TABLE 9-11

Clinical Trials of Lipid-Lowering Medication Therapy in Children and Adolescents

Study Authors, Type, and DurationMedicationNo. of Subjects, Gender, ConditionDaily DoseEffect on Lipid Profile, %
TCLDL CholesterolHDL CholesterolTriglycerides
Bile acid–binding resins
    Tonstad et al, RCT, 1 yCholestyramine72, male and female, FH (LDL ≥ 190 mg/dL without family history of premature CVD or LDL ≥ 160 with family history after 1-y diet; ages 6–11 y)8 g−12−178NA
    McCrindle et al, RCT crossover, 2 × 8 wkCholestyramine40, male and female, FH (1 parent with FH; LDL cholesterol ≥ 131 mg/dL; on diet; ages 10–18 y)8 g−7 to −11−10 to −152 to 46 to 9
    Tonstad et al, RCT, 8 wk; open label, 44–52 wkColestipol66, male and female, FH (TC ≥ 239 mg/dL and triglycerides ≤ 115 mg/dL; ages 10–16 y)2–12 g−17−20−7−13
    McCrindle et al, RCT crossover, 2 × 18 wkColestipol36, male and female, FH/FCHL (LDL ≥ 160 mg/dL after 6 mo of diet counseling; ages 8–18 y)10 g−7−10212
    Stein et al, RCT, 8 wk; open label, 18 wkColesevelam191, male and female, FH (LDL ≥ 190 mg/dL or LDL ≥ plus 2 additional risk factors after 6 mo of diet counseling; ages 10–17 y1.87 g−3−656
3.75 g−7−1385
HMG-CoA reductase inhibitors (statins)
    McCrindle et al, RCT; open label, 26 wkAtorvastatin187, male and female, FH/severe hyperlipidemia (LDL cholesterol ≥ 190 mg/dL or LDL cholesterol ≥ 160 mg/dL with family history; triglycerides < 400 mg/dL; ages 10 – 17 y)10–20 mg−30−406−13
    Van der Graaf et al, open label, 2 yFluvastatin85, male and female, FH (LDL cholesterol ≥ 190 mg/dL or LDL cholesterol ≥ 160 mg/dL and ≥1 risk factor or LDL receptor mutation; ages 10–16 y)80 mg−27−345−5
    Lambert et al, RCT, 8 wkLovastatin69, male, FH (LDL cholesterol > 95th percentile, family history of atherosclerosis and hyperlipidemia; on diet; mean age: 13 y)10 mg−17−219−18
20 mg−19−2429
30 mg−21−27113
40 mg−29−363−9
    Stein et al, RCT, 48 wkLovastatin132, male, FH (LDL 189–503 mg/dL + family history of high LDL; or 220–503 mg/dL + family history of CAD death; AHA diet ≥4 mo; ages 10–17 y)10 mg−13−1744
20 mg−19−2448
40 mg−21−2756
    Clauss et al, RCT, 24 wkLovastatin54, female, FH (family history of FH; LDL 160–400 mg/dL and triglycerides < 350 mg/dL; 4-wk diet placebo run-in and 20-wk treatment; ages 10–17 y, postmenarcheal)40 mg−22−273−23
    Knipscheer et al, RCT, 12 wkPravastatin72, male and female, FH (family history hypercholesterolemia or premature atherosclerosis; LDL > 95th percentile; diet for 8 wk; ages 8–16 y)5 mg−18−2342
10 mg−17−2467
20 mg−25−33113
    Wiegman et al, RCT, 2 yPravastatin214, male and female, FH (LDL cholesterol ≥ 155 mg/dL and triglycerides ≤ 350 mg/dL; diet for 3 mo; ages 8–18 y)20–40 mg−19−246−17
    Rodenburg et al, open-label, 2-y RCT; 4.5-y open-label follow-upPravastatin186, male and female, FH (LDL cholesterol ≥ 154 mg/dL and triglycerides < 154 mg/dL; diet for 3 mo; ages 8–18 y)20 mg (ages <14 y) or 40 mg (ages ≥ 14 y)−23−293−2
    de Jongh et al, RCT, 48 wkSimvastatin173, male and female, FH (LDL cholesterol = 158–397 mg/dL; ages 10–17 y)10–40 mg−31−413−9
    de Jongh et al, RCT, 28 wkSimvastatin50, male and female, FH (LDL cholesterol > 95th percentile, family history of hyperlipidemia, or LDL receptor mutation; ages 9–18 y)40 mg−30−405−17
    Avis et al, RCT, 12 wk; then, 40-wk open-label follow-upRosuvastatin177, male and female, FH (LDL cholesterol ≥ 190 mg/dL or LDL cholesterol > 160 mg/dL plus positive family history of early CVD or ≥2 other risk factors for CVD)5 mg−30−384−13
10 mg−34−4510−15
20 mg−39−509−16
Other agents
    Wheeler et al, RCT, 26 wkBezafibrate14, male and female, FH (TC > 269 mg/dL, normal triglycerides + family history of FH or premature CAD; ages 4–15 y)10–20 mg−22NC15−23
    Colletti et al, open label, 1–19 moNiacin21, male and female, FH (mean LDL = 243 ± 45 mg/dL on low-fat diet; mean triglycerides = 87 ± 39 mg/dL; ages 4–14 y)500–2200 mg−13−17413
    McCrindle et al, RCT crossover, 2 × 18 wkPravastatin and colestipol36, male and female, FH/FCHL (LDL > 160 mg/dL + family history of FH or premature CAD; triglycerides > 177 mg/dL in 10 of the 36; ages 10–18 y)Pravastatin, 10 mg (with colestipol, 5g)−13−1748
    van der Graaf et al, RCT, 6 and 27 wk; open label to 53 wkSimvastatin and ezetimibe248, male and female, FH (LDL > 159 mg/dL + genotype-confirmed FH or + parental genotype-confirmed FH or + parental LDL > 210 mg/dL or + tendinous xanthomas or LDL > 189 mg/dL + family history of hypercholesterolemia; ages 10–17 y)Simvastatin 10–40 mg (with ezetimibe, 10 mg)−38−497−17
Addendum
    Goldberg et al, ω-3 fatty acid review in adults; no RCTs in childrenω-3 fish oils (1 g per capsule)a1–4 g/dNC17 to 316 to 17−30 to −40
  • FH indicates heterozygous familial hypercholesterolemia; NA, not available; FCHL, familial combined hyperlipidemia; HMG-CoA, hydroxymethylglutaryl coenzyme A; CAD, coronary artery disease; NC, not calculated.

  • a There is only one FDA-approved fish-oil preparation, but there are many generic forms of fish-oil capsules that are commercially available. The University of Wisconsin maintains a preventive cardiology patient education Web site (www.heartdecision.org). The fish-oil section includes information about the content of various preparations. The Web site is updated every 6 months (www.heartdecision.org/chdrisk/v_hd/patient_edu_docs/Fish_Oil_11-2007.pdf).

  • Adapted from McCrindle BW, Urbina EM, Dennison BA, et al; American Heart Association Atherosclerosis, Hypertension, and Obesity in Youth Committee; American Heart Association, Council of Cardiovascular Disease in the Young; American Heart Association, Council on Cardiovascular Nursing. Circulation. 2007;115(14):1948–1967.