Efficacy and Safety of Ezetimibe Monotherapy in Children with Heterozygous Familial or Nonfamilial Hypercholesterolemia

Kusters, D. Meeike; Caceres, Maria; Coll, Mauricio; Cuffie, Cynthia; Gagné, Claude; Jacobson, Mark Z.; Kwiterovich, Peter O.; Lee, Raymond; Lowe, Robert S.; Massaad, Rachid; McCrindle, Brian W.; Musliner, Thomas; Triscari, Joseph; Kastelein, John J.P.

doi:10.1016/j.jpeds.2015.02.043

Cited by 61 publications

(33 citation statements)

References 38 publications

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“…For children who fail to achieve LDL cholesterol targets, the addition of ezetimibe or a bile acid sequestrant may be considered (25,31). Ezetimibe is a selective cholesterol absorption inhibitor that reduces LDL cholesterol levels by up to 20% when used as monotherapy or in combination with a statin (93,94). Ezetimibe is approved for use from the age of 10 years in the USA and Europe and appears to be safe and well-tolerated.…”

Section: Diet and Lifestyle Measures Current Knowledge And Practicementioning

confidence: 99%

Knowns and unknowns in the care of pediatric familial hypercholesterolemia

Martin

Gidding

Wiegman

et al. 2017

Journal of Lipid Research

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Familial hypercholesterolemia (FH) is a common genetic disorder that causes elevated LDL cholesterol levels from birth. Untreated FH accelerates atherosclerosis and predisposes individuals to premature coronary artery disease (CAD) in adulthood. Mendelian randomization studies have demonstrated that LDL cholesterol has both a causal and cumulative effect on the risk of CAD. This supports clinical recommendations that children with FH commence pharmacological treatment from the age of 8 to 10 years, to reduce the burden of hypercholesterolemia. Worldwide, the majority of children with FH remain undiagnosed. Recent evidence suggests that the frequency of FH is at least 1 in 250 and this constitutes a public health issue. We review and identify the knowns and unknowns concerning the detection and management of pediatric FH that impact on the developing model of care for this condition.

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Section: Diet and Lifestyle Measures Current Knowledge And Practicementioning

confidence: 99%

Knowns and unknowns in the care of pediatric familial hypercholesterolemia

Martin

Gidding

Wiegman

et al. 2017

Journal of Lipid Research

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“…125 Indeed, early treatment initiation should be considered in severe cases. Although data on the safety (during 3 months) and efficacy of ezetimibe monotherapy in children 6 to 10 years of age diagnosed as having heFH exist, 126 ezetimibe is not approved for use in children younger than 10 years. The only bile acid sequestrant approved for use in children with heFH is colesevelam, 127 with the caveat that it has not been studied in children younger than 10 years or in premenarchal girls.…”

Section: Treatmentmentioning

confidence: 99%

My Approach to the Patient With Familial Hypercholesterolemia

Safarova

Kullo

2016

Mayo Clinic Proceedings

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Familial hypercholesterolemia (FH), a relatively common Mendelian genetic disorder, is associated with a dramatically increased lifetime risk of premature atherosclerotic cardiovascular disease due to elevated plasma low-density lipoprotein cholesterol (LDL-C) levels. The diagnosis of FH is based on clinical presentation or genetic testing. Early identification of patients with FH is of great public health importance because preventive strategies can lower the absolute lifetime cardiovascular risk and screening can detect affected relatives. However, low awareness, detection, and control of FH pose hurdles in the prevention of FH-related cardiovascular events. Of the estimated 0.65 million to 1 million patients with FH in the United States, less than 10% carry a diagnosis of FH. Based on registry data, a substantial proportion of patients with FH are receiving no or inadequate lipid-lowering therapy. Statins remain the mainstay of treatment for patients with FH. Lipoprotein apheresis and newly approved lipid-lowering drugs are valuable adjuncts to statin therapy, particularly when the LDL-C–lowering response is suboptimal. Monoclonal antibodies targeting proprotein convertase subtilisin/kexin type 9 provide an additional approximately 60% lowering of LDL-C levels and are approved for use in patients with FH. For homozygous FH, 2 new drugs that work independent of the LDL receptor pathway are available: an apolipoprotein B antisense oligonucleotide (mipomersen) and a microsomal triglyceride transfer protein inhibitor (lomitapide). This review attempts to critically examine the available data to provide a summary of the current evidence for managing patients with FH, including screening, diagnosis, treatment, and surveillance.

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“…Ezetimibe is a selective cholesterol absorption inhibitor that inhibits the uptake of dietary and biliary cholesterol into the enterocytes. A recent study showed safety and efficacy of ezetimibe monotherapy in young children with HeFH or nonfamilial hypercholesterolemia [11]. However, long-term follow-up is still needed.…”

Section: Current Treatmentmentioning

confidence: 99%