Findings

What do systematic reviews say?

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Evidence found and GRADE certainty

13 questions · 13 findings · 2 reviews · Cystic Fibrosis and Genetic Disorders

No studies found 2 · 15.4%GRADE not reported 0 · 0%Very low 3 · 23.1%Low 6 · 46.2%Moderate 1 · 7.7%High 1 · 7.7%

Statins with cholesterol supplementation vs cholesterol supplementation only

Statin‐related adverse events · children and adults with Smith‐Lemli‐Opitz syndrome

It is very uncertain whether statins with cholesterol supplementation changes statin‐related adverse events compared with cholesterol supplementation only (very low certainty).

1 finding from 1 review

Statins with cholesterol supplementation vs cholesterol supplementation only

Changes in biochemical markers plasma dehydrocholesterol levels (mM); plasma cholesterol levels (mM); plasma 7DHC levels (mM); plasma CoQ levels (uM); CSF cholesterol levels (ug/mL); CSF dehydrocholesterol levels (ug/mL) · children and adults with Smith‐Lemli‐Opitz syndrome
1 finding from 1 review

Intervention not reported

Change in carotid intima‐media thickness (mm) ‐ at 2 years · children with familial hypercholesterolemia
1 finding from 1 review

Intervention not reported

Change in serum LDL cholesterol level (%) ‐ at end of · children with familial hypercholesterolemia
1 finding from 1 review

Intervention not reported

Change in measures of growth and maturation: change in puberty proportion with Tanner stage ≧ 1 level ‐ at 2 years · children with familial hypercholesterolemia

The intervention showed no clear difference in change in measures of growth and maturation: change in puberty… (low certainty).

1 finding from 1 review

Intervention not reported

Liver dysfunction: proportion with changed aspartate aminotransferase or alanine aminotransferase levels (> 3x ULN) ‐ at all time points · children with familial hypercholesterolemia
1 finding from 1 review

Intervention not reported

Myopathy: proportion with changed serum creatine kinase levels (>10x ULN) ‐ at all time points · children with familial hypercholesterolemia
1 finding from 1 review

Intervention not reported

Change in endothelial function: Change in flow‐mediated dilatation of brachial artery (%) · children with familial hypercholesterolemia
1 finding from 1 review

Intervention not reported

Adverse events ‐ at one year · children with familial hypercholesterolemia

The intervention showed no clear difference in adverse events ‐ at one year (moderate certainty).

1 finding from 1 review