatin treatment in 19 girls and 11 boys (age range, 4.1-18.5 yr) with HeFH. Pravastatin was started at 10 mg/d, with a forced titration by 10 mg at 2, 4, 6, and 12 months until the target cholesterol level [< or =194 mg/dl (< or =5 mmol/liter)] was reached. By 2, 4, 6, 12, and 24 months of treatment, the total cholesterol levels had, respectively, decreased by 19, 20, 23, 27, and 26%, and the low-density lipoprotein cholesterol levels had decreased by 25, 27, 29, 33, and 32% compared with the dietary baseline values. Seventeen percent of patients had lipid deposits (carotid plaque, xanthomas, or corneal arcus) at baseline, and 27% had deposits at 1 yr. The side effects were mild, and no clinically significant elevations in alanine aminotransferase, creatine kinase, or creatinine were seen. Growth and pubertal maturation remained normal in all subjects. In conclusion, pravastatin treatment was safe and well tolerated. The efficacy in children with slight or moderate hypercholesterolemia was satisfactory, but in children with severe hypercholesterolemia, it was insufficient.

Drug Therapy of High-Risk Lipid Abnormalities in Children and Adolescents

“…A recent non-placebocontrolled study of 30 children and adolescents with familial hypercholesterolemia treated with pravastatin to achieve a target LDL cholesterol level showed sustained efficacy and safety over a period of 24 months. 106 Firth and colleagues 107 presented data from an open-label dosage-titration study of fluvastatin in 29 boys with familial hypercholesterolemia. The majority had been titrated up to 80 mg/d, and an interim analysis after 2 years showed marked reductions in LDL with excellent tolerance.…”

Usage of lipid-lowering drugs such as atorvastatin and rosuvastatin in the management of hyperlipidemia in multiple vessel wall block patients

“…Physicians should remain aware of the doses of different statins while applying the results of this present study to clinical practice. 10 This is because different statins, with dose adjustment, can be therapeutically equivalent in reducing LDL-C as concluded Wlodarczyk et al, in a meta-analysis. A unique finding of this study is a reduction in HDL-C levels with both statins.…”

The Use of Statins in Pediatrics: Knowledge Base, Limitations, and Future Directions

“…Elevations in alanine aminotransferase and/or aspartate aminotransferase without clinical hepatotoxicity have been reported in 1% to 5% of children who were treated with simvastatin or atorvastatin 54,55 and in very few children treated with lovastatin or pravastatin. 52,55,84,85 These elevations were observed to be mild, asymptomatic, and reversible after discontinuation of the statin. 49,64,66 To date, clinical trials that have been performed in adolescents that have included assessments of growth, hormone synthesis, pubertal development, significant myopathy, and rhabdomyolysis have provided reassuring results; nonetheless, these studies were of relatively short duration and underpowered to detect infrequent or rare adverse effects.…”