ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: Cochrane Database Syst Rev. 2019;2019(11). doi: 10.1002/14651858.CD006401.pub5

Short summary: This systematic review shows that statin treatment is an effective lipid-lowering therapy in children with Familial Hypercholesterolemia (FH). Few or no safety issues were identified.

Comment: Cochrane is an international charitable organisation that was formed to inform health decision-making about treatments. A global independent network of researchers provide systematic reviews and meta-analyses that facilitate evidence-based choices about health.

The current study assessed the effectiveness and safety of statin use in children with heterozygous FH. Nine randomized placebo-controlled studies with data on 1177 participants were included. There is high-quality evidence that statins reduce mean LDL-C concentration at all time points. The important take-home message is that differences in frequency of adverse effects, such as myopathy and impaired liver function, are small or non-existent between the treatment and placebo groups. None of the studies reported rhabdomyolysis or clinical adverse events.

The reviewers noted that the intervention and follow-up periods of included studies were short (median 24 weeks; range of 6 weeks to 2 years). Nevertheless, a few documentations reported improvement in the function of the arterial endothelium in children with FH, as well as in carotid intima-media thickness. This review should encourage paediatricians to screen children for hyperlipidaemia and refer them for treatment if needed.