Table 1Included studies on statin treatment in children with FH - description (Adapted from published review (Arambepola, C., Farmer, A. J., Perera, R. et al, 2006))

StudyStudy designFollow upCharacteristics of participantsInterventionControlJadad score (quality assessment)changes ± sd in lipid profiles from baseline (mmol/l)changes ± sd in lipid profiles from baseline (mmol/lfunction(mm)
Age rangen (males)Criteria of LDL-C (mmol/l) for inclusion
Wiegman (2004)RCT96w8–18 years214 (100)≥ 4.0Pravastatin 40mg/d if ≥14 y of age; 20mg/d if <14 y of agePlacebo52 year follow-up:
TC: pravastatin 20mg (under 14yrs) and 40mg over 14 years +1.44 (±1.1), p<0.001.
LDL-C: pravastatin 20mg (under 14yrs) and 40mg over 14 years +1.46 (±1.0), p<0.001
HDL-C: pravastatin 20mg (under 14yrs) and 40mg over 14 years +0.03 ns
2 year follow-up: pravastatin 20mg (under 14yrs) and 40mg over 14 years −0.010 (±0.048) p=0.02
de Jongh (2002a)RCT48w10–17 years175 (99)4.9–13.0Simvastatin 10mg/d for 8w; 20mg/d/for 8w; 40 mg/dPlacebo4Week 48:
TC: simvastatin 40mg −30.9% (±11.5);
LDL-C: simvastatin 40mg −40.7% (±39.2)
HDL-C: simvastatin 40mg +3.3% (±14.9).
Stein (1999)RCT48w10–17 years132 (132)≥ 4.9Lovastatin 10mg/d for 8w; 20mg/d for 8w; 40mg/dPlacebo4Week 48:
TC: lovastatin 40mg +0.51 (±0.5), p<0.001 vs placebo;
LDL-C: lovastatin 40mg +0.64 (±0.5), p<0.001 vs placebo;
HDL-C: lovastatin 40mg +0.01 ns
de Jongh (2002b)RCT28w9–18 years50 (26)Above 95th percentile for age and sexSimvastatin10mg/d for 8w; 20mg/d for 8w; 40mg/dPlacebo1Week 28:
TC: simvastatin 40mg −2.16 (±1.04), p=0.0001; LDL-C: simvastatin 40 mg −2.13 (±0.99) p=0.0001;
HDL-C: simvastatin 40 mg −0.05 (±0.17) p=0.08.
Week 28:
FMD significant increase in simvastatin FH group (p<0.0001).
McCrindle (2003)RCT26w10–17 years187> 4.1Atorvastatin 10mg/d; 20mg/d if LDL-C ≥ 3.4 at week 4Placebo3Week 26:
TC: atorvastatin 10–20mg titrated depending upon response, −31.4% (± 1.0);
LDL-C: atorvastatin 10–20mg titrated depending upon response, −39.6% (± 1.1);
HDL-C: atorvastatin 10–20mg titrated depending upon response, +2.8% (±1.3);
Clauss (2005)RCT24w10–17 years post menarche females54 (0)4.1–10.3Lovastatin 20mg/d for 4w; 40 mg/dPlacebo5Week 24:
TC: lovastatin 40mg −21.8% (±2.5);
LDL-C: lovastatin 40mg −26.8% (±3.4);
HDL-C: lovastatin 40mg +2.5% (±2.5);
Knipscheer (1996)RCT (4 randomised arms)12w8–16 years72 (25)Above 95th percentile for age and sexPravastatin:
  1. 5 mg/d
  2. 10 mg/d
  3. 20 mg/d
Placebo3Week 12:
TC: pravastatin 20mg −24.6% (95% CI 21.0 to 28.1);
LDL-C: pravastatin 20mg −32.9% (95% CI 28.6 to 37.0);
HDL-C: pravastatin 20mg + 10.8% mean change (95% CI 3.4 to 18.8).
Couture (1998)RCT6w8–17 years63 (37)Above 95th percentile for age and sexSimvastatin 20 mg/d (for 3 groups according - gene mutations)Placebo3
McCrindle (2002)Randomised cross over trial18w8–18 years40 (25)> 4.15Pravastatin 10mg/d + colestipol5g/dColestipol 10g/d-Week 18:
TC: colestipol 10g only −0.63±0.80; colestipol 5g + pravastatin 10mg −1.06±1.11 p=0.041;
LDL-C: colestipol 10g only −0.65±0.80; colestipol 5g + pravastatin 10mg −1.07± 1.06 p=0.066;
HDL-C: colestipol 10g only −0.01±0.18; colestipol 5g + pravastatin 10mg +0.03± 0.13 p=0.63;
Stefanutti (2005)Non-randomised parallel matched trial48w4–11 years16 (7)Not statedSimvastatin 10mg/d + step II AHA dietStep II AHA diet-Month 12
TC: simvastatin 10mg −24%;
LDL-C: simvastatin 10mg −29% p<0.01;
HDL-C: simvastatin 10mg +7% (no sd reported)
Lambert (1996)Time series comparison (4 randomised arms)8w≤ 17 years69 (69)Above 95th percentile for age and sexLovastatin:
  1. 10 mg/d
  2. 20 mg/d
  3. 30 mg/d
  4. 40 mg/d
Placebo/4w prior to randomisation-Week 8:
TC: lovastatin 40mg +29% (26–32);
LDL-C: lovastatin 40mg +36% (33–39);
HDL-C: lovastatin 40mg +3%

From: 5, Management (pharmacological treatment)

Cover of Identification and Management of Familial Hypercholesterolaemia (FH)
Identification and Management of Familial Hypercholesterolaemia (FH) [Internet].
NICE Clinical Guidelines, No. 71.
National Collaborating Centre for Primary Care (UK).
Copyright © 2008, Royal College of General Practitioners.

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