Long-term follow-up of statin treatment in a cohort of children with familial hypercholesterolemia: Efficacy and tolerability

Pediatric Drugs

Volumn 13, Issue 4, 2011, Pages 267-275

  Carreau, Valerie ^a   Girardet, Jean Philippe ^b   Bruckert, Eric ^a  

^a PITIÉ SALPÊTRIÈRE HOSPITAL   (France)

^b ARMAND TROUSSEAU HOSPITAL   (France)

Author keywords

children; familial hypercholesterolemia; observational study; Pravastatin; real life clinical practice

Indexed keywords

APOLIPOPROTEIN B; ATORVASTATIN; CHOLESTEROL; COLESTYRAMINE; EZETIMIBE; FIBRIC ACID DERIVATIVE; HIGH DENSITY LIPOPROTEIN CHOLESTEROL; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE INHIBITOR; LOW DENSITY LIPOPROTEIN CHOLESTEROL; LOW DENSITY LIPOPROTEIN RECEPTOR; PRAVASTATIN; ROSUVASTATIN;

ALOPECIA; APHERESIS; ARM PAIN; ARTICLE; ARTICULAR PAIN; BURNING SENSATION; CHILD; CLINICAL DECISION MAKING; CLINICAL PRACTICE; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG SUBSTITUTION; DRUG TOLERABILITY; DRUG WITHDRAWAL; DYSURIA; ELBOW PAIN; FAMILIAL HYPERCHOLESTEROLEMIA; FEMALE; FOLLOW UP; GENE MUTATION; GYNECOMASTIA; HAND PAIN; HEADACHE; HUMAN; ISCHEMIC HEART DISEASE; LEG PAIN; MAJOR CLINICAL STUDY; MALE; MUSCLE CRAMP; MYALGIA; NECK PAIN; PAIN; PRIORITY JOURNAL; PRURITUS; SCHOOL CHILD; SHOULDER PAIN; SIDE EFFECT; THIGH CRAMP;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; FEMALE; FOLLOW-UP STUDIES; FRANCE; HUMANS; HYDROXYMETHYLGLUTARYL-COA REDUCTASE INHIBITORS; HYPERLIPOPROTEINEMIA TYPE II; MALE; PRAVASTATIN; SEVERITY OF ILLNESS INDEX; TIME FACTORS; YOUNG ADULT;

EID: 79959606300     PISSN: 11745878     EISSN: 11792019     Source Type: Journal    
DOI: 10.2165/11591650-000000000-00000     Document Type: Article

References (39)

1. Goldstein JL, Hobbs HH, Brown MS. Familial hypercholesterolemia. In: Scriver CR, Beaudet AL, Sly WS, et al., editors. The metabolic and molecular basis of inherited diseases. New York: McGraw-Hill, 2001: 2863-913
2. Rodenburg J, Vissers MN, Wiegman A, et al. Familial hypercholesterolemia in children. Current Opin Lipidol 2004 Aug; 15 (4): 405-11 (Pubitemid 39061620)
3. Austin MA, Hutter CM, Zimmern RL, et al. Genetic causes of monogenic heterozygous familial hypercholesterolemia: a HuGE prevalence review. Am J Epidemiol 2004; 160: 407-20 (Pubitemid 39166973)
4. Civeira F. Guidelines for the diagnosis and management of heterozygous familial hypercholesterolemia. Atherosclerosis 2004; 173: 55-68 (Pubitemid 38224196)
5. Multiple risk factor intervention trial: risk factor changes andmortality results. Multiple Risk Factor Intervention Trial Research Group. JAMA 1982; 248: 1465-77
6. Wiegman A, Rodenburg J, de Jongh S, et al. Family history and cardiovascular risk in familial hypercholesterolemia: data in more than 1000 children. Circulation 2003; 107: 1473-8 (Pubitemid 36378483)
7. Mortality in treated heterozygous familial hypercholesterolaemia: implications for clinical management. Scientific Steering Committee on behalf of the Simon Broome Register Group. Atherosclerosis 1999; 142: 105-12
8. Li S, Chen W, Srinivasan SR, et al. Childhood cardiovascular risk factors and carotid vascular changes in adulthood. The Bogalusa Study. JAMA 2003; 290 (17): 2271-6 (Pubitemid 37430559)
9. Berenson GE, Srinavasan SR, Bao W, et al. Association between multiple cardiovascular risk factors and atherosclerosis in children and young adults. N Engl J Med 1998; 338 (23): 1650-6 (Pubitemid 28267909)
10. Wiegman A, de Groot E, Hutten BA, et al. Arterial intima-media thickness in children heterozygous for familial hypercholesterolemia. Lancet 2004; 363: 369-70 (Pubitemid 38169546)
11. Aggoun Y, Bonnet D, Sidi D, et al. Arterial mechanical changes in children with familial hypercholesterolemia. Arterioscler Thromb Vasc Biol 2000; 20 (9): 2070-5
12. Stary HC. Evolution and progression of atherosclerotic lesions in coronary arteries of children and young adults. Arteriosclerosis 1989; 9: I19-32 (Pubitemid 19046963)
13. McGill Jr HC, McMahan CA, Malcom GT, et al. Effects of serum lipoproteins and smoking on atherosclerosis in young men and women. The PDAY Research Group. Pathobiological Determinants of Atherosclerosis in Youth. Arterioscler Thromb Vasc Biol 1997; 17: 95-106 (Pubitemid 27057330)
14. Berenson GS, Srinivasan SR, Bao W, et al. Association between multiple cardiovascular risk factors and atherosclerosis in children and young adults. The Bogalusa Heart Study. N Engl J Med 1998; 338: 1650-6 (Pubitemid 28267909)
15. National Cholesterol Education Program (NCEP): highlights of the report of the Expert Panel on Blood Cholesterol Levels in Children and Adolescents. Pediatrics 1992; 89: 495-501
16. de Jongh S, Lilien MR, opt RJ, et al. Early statin therapy restores endothelial function in children with familial hypercholesterolemia. J Am Coll Cardiol 2002; 40: 2117-21 (Pubitemid 36005896)
17. Daniels SR, Greer FR. Lipid screening and cardiovascular health in childhood. Pediatrics 2008; 122: 198-208
18. The Writing Group for the DISC Collaborative Research Group. Efficacy and safety of lowering dietary intake of fat and cholesterol in children with elevated low-density lipoprotein cholesterol. The Dietary Intervention Study in Children (DISC). JAMA 1995; 273 (18): 1429-35
19. Wahrburg U. What are the health effects of fat? Eur J Nutr 2004; 43 Suppl. 1: I6-11
20. Katan MB. Trans fatty acids and plasma lipoproteins. Nutr Rev 2000; 58: 188-91 (Pubitemid 30419232)
21. Tonstad S, Knudtzon J, Sivertsen M, et al. Efficacy and safety of cholestyramine therapy in peripubertal and prepubertal children with familial hypercholesterolemia. J Pediatr 1996; 129: 42-9 (Pubitemid 26277245)
22. Rodenburg J, Vissers MN, Wiegman A, et al. Statin treatment in children with familial hypercholesterolemia: the younger, the better. Circulation 2007; 116: 664-8 (Pubitemid 47356351)
23. Arambepola C, Farmer AJ, Perera R, et al. Statin treatment for children and adolescents with heterozygous familial hypercholesterolaemia: a systematic review and meta-analysis. Atherosclerosis 2007; 195: 339-47 (Pubitemid 350110336)
24. Avis HJ, Vissers MN, Stein EA, et al. A systematic review and meta-analysis of statin therapy in children with familial hypercholesterolemia. Arterioscler Thromb Vasc Biol 2007; 27: 1803-10 (Pubitemid 47207177)
25. van der Graff A, Rodenburg J, Vissers MN, et al. Atherogenic lipoprotein particle size and concentrations and the effect of pravastatin in children with familial hypercholesterolemia. J Pediatr 2008; 152: 873-8
26. Wiegman A, Hutten BA, De Groot E, et al. Efficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized controlled trial. JAMA 2004; 292: 331-7 (Pubitemid 38944256)
27. Treweek S, Zwarenstein M. Making trials matter: pragmatic and explanatory trials and the problem of applicability. Trials 2009; 10: 37
28. Brass EP. The gap between clinical trials and clinical practice: the use of pragmatic clinical trials to inform regulatory decision making. Clin Pharmacol Ther 2010; 87: 351-5
29. Knipscheer HC, Boelen CC, Kastelein JJ, et al. Short-term efficacy and safety of pravastatin in 72 children with familial hypercholesterolemia. Pediatr Res 1996; 39 (5): 867-71 (Pubitemid 26130029)
30. Hedman M, Matikainen T, Föhr A, et al. Efficacy and safety of pravastatin in children and adolescents with heterozygous familial hypercholesterolemia: a prospective clinical follow-up study. J Clin Endocrinol Metab 2005; 90 (4): 1942-52 (Pubitemid 40586225)
31. Luc G, Girardet JP, Bruckert É, et al. Recommendations for hypercholesterolemic children [in French]. Presse Med 2011 Feb; 40 (2): 138-50
32. Saint-Jore B, Varret M, Dachet C, et al. Autosomal dominant type II a hypercholesterolemia: evaluation of the respective contributions of LDLR and APOB gene defects as well as a third major group of defects. Eur JHum Genet 2000; 8 (8): 621-30 (Pubitemid 30639369)
33. Abifadel M, Varret M, Rabes JP, et al. Mutations in PCSK9 cause autosomal dominant hypercholesterolemia. Nat Genet 2003; 34: 154-6 (Pubitemid 36666925)
34. Marduel M, Carrié A, Sassolas A, et al. Molecular spectrum of autosomal dominant hypercholesterolemia in France. Hum Mutat. Epub 2010 Aug 31
35. Law MR, Wald NJ, Rudnicka AR. Quantifying effect of statins on low density lipoprotein cholesterol, ischaemic heart disease, and stroke: systematic review and meta-analysis. BMJ 2003 Jun 28; 326 (7404): 1423
36. McPherson R, Frohlich J, Fodor G, et al. Canadian Cardiovascular Society position statement: recommendations for the diagnosis and treatment of dyslipidemia and prevention of cardiovascular disease. Can J Cardiol 2006 Sep; 22 (11): 913-27 (Pubitemid 44596492)
37. Bruckert E, Hagem G, Dejager S, et al. Mild to moderate muscular symptoms with high-dosage statin therapy in hyperlipidemic patients: thePRIMO study. Cardiovasc Drugs Ther 2005 Dec; 19 (6): 403-14 (Pubitemid 43228152)
38. Evans M, Rees A. The myotoxicity of statins. Curr Opin Lipidol 2002 Aug; 13 (4): 415-20 (Pubitemid 34831945)
39. Avis HJ, Hutten BA, Gagné C, et al. Efficacy and safety of rosuvastatin therapy for children with familial hypercholesterolemia. J Am Coll Cardiol 2010; 55: 1121-6