Paediatric familial hypercholesterolaemia: statins remain a safe short-term choice

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.