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Optimizing Treatment of Familial Hypercholesterolemia in Children and Adolescents.

Luirink IK, Hutten BA, Wiegman A - Curr Cardiol Rep (2015)

Bottom Line: Lifestyle adjustments are the first step in treatment.If this is not sufficient, statins are the preferred initial pharmacological therapy and they have been proven effective and safe.However, studies in children are still to be awaited.

View Article: PubMed Central - PubMed

Affiliation: Department of Pediatrics, Emma Children's Hospital, Academic Medical Center, University of Amsterdam, Meibergdreef 9, 1105 AZ, Amsterdam, The Netherlands, i.k.luirink@amc.uva.nl.

ABSTRACT
Cardiovascular disease (CVD) is still the most prominent cause of death and morbidity in the world, and one of the major risk factors for developing CVD is hypercholesterolemia. Familial hypercholesterolemia (FH) is a dominantly inherited disorder characterized by markedly elevated plasma low-density lipoprotein cholesterol and premature coronary heart disease. Currently, several treatment options are available for children with FH. Lifestyle adjustments are the first step in treatment. If this is not sufficient, statins are the preferred initial pharmacological therapy and they have been proven effective and safe. However, treatment goals are often not achieved and, hence, there is a need for novel treatment options. Currently, several options are being studied in adults and first results are promising. However, studies in children are still to be awaited.

No MeSH data available.


Related in: MedlinePlus

Novel lipid-regulating drug targets. Novel drugs target either very low-density lipoprotein (VLDL) production, by inhibiting apolipoprotein B synthesis [apolipoprotein B (ApoB) antisense oligonucleotide, mipomersen] or lipid loading onto nascent ApoB [microsomal triglyceride transfer protein (MTP) inhibitor, lomitapide], or low-density lipoprotein catabolism by increasing low-density lipoprotein receptor recycling (PCSK9 inhibitors) (with permission [6••])
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Related In: Results  -  Collection


getmorefigures.php?uid=PMC4537493&req=5

Fig1: Novel lipid-regulating drug targets. Novel drugs target either very low-density lipoprotein (VLDL) production, by inhibiting apolipoprotein B synthesis [apolipoprotein B (ApoB) antisense oligonucleotide, mipomersen] or lipid loading onto nascent ApoB [microsomal triglyceride transfer protein (MTP) inhibitor, lomitapide], or low-density lipoprotein catabolism by increasing low-density lipoprotein receptor recycling (PCSK9 inhibitors) (with permission [6••])

Mentions: Much is expected from the agents targeting PCSK9, antisense oligonucleotides targeting apolipoprotein B, and microsomal triglyceride transfer protein inhibitors to reduce LDL-C beyond the levels attainable with statin monotherapy [13] (Fig. 1). The safety and efficacy results of trials with these drugs in children are still awaited [29].Fig. 1


Optimizing Treatment of Familial Hypercholesterolemia in Children and Adolescents.

Luirink IK, Hutten BA, Wiegman A - Curr Cardiol Rep (2015)

Novel lipid-regulating drug targets. Novel drugs target either very low-density lipoprotein (VLDL) production, by inhibiting apolipoprotein B synthesis [apolipoprotein B (ApoB) antisense oligonucleotide, mipomersen] or lipid loading onto nascent ApoB [microsomal triglyceride transfer protein (MTP) inhibitor, lomitapide], or low-density lipoprotein catabolism by increasing low-density lipoprotein receptor recycling (PCSK9 inhibitors) (with permission [6••])
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

Show All Figures
getmorefigures.php?uid=PMC4537493&req=5

Fig1: Novel lipid-regulating drug targets. Novel drugs target either very low-density lipoprotein (VLDL) production, by inhibiting apolipoprotein B synthesis [apolipoprotein B (ApoB) antisense oligonucleotide, mipomersen] or lipid loading onto nascent ApoB [microsomal triglyceride transfer protein (MTP) inhibitor, lomitapide], or low-density lipoprotein catabolism by increasing low-density lipoprotein receptor recycling (PCSK9 inhibitors) (with permission [6••])
Mentions: Much is expected from the agents targeting PCSK9, antisense oligonucleotides targeting apolipoprotein B, and microsomal triglyceride transfer protein inhibitors to reduce LDL-C beyond the levels attainable with statin monotherapy [13] (Fig. 1). The safety and efficacy results of trials with these drugs in children are still awaited [29].Fig. 1

Bottom Line: Lifestyle adjustments are the first step in treatment.If this is not sufficient, statins are the preferred initial pharmacological therapy and they have been proven effective and safe.However, studies in children are still to be awaited.

View Article: PubMed Central - PubMed

Affiliation: Department of Pediatrics, Emma Children's Hospital, Academic Medical Center, University of Amsterdam, Meibergdreef 9, 1105 AZ, Amsterdam, The Netherlands, i.k.luirink@amc.uva.nl.

ABSTRACT
Cardiovascular disease (CVD) is still the most prominent cause of death and morbidity in the world, and one of the major risk factors for developing CVD is hypercholesterolemia. Familial hypercholesterolemia (FH) is a dominantly inherited disorder characterized by markedly elevated plasma low-density lipoprotein cholesterol and premature coronary heart disease. Currently, several treatment options are available for children with FH. Lifestyle adjustments are the first step in treatment. If this is not sufficient, statins are the preferred initial pharmacological therapy and they have been proven effective and safe. However, treatment goals are often not achieved and, hence, there is a need for novel treatment options. Currently, several options are being studied in adults and first results are promising. However, studies in children are still to be awaited.

No MeSH data available.


Related in: MedlinePlus