Efficacy and Safety of Statin Therapy in Children With Familial Hypercholesterolemia

Wiegman, Albert; Hutten, Barbara A.; Groot, Eric de; Rodenburg, Jessica; Bakker, Henk D.; Büller, Harry R.; Sijbrands, Eric J.G.; Kastelein, John J.P.

doi:10.1001/jama.292.3.331

Cited by 561 publications

(335 citation statements)

References 24 publications

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“…The effects of pharmacological therapy with statins in children with FH has been evaluated in a few randomized, controlled studies. Wiegman et al randomly assigned 106 children with FH to treatment with pravastatin and 108 children with FH to placebo group [100]. After 2 years of treatment, there was statistically significant decrease of absolute values of IMT in carotid arteries (the mean of CCA, bulb, and ICA IMT) in children treated with statins.…”

Section: Is Intima-media Thickening Reversible?mentioning

confidence: 99%

Intima–media thickness measurements in children with cardiovascular risk factors

2009

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Measuring intima-media thickness (IMT) is now a standard diagnostic procedure in assessing cardiovascular risk and hypertensive target-organ damage (TOD) in adults. There is also an increasing number of pediatric publications evaluating IMT in children from high-risk groups, such as those with arterial hypertension, diabetes, chronic kidney disease, obesity, dyslipidemia, and homocystinurias. It has been shown that carotid IMT is strongly related with other markers of TOD in children with arterial hypertension and with metabolic cardiovascular risk factors. In children with coarctation of the aorta, carotid IMT correlated both with blood pressure and even with mild residual aortic gradient. On the other hand, studies in children with high cardiovascular risk have shown that normalization of blood pressure and metabolic abnormalities led to regression of arterial changes and decrease of IMT. Although not yet accepted as standard pediatric procedure, IMT measurement is emerging as a promising method of assessing TOD and cardiovascular risk and monitoring treatment efficacy. From a practical point of view, clinical utility of IMT measurements seems to be similar to use of echocardiography in assessing left ventricular mass. However, IMT measurements in children and adolescents should be standardized to avoid bias caused by the use of different measurement methods.

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Section: Is Intima-media Thickening Reversible?mentioning

confidence: 99%

Intima–media thickness measurements in children with cardiovascular risk factors

2009

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“…In Japan, there are currently no guidelines for the pharmacological treatment of children with hetero FH; however, recent studies of children and adolescents with hetero FH have established the effectiveness and safety of statin therapy 13,14) . Based on these data, the American Academy of Pediatrics (AAP) released revised recommendations for the management of hypercholesterolemia 15) .…”

Section: Discussionmentioning

confidence: 99%

An 11-Year-Old Boy with Familial Hypercholesterolemia Showing Multiple Xanthomas and Advanced Atherosclerosis, Who Responded to Lipid-Lowering Therapy Using Statin

Ohshiro

Shimabukuro

Sunagawa

et al. 2009

JAT

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Familial hypercholesterolemia (FH) is characterized by a high level of LDL-cholesterol (LDL-C) and a high prevalence of atherosclerotic coronary heart disease; however, hypercholesterolemia is usually the only clinical finding in children with heterozygous FH in their first decade of life. We report a case of FH in an 11-year-old boy who presented with multiple xanthomas at both elbows, thickened Achilles tendons, and hyperplasia of the intima-media complex of the carotid artery. Echocardiogram revealed partial calcification of the aortic and mitral valves, but no stenosis of the coronary arteries was detected on 3D-computed tomography. The activity of LDL receptors was reduced to 32% by lymphocyte assay. The family history showed vertical transmission of hypercholesterolemia from father to son, thereby suggesting dominant inheritance. After 12 months of treatment with statin and resin, his LDL-C decreased from 446 to 220 mg/dL, thickening of the Achilles tendons decreased from 16 − 18 mm to 13 mm, and hyperplasia of the intima-media complex decreased from 1.3 mm to 0.7 mm. These findings suggest that our patient had heterozygous FH. However, based on his advanced atherosclerosis, we cannot exclude the possibility that our patient may be accompanying dyslipidemia due to causes in addition to heterozygous FH. J Atheroscler

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“…In each trial, the agent was specifically studied in familial hypercholesterolemia, using an established surrogate outcome (changes in lipid profiles). Impressively, all 8 studies led to efficacy‐related labeling changes 21, 22, 23, 24, 25, 26, 27, 28. The use of a specific patient population with markedly abnormal lipid profiles has been identified as a critical feature in the success of these trials.…”

Section: Approaches To Improving Clinical Trials In Children With Carmentioning

confidence: 99%

Recommendations to Enhance Pediatric Cardiovascular Drug Development: Report of a Multi‐Stakeholder Think Tank

Török

Kannankeril

et al. 2018

JAHA

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Efficacy and Safety of Statin Therapy in Children With Familial Hypercholesterolemia

Cited by 561 publications

References 24 publications

Intima–media thickness measurements in children with cardiovascular risk factors

Intima–media thickness measurements in children with cardiovascular risk factors

An 11-Year-Old Boy with Familial Hypercholesterolemia Showing Multiple Xanthomas and Advanced Atherosclerosis, Who Responded to Lipid-Lowering Therapy Using Statin

Recommendations to Enhance Pediatric Cardiovascular Drug Development: Report of a Multi‐Stakeholder Think Tank

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