Pediatric familial hypercholesterolemia: meta-analysis finds statins are effective and safe

  • Anagnostis P & al.
  • Endocrine
  • 24 Apr 2020

  • curated by Emily Willingham, PhD
  • Clinical Essentials
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Takeaway

  • For pediatric familial hypercholesterolemia, statins are safe and effective according to the results of this meta-analysis of 10 randomized controlled trials.

Why this matters

  • Statins are approved in the United States and Europe for pediatric use for ages 10 years and up.
  • These authors say that nevertheless, their efficacy and safety for this population “are not well documented.”

Key results

  • Vs placebo, statins were linked to average relative reductions (95% CIs) in:
    • Total cholesterol: −25.5% (−30.4% to −20.5%; I2, 91%).
    • Low-density lipoprotein cholesterol: −33.8% (−40.1% to −27.4%; I2, 90%).
    • Triglycerides: −8.4% (−14.8% to −2.03%; I2, 26%).
    • Apolipoprotein B: −28.8% (−33.9% to −23.6%; I2, 83%).
  • They were linked to increases in high-density lipoprotein cholesterol:
    • 3.1% (1.1%-5.2%; I2, 0%).
  • Biomarkers (liver, muscle) did not differ with statin vs placebo.
  • Adverse event incidence was similar between placebo, treatment (
  • The analysis showed no effects on sexual development (Tanner stage) or growth.

Study design

  • Meta-analysis of 10 randomized controlled trials with 1191 participants; ages, 13.3 ± 2.5 years.
  • Funding: None disclosed.

Limitations

  • Statin types could not be compared head to head or dose to dose.
  • Some missing data were imputed.