Pediatric familial hypercholesterolemia: statins remain a safe short-term choice

  • Vuorio A & al.
  • Cochrane Database Syst Rev
  • 07/11/2019

  • Emily Willingham, PhD
  • Clinical Essentials
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Takeaway

  • In pediatric familial hypercholesterolemia, statins remain a safe short-term therapy, but long-term safety is still not established.
  • The authors of this Cochran review recommend careful monitoring and follow-up and assurance of referral to adult lipidologist at age 18 years.

Why this matters

  • These authors note that if there were any safety issues identified, that would militate against using statins, which are not acutely life-saving in this population.
  • This review updates the 2017 version with the addition of a new reference but no changes in conclusion.

Key results

  • 9 randomized placebo-controlled trials included with 1177 participants.
  • Intervention duration, follow-up ranged from 6 weeks to 2 years, with a median of 24 weeks.
  • Statins effectively reduced cholesterol.
  • No liver function effects, myopathy, rhabdomyolysis, or adverse events greater than placebo.

Study design

  • Randomized, placebo-controlled studies with pediatric participants (aged 4-18 years) included, with evidence covering up to November 4, 2019.
  • Study duration, 12-104 weeks.
  • Funding: Finnish Office for Health Technology Assessment; National Institute for Health Research, UK; others.

Limitations

  • The limitations are those of the included studies, especially a lack of long-term data.