Prevention & lipidsResearch article
Effectiveness and safety of treatment for heterozygous familial hypercholesterolaemia and multifactorial dyslipidaemia in children: an overview of systematic reviews
- Overview of systematic reviews
- AMSTAR-2 appraisal
- GRADE certainty of evidence
Authors' conclusion
In summary, statins are effective lipid-lowering therapy in paediatric FH, with long-term safety requiring further investigation.
Study context
- Population
- Children with heterozygous familial hypercholesterolaemia (heFH) or multifactorial dyslipidaemia (MFD)8 systematic reviews (SRs)
- Comparison
- Lipid-lowering treatments vs placebo or control in children
Main finding
LDL-C, statins vs placebo in heFH · 2-year follow-up
MD
−32.15 %
95% CI −34.9 to −29.4
Statins vs Placebo
MD −32.15 % (95% CI −34.9 to −29.4)
As reported in the abstract
Further findings
| Outcome | MD (mg/dL) (95% CI) | |
|---|---|---|
| LDL-C, ezetimibe (heFH) · 1 RCT, n = 127 | −63 (−79.5 to −46.5) | |
| −100−50050Difference, mg/dL (linear scale) |
| Outcome | MD in change (%) |
|---|---|
| LDL-C change, PCSK9 inhibitors (heFH) — Evolocumab vs Placebo · 1 RCT, n = 157 · moderate CoE | −43.3 |
| LDL-C change, PCSK9 inhibitors (heFH) — Alirocumab vs Placebo · 1 RCT, n = 153 · moderate CoE | −33.8 |
Bile acid sequestrants and fibrates
- Significant lipid-lowering effects; Certainty of evidence (CoE) low/very low
Non-pharmacological interventions (paediatric FH)
- Mainly did not improve lipid profile (CoE very low)
Behavioural counselling (MFD)
- Modest lipid-lowering effects that attenuated over time (2 randomized controlled trials (RCTs); low CoE)
Safety
Adverse events, short-term follow-up
- No significant differences between groups for all drugs
Original publication
Effectiveness and safety of treatment for heterozygous familial hypercholesterolaemia and multifactorial dyslipidaemia in children: an overview of systematic reviews. Eur J Prev Cardiol. Published 30 March 2026. doi:10.1093/eurjpc/zwag125
Summarised from the published abstract; every number is checked against it