An eight-week trial investigating the efficacy and tolerability of atorvastatin for children and adolescents with heterozygous familial hypercholesterolemia

Pediatric Cardiology

Volumn 32, Issue 4, 2011, Pages 433-441

  Gandelman, Kuan ^a   Glue, Paul ^b   Laskey, Rachel ^a   Jones, Jonathan ^c   LaBadie, Robert ^a   Ose, Leiv ^d  

^a PFIZER INC   (United States)

^b UNIVERSITY OF OTAGO   (New Zealand)

^c PFIZER GLOBAL RESEARCH AND DEVELOPMENT   (United Kingdom)

^d OSLO UNIVERSITY HOSPITAL   (Norway)

Author keywords

Heterozygous familial hypocholesterolemia; Low density lipoprotein cholesterol; Statin

Indexed keywords

APOLIPOPROTEIN A1; APOLIPOPROTEIN B; ATORVASTATIN; HIGH DENSITY LIPOPROTEIN CHOLESTEROL; LOW DENSITY LIPOPROTEIN CHOLESTEROL; TRIACYLGLYCEROL; VERY LOW DENSITY LIPOPROTEIN CHOLESTEROL;

ABDOMINAL PAIN; ADOLESCENT; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ALLERGIC RHINITIS; ARTHRALGIA; ARTICLE; ASTHMA; BACTERIAL INFECTION; BRONCHOPNEUMONIA; CHILD; CHOLESTEROL BLOOD LEVEL; CLINICAL ARTICLE; CLINICAL TRIAL; CREATININE BLOOD LEVEL; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG INDUCED HEADACHE; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; EAR INFECTION; FAMILIAL HYPERCHOLESTEROLEMIA; FEMALE; GASTRITIS; GASTROENTERITIS; GENETIC ANALYSIS; HAND FRACTURE; HETEROZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA; HUMAN; INFLUENZA; LABORATORY TEST; LOWER RESPIRATORY TRACT INFECTION; MALE; MUSCULOSKELETAL PAIN; NAUSEA; PAIN; PATIENT COMPLIANCE; PRESCHOOL CHILD; RHINITIS; RHINOPHARYNGITIS; SCHOOL CHILD; SIDE EFFECT; TABLET FORMULATION; TONSILLITIS; TOOTH PAIN; UPPER RESPIRATORY TRACT INFECTION; URTICARIA; VIRUS INFECTION; VITAL SIGN; VOMITING;

ADOLESCENT; CHILD; CHOLESTEROL, HDL; CHOLESTEROL, LDL; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG TOLERANCE; FEMALE; FOLLOW-UP STUDIES; HEPTANOIC ACIDS; HETEROZYGOTE; HUMANS; HYDROXYMETHYLGLUTARYL-COA REDUCTASE INHIBITORS; HYPERLIPOPROTEINEMIA TYPE II; MALE; PYRROLES; RETROSPECTIVE STUDIES; TREATMENT OUTCOME;

EID: 79953703680     PISSN: 01720643     EISSN: None     Source Type: Journal    
DOI: 10.1007/s00246-011-9885-z     Document Type: Article

References (21)

1. Clauss SB, Holmes KW, Hopkins P et al (2005) Efficacy and safety of lovastatin therapy in adolescent girls with heterozygous familial hypercholesterolemia. Pediatrics 116:682-688 (Pubitemid 44336686)
2. Corretti MC, Anderson TJ, Benjamin EJ et al (2002) Guidelines for the ultrasound assessment of endothelial-dependent flow-mediated vasodilation of the brachial artery: a report of the International Brachial Artery Reactivity Task Force. J Am Coll Cardiol 39:257-265 (Pubitemid 34084631)
3. Cuthbert JA, East CA, Bilheimer DW et al (1986) Detection of familial hypercholesterolemia by assaying functional low-density-lipoprotein receptors on lymphocytes. N Engl J Med 314:879-883 (Pubitemid 16126115)
4. Daniels SR, Greer FR, Committee on Nutrition (2008) Lipid screening and cardiovascular health in childhood. Pediatrics 122:198-208
5. de Ferranti S, Ludwig DS (2008) Storm over statins: the controversy surrounding pharmacologic treatment of children. N Engl J Med 359:1309-1312
6. de Jongh S, Lilien MR, op't Roodt J et al (2002) Early statin therapy restores endothelial function in children with familial hypercholesterolemia. J Am Coll Cardiol 40:2117-2121
7. de Jongh S, Ose L, Szamosi T et al (2002) Efficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized, double-blind, placebo-controlled trial with simvastatin. Circulation 106:2231-2237 (Pubitemid 35222218)
8. European Medicines Agency (2006) Assessment of the paediatric needs for cardiovascular products. http://www.emea.europa.eu/pdfs/ human/paediatrics/ 43964906en.pdf. Retrieved 14 June 2010
9. European Medicines Agency (2008) Guideline on reporting the results of population pharmacokinetic analyses. http://www.ema.eur opa.eu/pdfs/human/ewp/ 18599006enfin.pdf. Retrieved 14 June 2010
10. European Medicines Agency (2008) Meeting highlights from the Paediatric Committee, 2-4 June 2008. www.ema.europa.eu/ pdfs/human/pdco/29649408en.pdf. Retrieved 23 June 2010
11. Food and Drug Administration (1999) Guidance for industry: population pharmacokinetics. http://www.fda.gov/downloads/Drugs/ GuidanceComplianceRegulatoryInformation/Guidances/ucm072 137.pdf. Retrieved 18 June 2010
12. Gandelman K, Glue P, Malhotra B, Gastonguay M, Knebel W, Jen F (2010) Population pharmacokinetic and pharmacodynamic modeling of atorvastatin and its active metabolite in pediatric patients with heterozygous familial hypercholesterolemia. Clin Pharmacol Ther 87:S61-S62
13. Goldstein JL, Brown MS (1992) Familial hypercholesterolemia. In: Stanburg JB, Wyngaarden JB, Fredrickson DS (eds) The Metabolic basis of inherited disease, 7th edn. McGraw-Hill, New York, pp 1215-1250
14. Knipscheer HC, Boelen CC, Kastelein JJ et al (1996) Short-term efficacy and safety of pravastatin in 72 children with familial hypercholesterolemia. Pediatr Res 39:867-871 (Pubitemid 26130029)
15. Lebenthal Y, Horvath A, Dziechciarz P et al (2010) Are treatment targets for hypercholesterolemia evidence based? Systematic review and meta-analysis of randomised controlled trials. Arch Dis Child 95:673-680
16. McCrindle BW, Ose L, Marais AD et al (2003) Efficacy and safety of atorvastatin in children and adolescents with familial hypercholesterolemia or severe hyperlipidemia: a multicenter, randomized, placebo-controlled trial. J Pediatr 143:74-80 (Pubitemid 36928572)
17. McCrindle BW, Urbina EM, Dennison BA et al (2007) Drug therapy of high-risk lipid abnormalities in children and adolescents: a scientific statement from the American Heart Association Atherosclerosis, Hypertension, and Obesity in Youth Committee, Council of Cardiovascular Disease in the Young, with the Council on Cardiovascular Nursing. Circulation 115:1948-1967 (Pubitemid 46598908)
18. Rodenburg J, Vissers MN et al (2007) Statin treatment in children with familial hypercholesterolemia: the younger, the better. Circulation 116:664-668 (Pubitemid 47356351)
19. Stein EA, Illingworth DR, Kwiterovich PO Jr et al (1999) Efficacy and safety of lovastatin in adolescent males with heterozygous familial hypercholesterolemia: a randomized controlled trial. JAMA 281:137-144 (Pubitemid 29045345)
20. Vuorio AF, Aalto-Setälä K, Koivisto UM et al (2001) Familial hypercholesterolaemia in Finland: common, rare, and mild mutations of the LDL receptor and their clinical consequences. Finnish FH Group. Ann Med 33:410-421 (Pubitemid 32881033)
21. Wiegman A, Hutten BA, de Groot E et al (2004) Efficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized controlled trial. JAMA 292:331-337 (Pubitemid 38944256)