Statins for children with familial hypercholesterolemia

Vuorio, Alpo; Kuoppala, Jaana; Kovanen, Petri T.; Humphries, Steve E.; Tonstad, Serena; Wiegman, Albert; Drogari, Euridiki; Ramaswami, Uma

doi:10.1002/14651858.cd006401.pub5

Cited by 61 publications

(57 citation statements)

References 111 publications

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“…The short-and intermediate-term efficacy and tolerance of statins in children and adolescents have been confirmed by observational studies and meta-analyses [25][26][27]. Statin treatment is generally well tolerated, with good adherence to treatment [28].…”

Section: Children and Adolescentsmentioning

confidence: 91%

The Knowns and Unknowns of Contemporary Statin Therapy for Familial Hypercholesterolemia

Pang

Chan

Watts

2020

Curr Atheroscler Rep

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Purpose of Review Statins are first-line therapy for lowering low-density lipoprotein (LDL) cholesterol in familial hypercholesterolemia (FH), particularly in heterozygous patients. We review advances and new questions on the use of statins in FH. Recent Findings Cumulative evidence from registry data and sub-analyses of clinical trials mandates the value of statin therapy for prevention of atherosclerotic cardiovascular disease (ASCVD) in FH. Statins are safe in children and adolescents with FH, with longer term cardiovascular benefits. The potentially toxic effects of statins in pregnancy need to be considered, but no association has been reported in prospective cohort studies with birth defects. There is no rationale for discontinuation of statins in elderly FH unless indicated by adverse events. FH is undertreated, with > 80% of statin-treated FH patients failing to attain LDL cholesterol treatment targets. This may relate to adherence, tolerability, and genetic differences in statin responsiveness. Statin treatment from childhood may reduce the need for stringent cholesterol targets. Combination of statins with ezetimibe and PCSK9 inhibitors significantly improves the efficacy of treatment. Whether statin use could improve the clinical course of FH patients with COVID-19 and other respiratory infections remains an unsolved issue for future research. Summary Statins are the mainstay for primary and secondary prevention of ASCVD in FH. Sustained long-term optimal statin treatment from an early age can effectively prevent ASCVD over decades of life. Despite their widespread use, statins merit further investigation in FH.

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Section: Children and Adolescentsmentioning

confidence: 91%

The Knowns and Unknowns of Contemporary Statin Therapy for Familial Hypercholesterolemia

Pang

Chan

Watts

2020

Curr Atheroscler Rep

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“…If left untreated, the severe hypercholesterolemia causes pre-mature atherosclerosis. The standard treatment in HeFH children is statin therapy, which should start when the child is between 8 and 12 years of age (4). Homozygous familial hypercholesterolemia (HoFH) is the severe form of familial hypercholesterolemia (FH) affecting approximately 1 in 300,000 persons worldwide and causing four-to five-fold elevated levels of serum LDL-C (5).…”

Section: Introductionmentioning

confidence: 99%

Hospitalized Children With Familial Hypercholesterolemia and COVID-19: A Case for Preventive Anticoagulation

Vuorio

Raal

Kovanen

2021

Front. Cardiovasc. Med.

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“…The homozygous variant generally debuts in the first decade of life with a very high risk of death from coronary heart disease before the age of 30. Since its two alleles have to be mutated, this variant is extremely rare with a prevalence of 1 case per million inhabitants who tend to have extremely high calculated LDL (LDL-C) levels between 800 and 1000 mg/dl; hence, these variants lead to greater cardiovascular risk [5,6]. The heterozygous phenotype is the most common variant with a prevalence in the Caucasian population of 1 case per 500 inhabitants, which gives an approximate estimate of about 10 million affected in the world, the majority of whom (up to 80%) remain undiagnosed.…”

Section: Introductionmentioning

confidence: 99%

Reducción de eventos cardiovasculares de Lomitapida versus Estatinas en pacientes con diagnóstico de hipercolesterolemia familiar. Protocolo de revisión sistemática.

Valdez

Avila

Espinoza³

et al. 2021

REV-SEP

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Introducción: La hipercolesterolemia familiar (HF) un trastorno genético autosómico dominante que produce hipercolesterolemia y desarrollo prematuro de enfermedades cardiovasculares. Las estatinas han sido el medicamento de elección en estos pacientes, sin embargo, un buen porcentaje de pacientes no pueden alcanzar sus objetivos terapéuticas con las dosis máximas recomendadas por lo que la Lomitapida se podría establecer como una nueva alternativa de tratamiento. Objetivo: El objetivo de esta revisión sistemática es determinar si la Lomitapida reduce los eventos cardiovasculares en pacientes con diagnóstico de Hipercolesterolemia familiar comparado con estatinas. Métodos: Se incluirán ensayos controlados aleatorios (ECA) y cuasialeatorios de pacientes con diagnóstico de HF. Las medidas de resultado primarias: 1. Niveles de LDL, HDL pos tratamiento. 2. Presencia de eventos cardiovasculares. Las búsquedas electrónicas se realizarán en PUBMED, The Cochrane Central Register of Controlled Trials (CENTRAL), EMBASE y Scientific electronic library (Scielo). La evaluación del riesgo de sesgo se utilizará la herramienta de Cochrane. Las medidas del efecto del tratamiento serán las diferencias de medias (DM) y los intervalos de confianza (IC) del 95%. La evaluación de heterogeneidad se realizará mediante la inspección visual del diagrama de embudo. La evaluación de la calidad de la evidencia se realizará usando la evaluación GRADE.

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Statins for children with familial hypercholesterolemia

Cited by 61 publications

References 111 publications

The Knowns and Unknowns of Contemporary Statin Therapy for Familial Hypercholesterolemia

The Knowns and Unknowns of Contemporary Statin Therapy for Familial Hypercholesterolemia

Hospitalized Children With Familial Hypercholesterolemia and COVID-19: A Case for Preventive Anticoagulation

Reducción de eventos cardiovasculares de Lomitapida versus Estatinas en pacientes con diagnóstico de hipercolesterolemia familiar. Protocolo de revisión sistemática.

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