Albert Dirisamer, Nilouparak Hachemian, Robert Alexander Bucek, Florian Wolff, Markus Reiter, Prof.Kurt Widhalm

Abstract

Familial  hypercholesterolaemia (FH) is a severe disorder of lipid metabolism associated with an enhanced risk to develop cardiovascular disease later in life, with atherosclerotic lesions beginning already in childhood. These are facts which make an early diagnosis and therapy necessary to prevent or delay such complications. The aim of this study was to investigate the efficacy and safety of low-dose simvastatin, a potent HMG-CoA reductase inhibitor, in children and adolescents with FH. Therefore, 20 children and adolescents (12 females, 8 males) aged between 10 and 17 years with FH were recruited for this 1-year simvastatin study. According to baseline levels of low density lipoprotein (LDL)-C, girls and boys were divided into two groups, one group (with LDL-C < 220 mg/dl) starting with a simvastatin dosage of 5 mg/day, the other (with LDL-C > 220 mg/dl) 10 mg/day with the possibility to increase dosages up to a daily maximum of 20 mg, if not reaching LDL-C concentrations of < 170 mg/dl within the first period. Every 4-8 weeks, weight, height, lipids, Lp(a) and routine safety parameters of all participants were determined by a paediatrician, documenting exactly all side-effects. The percentage decrease was 25 % for LDL-C in the 5 mg simvastatin period
(19 % for total cholesterol (tChol), 30 % for LDL-C in the 10 mg period (26 % for tChol) and 36 % decrease for LDL-C in the 20 mg period (29 % for tChol). The changes for high density lipoprotein (HDL)-C were -5.9 % (5 mg), + 2.9 % (10 mg) and -10.9 % (20 mg) the percentage decrease in triglycerides was 12.6 % (5 mg), 14.3 % (10 mg) and 21 % (20 mg). The side-effects of simvastatin were of no clinical relevance and all disappeared after a couple of dasys.

Conclusion: Our results showed  that simvastatin seems to be an effective and safe medical therapy evedn in children and adolescents with familial hypercholesterolaemia.


Keywords
Children - Familial hypercholesterolaemia - HMG-CoA reductase inhibitor - Simvastatin


Abbreviations
CHD cardiovascular heart disease - CVD cardiovascular disease - FH familial hypercholesterolaemia - HDL high density lipoprotein - LDL low density lipoprotein - Lp(a) lipoprotein (a) - tChol total cholesterol - VLDL very low density lipoprotein


This study was supported in part by MSD
A. Dirisamer, N. Hachemian, K. Widhalm
Departments of Paediatrics
University of Vienna, Währinger Gürtel 18-20
1090  Vienna, Austria
E-Mail: kurt.widhalm@akh-wien.ac.at
Tel.: +43-1-404002337
Fax: +43-1-404002338

A. Dirisamer, F. Wolf
Department of Radiology
University of Vienna, Vienna, Austria

R.A. Bucek, M. Reiter
Zentralröntgeninstitut
Krankenhaus Rudolfstiftung
Vienna, Austria


Eur J Pediatr (2003) 162: 421-425, DOI 10.1007/s00431-003-1181-3
Received: 24 April 2002 / Accepted: 9 January 2003 / Published online: 15 March 2003, Springer Verlag 2003