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1
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84055213533
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Expert panel on integrated guidelines for cardiovascular health and risk reduction in children and adolescents: Summary report
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These guidelines, commissioned by the National Heart, Lung and Blood Institute, provided graded recommendations for lipid screening, diagnosis and management. They are noteworthy in that they do not consider dyslipidemia in isolation, but provide specific guidance as to how the presence of additional risk factors and risk conditions influence decision-making regarding the management of dyslipidemia
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Expert Panel on Integrated Guidelines for Cardiovascular Health and Risk Reduction in Children and Adolescents: summary report. Pediatrics 2011; 128(SUPPL. 5):S213-S256. These guidelines, commissioned by the National Heart, Lung and Blood Institute, provided graded recommendations for lipid screening, diagnosis and management. They are noteworthy in that they do not consider dyslipidemia in isolation, but provide specific guidance as to how the presence of additional risk factors and risk conditions influence decision-making regarding the management of dyslipidemia.
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(2011)
Pediatrics
, vol.128
, Issue.SUPPL. 5
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2
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84855987839
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Universal screening and drug treatment of dyslipidemia in children and adolescents
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This commentary highlights the controversy in association with the NHLBI Expert Panel recommendations for universal lipid screening and the choice of a statin for first-line, lipid-lowering drug therapy
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Psaty BM, Rivara FP. Universal screening and drug treatment of dyslipidemia in children and adolescents. JAMA 2012; 307:257-258. This commentary highlights the controversy in association with the NHLBI Expert Panel recommendations for universal lipid screening and the choice of a statin for first-line, lipid-lowering drug therapy.
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(2012)
JAMA
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, pp. 257-258
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Psaty, B.M.1
Rivara, F.P.2
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3
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84856015484
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Is universal pediatric lipid screening justified?
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This responsive commentary by two of the members of the NHLBI Expert Panel highlights the rationale for contrary viewpoints regarding the lipid recommendations
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Gillman MW, Daniels SR. Is universal pediatric lipid screening justified? JAMA 2012; 307:259-260. This responsive commentary by two of the members of the NHLBI Expert Panel highlights the rationale for contrary viewpoints regarding the lipid recommendations.
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JAMA
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Gillman, M.W.1
Daniels, S.R.2
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5
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3142717531
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Efficacy and safety of statin therapy in children with familial hypercholesterolemia: A randomized controlled trial
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DOI 10.1001/jama.292.3.331
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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; 292:331-337. (Pubitemid 38944256)
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Journal of the American Medical Association
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Wiegman, A.1
Hutten, B.A.2
De Groot, E.3
Rodenburg, J.4
Bakker, H.D.5
Buller, H.R.6
Sijbrands, E.J.G.7
Kastelein, J.J.P.8
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6
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34548186503
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Statin treatment in children with familial hypercholesterolemia: The younger, the better
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DOI 10.1161/CIRCULATIONAHA.106.671016, PII 0000301720070807000015
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Rodenburg J, Vissers MN, Wiegman A, et al. Statin treatment in children with familial hypercholesterolemia: the younger, the better. Circulation 2007; 116:664-668. (Pubitemid 47356351)
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(2007)
Circulation
, vol.116
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Rodenburg, J.1
Vissers, M.N.2
Wiegman, A.3
Van Trotsenburg, A.S.P.4
Van Der Graaf, A.5
De Groot, E.6
Wijburg, F.A.7
Kastelein, J.J.P.8
Hutten, B.A.9
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7
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0026542685
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National cholesterol education program: Report of the expert panel on blood cholesterol levels in children and adolescents
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American Academy of Pediatrics.
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American Academy of Pediatrics. National Cholesterol Education Program: report of the Expert Panel on blood cholesterol levels in children and adolescents. Pediatrics 1992; 89:525-584.
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(1992)
Pediatrics
, vol.89
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8
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79956263939
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Pediatric aspects of familial hypercholesterolemias: Recommendations from the national lipid association expert panel on familial hypercholesterolemia
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These guidelines from an Expert Panel of the National Lipid Association provide recommendations specific to children and adolescents with familial hypercholesterolemia. The major recommendations, including universal lipid screening, are similar to those made for the general population in the NHLBI Expert Panel guidelines
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Daniels SR, Gidding SS, de Ferranti SD. Pediatric aspects of familial hypercholesterolemias: recommendations from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Clin Lipidol 2011; 5:S30-S37. These guidelines from an Expert Panel of the National Lipid Association provide recommendations specific to children and adolescents with familial hypercholesterolemia. The major recommendations, including universal lipid screening, are similar to those made for the general population in the NHLBI Expert Panel guidelines.
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(2011)
J Clin Lipidol
, vol.5
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Daniels, S.R.1
Gidding, S.S.2
De Ferranti, S.D.3
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9
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80053292217
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Management of familial hypercholesterolemia in children and young adults: Consensus paper developed by a panel of lipidologists, cardiologists, paediatricians, nutritionists, gastroenterologists, general practitioners and a patient organization
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These guidelines from a multisocietal and multidisciplinary group from Begium are specific to children and adolescents with familial hypercholesterolemia. Recommendations highlighting the role of genetic testing are provided
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Descamps OS, Tenoutasse S, Stephenne X, et al. Management of familial hypercholesterolemia in children and young adults: consensus paper developed by a panel of lipidologists, cardiologists, paediatricians, nutritionists, gastroenterologists, general practitioners and a patient organization. Atherosclerosis 2011; 218:272-280. These guidelines from a multisocietal and multidisciplinary group from Begium are specific to children and adolescents with familial hypercholesterolemia. Recommendations highlighting the role of genetic testing are provided.
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(2011)
Atherosclerosis
, vol.218
, pp. 272-280
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Descamps, O.S.1
Tenoutasse, S.2
Stephenne, X.3
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10
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84858311039
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Screening for familial hypercholesterolaemia
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This is a general discussion of screening strategies for familial hypercholesterolemia, making the case for cascade screening, but also highlights the need for follow-up and effective management
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Bender R, Bell DA, Hooper AJ, et al. Screening for familial hypercholesterolaemia. Pathology 2012; 44:122-128. This is a general discussion of screening strategies for familial hypercholesterolemia, making the case for cascade screening, but also highlights the need for follow-up and effective management.
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(2012)
Pathology
, vol.44
, pp. 122-128
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Bender, R.1
Bell, D.A.2
Hooper, A.J.3
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11
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84870056536
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The evaluation of cascade testing for familial hypercholesterolemia
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[Epub ahead of print]. This study represents a computer simulation of screening strategies for familial hypercholesterolemia and highlights some limitations of cascade screening
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Morris JK, Wald DS, Wald NJ. The evaluation of cascade testing for familial hypercholesterolemia. Am J Med Genet 2011 [Epub ahead of print]. This study represents a computer simulation of screening strategies for familial hypercholesterolemia and highlights some limitations of cascade screening.
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(2011)
Am J Med Genet
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Morris, J.K.1
Wald, D.S.2
Wald, N.J.3
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12
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84857234423
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Paediatric screening for hypercholesterolaemia in europe
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This commentary from European pediatric lipid experts reviews different screening strategies and makes a case for universal lipid screening in early childhood
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Kusters DM, de Beaufort C, Widhalm K, et al. Paediatric screening for hypercholesterolaemia in Europe. Arch Dis Childhood 2012; 97:272-276. This commentary from European pediatric lipid experts reviews different screening strategies and makes a case for universal lipid screening in early childhood.
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(2012)
Arch Dis Childhood
, vol.97
, pp. 272-276
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Kusters, D.M.1
De Beaufort, C.2
Widhalm, K.3
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13
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84860403257
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Reduced penetrance of autosomal dominant hypercholesterolemia in a high percentage of families: Importance of genetic testing in the entire family
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This study highlights reduced penetrance of the familial hypercholesterolemia phenotype, which has implications for lipid screening versus genetic screening
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Garcia-Garcia AB, Ivorra C, Martinez-Hervas S, et al. Reduced penetrance of autosomal dominant hypercholesterolemia in a high percentage of families: importance of genetic testing in the entire family. Atherosclerosis 2011; 218:423-430. This study highlights reduced penetrance of the familial hypercholesterolemia phenotype, which has implications for lipid screening versus genetic screening.
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(2011)
Atherosclerosis
, vol.218
, pp. 423-430
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Garcia-Garcia, A.B.1
Ivorra, C.2
Martinez-Hervas, S.3
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14
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80054744511
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Child-parent screening for familial hypercholesterolemia
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This study outlines a strategy for a form of vertical cascade lipid screening in clinical practice that takes advantage of routine visits for immunization to target young parents
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Wald DS, Kasturiratne A, Godoy A, et al. Child-parent screening for familial hypercholesterolemia. J Pediatr 2011; 159:865-867. This study outlines a strategy for a form of vertical cascade lipid screening in clinical practice that takes advantage of routine visits for immunization to target young parents.
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(2011)
J Pediatr
, vol.159
, pp. 865-867
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Wald, D.S.1
Kasturiratne, A.2
Godoy, A.3
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15
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84862774518
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Follow-up of children diagnosed with familial hypercholesterolemia in a national genetic screening program
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This study highlights that diagnosis does not universally translate into medical follow-up and treatment, and supports the need for effective knowledge translation and implementation of guidelines
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Avis HJ, Kusters DM, Vissers MN, et al. Follow-up of children diagnosed with familial hypercholesterolemia in a national genetic screening program. J Pediatr 2012; 161:99-103. This study highlights that diagnosis does not universally translate into medical follow-up and treatment, and supports the need for effective knowledge translation and implementation of guidelines.
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(2012)
J Pediatr
, vol.161
, pp. 99-103
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Avis, H.J.1
Kusters, D.M.2
Vissers, M.N.3
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16
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79953066017
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Molecular basis of autosomal dominant hypercholesterolemia: Assessment in a large cohort of hypercholesterolemic children
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This genetic study provides evidence that the familial hypercholesterolemia phenotype in children signifies the presence of known genetic mutations in nearly all, highlighting familial hypercholesterolemia as the primary cause for children to have LDL-cholesterol levels exceeding the recommended cutpoints for lipidlowering medication
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Van der Graaf A, Avis HJ, Kusters DM, et al. Molecular basis of autosomal dominant hypercholesterolemia: assessment in a large cohort of hypercholesterolemic children. Circulation 2011; 123:1167-1173. This genetic study provides evidence that the familial hypercholesterolemia phenotype in children signifies the presence of known genetic mutations in nearly all, highlighting familial hypercholesterolemia as the primary cause for children to have LDL-cholesterol levels exceeding the recommended cutpoints for lipidlowering medication.
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(2011)
Circulation
, vol.123
, pp. 1167-1173
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Van Der Graaf, A.1
Avis, H.J.2
Kusters, D.M.3
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17
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79960201968
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Primary hyperlipidemias in children: Effect of plant sterol supplementation on plasma lipids and markers of cholesterol synthesis and absorption
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This trial provides further confirmation of the beneficial LDL-cholesterol-lowering effects of plant sterols for children with familial hypercholesterolemia and supports its use as recommended in the recent guidelines
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Guardamagna O, Abello F, Baracco V, et al. Primary hyperlipidemias in children: effect of plant sterol supplementation on plasma lipids and markers of cholesterol synthesis and absorption. Acta Diabetol 2011; 48:127-133. This trial provides further confirmation of the beneficial LDL-cholesterol-lowering effects of plant sterols for children with familial hypercholesterolemia and supports its use as recommended in the recent guidelines.
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(2011)
Acta Diabetol
, vol.48
, pp. 127-133
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Guardamagna, O.1
Abello, F.2
Baracco, V.3
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18
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79952529811
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A systematic review of bile acid sequestrant therapy in children with familial hypercholesterolemia
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Although bile acid sequestrants in the past were the first-line medication recommended for those children meeting LDL-cholesterol criteria for requiring medication, now supplanted by statins, this systematic review highlights their limitations but ongoing role as adjunctive therapy
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Davidson MH. A systematic review of bile acid sequestrant therapy in children with familial hypercholesterolemia. J Clin Lipidol 2011; 5:76-81. Although bile acid sequestrants in the past were the first-line medication recommended for those children meeting LDL-cholesterol criteria for requiring medication, now supplanted by statins, this systematic review highlights their limitations but ongoing role as adjunctive therapy.
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(2011)
J Clin Lipidol
, vol.5
, pp. 76-81
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Davidson, M.H.1
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19
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77955813602
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Are treatment targets for hypercholesterolemia evidence based? Systematic review and meta-analysis of randomised controlled trials
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Lebenthal Y, Horvath A, Dziechciarz P, et al. Are treatment targets for hypercholesterolemia evidence based? Systematic review and meta-analysis of randomised controlled trials. Arch Dis Childhood 2010; 95:673-680.
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(2010)
Arch Dis Childhood
, vol.95
, pp. 673-680
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Lebenthal, Y.1
Horvath, A.2
Dziechciarz, P.3
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20
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0042243555
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Efficacy and safety of atorvastatin in children and adolescents with familial hypercholesterolemia or severe hyperlipidemia: A multicenter, randomized, placebo-controlled trial
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DOI 10.1016/S0022-3476(03)00186-0
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McCrindle BW, Ose L, Marais AD. Efficacy and safety of atorvastatin in children and adolescents with familial hypercholesterolemia or severe hyperlipidemia: a multicenter, randomized, placebo-controlled trial. J Pediatr 2003; 143:74-80. (Pubitemid 36928572)
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(2003)
Journal of Pediatrics
, vol.143
, Issue.1
, pp. 74-80
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McCrindle, B.W.1
Ose, L.2
Marais, A.D.3
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21
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0037132672
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Early statin therapy restores endothelial function in children with familial hypercholesterolemia
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DOI 10.1016/S0735-1097(02)02593-7, PII S0735109702025937
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De Jongh S, Lilien MR, Op't Roodt J, et al. Early statin therapy restores endothelial function in children with familial hypercholesterolemia. J Am Coll Cardiol 2002; 40:2117-2121. (Pubitemid 36005896)
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(2002)
Journal of the American College of Cardiology
, vol.40
, Issue.12
, pp. 2117-2121
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De Jongh, S.1
Lilien, M.R.2
Op'T Roodt, J.3
Stroes, E.S.G.4
Bakker, H.D.5
Kastelein, J.J.P.6
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22
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79953161561
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Changes in the textural characteristics of intima-media complex in young patients with familial hypercholesterolemia: Implication for visual inspection on b-mode ultrasound
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Noto N, Okada T, Abe Y, et al. Changes in the textural characteristics of intima-media complex in young patients with familial hypercholesterolemia: implication for visual inspection on B-mode ultrasound. J Am Soc Echocardiogr 2011; 24:438-443.
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J Am Soc Echocardiogr
, vol.24
, pp. 438-443
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Noto, N.1
Okada, T.2
Abe, Y.3
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23
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84871264858
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Noninvasive markers of early atherosclerosis have been useful in defining the associations between risk factors and the vascular process in children and adolescents, and more recently in suggesting a benefit to treatment with statins. This study highlights ultrasound techniques that may increase definition of these subclinical vascular changes
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Noninvasive markers of early atherosclerosis have been useful in defining the associations between risk factors and the vascular process in children and adolescents, and more recently in suggesting a benefit to treatment with statins. This study highlights ultrasound techniques that may increase definition of these subclinical vascular changes.
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24
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84860615159
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Mipomersen, an apolipoprotein b synthesis inhibitor, lowers low-density lipoprotein cholesterol in high-risk statin-intolerant patients: A randomized, double-blind, placebo-controlled trial
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Visser ME, Waquener G, Baker BF, et al. Mipomersen, an apolipoprotein B synthesis inhibitor, lowers low-density lipoprotein cholesterol in high-risk statin-intolerant patients: a randomized, double-blind, placebo-controlled trial. Eur Heart J 2012; 33:1142-1149.
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Eur Heart J
, vol.33
, pp. 1142-1149
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Visser, M.E.1
Waquener, G.2
Baker, B.F.3
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25
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84864239186
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Therapeutic potential of mipomersen in the management of familial hypercholesterolaemia
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Gelsinger C, Steinhager-Thiessen E, Kassner U. Therapeutic potential of mipomersen in the management of familial hypercholesterolaemia. Drugs 2012; 72:1145-1155.
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(2012)
Drugs
, vol.72
, pp. 1145-1155
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Gelsinger, C.1
Steinhager-Thiessen, E.2
Kassner, U.3
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26
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84863494422
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Effect of a monoclonal antibody to pcsk9, regn727/sar236553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezetimibe therapy: A phase 2 randomised controlled trial
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Stein EA, Gipe D, Berqueron J, et al. Effect of a monoclonal antibody to PCSK9, REGN727/SAR236553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezetimibe therapy: a phase 2 randomised controlled trial. Arch Dis Childhood 2012; 380:29-36.
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(2012)
Arch Dis Childhood
, vol.380
, pp. 29-36
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Stein, E.A.1
Gipe, D.2
Berqueron, J.3
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27
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84858638369
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Effect of a monoclonal antibody to pcsk9 on ldl cholesterol
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Stein EA, Mellis S, Yancopoulos GD, et al. Effect of a monoclonal antibody to PCSK9 on LDL cholesterol. N Engl J Med 2012; 366:1108-1118.
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N Engl J Med
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, pp. 1108-1118
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Stein, E.A.1
Mellis, S.2
Yancopoulos, G.D.3
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28
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84863981772
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Rationale for cholesteryl ester transfer protein inhibition
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Hewing B, Fisher EA. Rationale for cholesteryl ester transfer protein inhibition. Curr Opin Lipidol 2012; 23:372-376.
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(2012)
Curr Opin Lipidol
, vol.23
, pp. 372-376
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Hewing, B.1
Fisher, E.A.2
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29
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33846151732
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Inhibition of microsomal triglyceride transfer protein in familial hypercholesterolemia
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DOI 10.1056/NEJMoa061189
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Cuchel M, Bloedon LT, Szapary PO, et al. Inhibition of microsomal triglyceride transfer protein in familial hypercholesterolemia. N Engl J Med 2007; 356: 148-156. (Pubitemid 46089675)
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New England Journal of Medicine
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Cuchel, M.1
Bloedon, L.T.2
Szapary, P.O.3
Kolansky, D.M.4
Wolfe, M.L.5
Sarkis, A.6
Millar, J.S.7
Ikewaki, K.8
Siegelman, E.S.9
Gregg, R.E.10
Rader, D.J.11
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30
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79959606300
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Long-term follow-up of statin treatment in a cohort of children with familial hypercholesterolemia: Efficacy and tolerability
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As it is unlikely that long-term clinical trials will be performed, observational data may have some utility in tracking safety of statins in children. This study provides longer term data that confirms ongoing safety with pravastatin
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Carreau V, Girardet JP, Bruckert E. Long-term follow-up of statin treatment in a cohort of children with familial hypercholesterolemia: efficacy and tolerability. Paediatr Drugs 2011; 13:267-275. As it is unlikely that long-term clinical trials will be performed, observational data may have some utility in tracking safety of statins in children. This study provides longer term data that confirms ongoing safety with pravastatin.
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(2011)
Paediatr Drugs
, vol.13
, pp. 267-275
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Carreau, V.1
Girardet, J.P.2
Bruckert, E.3
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31
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79953703680
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An eight-week trial investigating the efficacy and tolerability of atorvastatin for children and adolescents with heterozygous familial hypercholesterolemia
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Although recent guidelines recommend drug therapy at younger ages, the majority of clinical trials did not include very young children. This short-term trial showed similar safety and efficacy in prepubertal children 6-10 years of age compared to peripubertal and postpubertal children with familial hypercholesterolemia
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Gandelman K, Glue P, Laskey R, et al. An eight-week trial investigating the efficacy and tolerability of atorvastatin for children and adolescents with heterozygous familial hypercholesterolemia. Pediatr Cardiol 2011; 32:433-441. Although recent guidelines recommend drug therapy at younger ages, the majority of clinical trials did not include very young children. This short-term trial showed similar safety and efficacy in prepubertal children 6-10 years of age compared to peripubertal and postpubertal children with familial hypercholesterolemia.
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(2011)
Pediatr Cardiol
, vol.32
, pp. 433-441
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Gandelman, K.1
Glue, P.2
Laskey, R.3
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32
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0014693152
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Risks of ischaemic heart-disease in familial hyperlipoproteinaemic states
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Slack J. Risks of ischaemic heart-disease in familial hyperlipoproteinaemic states. Lancet 1969; 2:1380-1382.
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Lancet
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Slack, J.1
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33
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Reductions in all-cause, cancer, and coronary mortality in statin-treated patients with heterozygous familial hypercholesterolaemia: A prospective registry study
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Neil A, Cooper J, Betteridge J, et al. Reductions in all-cause, cancer, and coronary mortality in statin-treated patients with heterozygous familial hypercholesterolaemia: a prospective registry study. Eur Heart J 2008; 29:2625-2633.
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Eur Heart J
, vol.29
, pp. 2625-2633
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Neil, A.1
Cooper, J.2
Betteridge, J.3
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34
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79959728614
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Effect of lipid-lowering treatment on natural history of heterozygous familial hypercholesterolemia in past three decades
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This study of adults provides additional confirmation of the impact of more effective lipid-lowering drug therapy on reducing the risk of cardiovascular disease events
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Elis A, Zhou R, Stein EA. Effect of lipid-lowering treatment on natural history of heterozygous familial hypercholesterolemia in past three decades. Am J Cardiol 2011; 108:223-226. This study of adults provides additional confirmation of the impact of more effective lipid-lowering drug therapy on reducing the risk of cardiovascular disease events.
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(2011)
Am J Cardiol
, vol.108
, pp. 223-226
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Elis, A.1
Zhou, R.2
Stein, E.A.3
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35
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81355160365
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Reduction in mortality in subjects with homozygous familial hypercholesterolemia associated with advances in lipidlowering therapy
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Patients with homozygous familial hypercholesterolemia are a rare but difficult group of patients to manage. This study of a large cohort of homozygotes confirms the utility of aggressive lipid-lowering drug therapy in this resistant population
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Raal FJ, Pilcher GJ, Panz VR, et al. Reduction in mortality in subjects with homozygous familial hypercholesterolemia associated with advances in lipidlowering therapy. Circulation 2011; 124:2202-2207. Patients with homozygous familial hypercholesterolemia are a rare but difficult group of patients to manage. This study of a large cohort of homozygotes confirms the utility of aggressive lipid-lowering drug therapy in this resistant population.
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(2011)
Circulation
, vol.124
, pp. 2202-2207
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Raal, F.J.1
Pilcher, G.J.2
Panz, V.R.3
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36
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79955000769
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Liver transplantation as a treatment option for three siblings with homozygous familial hypercholesterolemia
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Kucukkartallar T, Yankol Y, Kanmaz T, et al. Liver transplantation as a treatment option for three siblings with homozygous familial hypercholesterolemia. Pediatr Transplant 2011; 15:281-284.
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(2011)
Pediatr Transplant
, vol.15
, pp. 281-284
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Kucukkartallar, T.1
Yankol, Y.2
Kanmaz, T.3
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37
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79951701629
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Preemptive liver transplantation in a child with familial hypercholesterolemia
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Maiorana A, Nobili V, Calandra S, et al. Preemptive liver transplantation in a child with familial hypercholesterolemia. Pediatr Transplant 2011; 15:E25-E29.
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Pediatr Transplant
, vol.15
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Maiorana, A.1
Nobili, V.2
Calandra, S.3
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38
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79951667867
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Liver transplantation as treatment for familial homozygous hypercholesterolemia: Too early or too late
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This commentary contextualizes the role and timing of liver transplantation for more definitive treatment of homozygous familial hypercholesterolemia in selected patients
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Malatack JJ. Liver transplantation as treatment for familial homozygous hypercholesterolemia: too early or too late. Pediatr Transplant 2011; 15:123-125. This commentary contextualizes the role and timing of liver transplantation for more definitive treatment of homozygous familial hypercholesterolemia in selected patients.
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(2011)
Pediatr Transplant
, vol.15
, pp. 123-125
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Malatack, J.J.1
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