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