Takeaway
- In paediatric familial hypercholesterolaemia, 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 referral to an adult endocrinologist at age 18 years.
Why this matters
- Statins 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 randomised 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
- Randomised, placebo-controlled studies with paediatric 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.
References
References