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Statin treatment for children and adolescents with heterozygous familial hypercholesterolaemia: A systematic review and meta-analysis

  • C. Arambepola
    Affiliations
    Division of Public Health and Primary Health Care, University of Oxford, Oxford OX3 7LF, UK
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  • A.J. Farmer
    Affiliations
    Division of Public Health and Primary Health Care, University of Oxford, Oxford OX3 7LF, UK
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  • R. Perera
    Affiliations
    Division of Public Health and Primary Health Care, University of Oxford, Oxford OX3 7LF, UK
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  • H.A.W. Neil
    Correspondence
    Corresponding author at: Division of Public Health and Primary Health Care, University of Oxford, Rosemary Rue Building, Old Road Campus, Headington, Oxford OX3 7LF, UK. Tel.: +44 1865 226777; fax: +44 1865 226777.
    Affiliations
    Division of Public Health and Primary Health Care, University of Oxford, Oxford OX3 7LF, UK

    Oxford Centre for Diabetes, Endocrinology & Metabolism, The Churchill Hospital, Oxford OX3 7LJ, UK
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      Abstract

      Aims

      To assess efficacy and safety of HMG-CoA reductase inhibitor (statin) treatment in children and adolescents with heterozygous familial hypercholesterolaemia.

      Methods

      MEDLINE, EMBASE, COCHRANE and Current Controlled Trials databases were searched. Study design, efficacy, and safety outcome-measures were extracted. Results of parallel-group randomised placebo controlled trials with low density (LDL) and high density lipoprotein cholesterol (HDL), and triglycerides as outcomes were pooled using standard meta-analytical methods.

      Results

      One hundred and fifty seven of 1060 identified papers studied familial hypercholesterolaemia, and 18 papers reported 7 prospective case series, 1 non-randomised trial, 2 trials with active treatment control groups, and 8 parallel-group randomised placebo controlled trials (RCT). The RCTs randomised 947 children, aged 8–18 years, for periods of 6–96 weeks with an estimated 850 person-years follow-up. There were no differences in clinical or laboratory adverse reactions between placebo and active treatment. Statins lowered LDL 32.5% (95% CI 24.3, 40.7), increased HDL 3.4% (0.8, 6.0), lowered triglycerides 3.0% (−11.6, 17.6), attenuated progression of carotid medial thickness, and improved endothelial function.

      Conclusions

      Statin monotherapy is efficacious, well tolerated and safe in the short-term, although long-term safety remains unclear. Current evidence supports treatment of children at highest cardiovascular risk, but results of on-going, longer-term studies may extend these indications.

      Keywords

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