Department of Endocrinology, Morbid Obesity and Preventive Medicine, Lipid Clinic, Oslo University Hospital, Oslo, Norway.
Pfizer Inc, New York, NY, USA.
J Clin Lipidol. 2016 Sep-Oct;10(5):1153-1162.e3. doi: 10.1016/j.jacl.2016.05.010. Epub 2016 Jun 7.
The efficacy and safety of atorvastatin in children/adolescents aged 10-17 years with heterozygous familial hypercholesterolemia (HeFH) have been demonstrated in trials of up to 1 year in duration. However, the efficacy/safety of >1 year use of atorvastatin in children/adolescents with HeFH, including children from 6 years of age, has not been assessed.
To characterize the efficacy and safety of atorvastatin over 3 years and to assess the impact on growth and development in children aged 6-15 years with HeFH.
A total of 272 subjects aged 6-15 years with HeFH and low-density lipoprotein cholesterol (LDL-C) ≥4.0 mmol/L (154 mg/dL) were enrolled in a 3-year study (NCT00827606). Subjects were initiated on atorvastatin (5 mg or 10 mg) with doses increased to up to 80 mg based on LDL-C levels.
Mean percentage reductions from baseline in LDL-C at 36 months/early termination were 43.8% for subjects at Tanner stage (TS) 1 and 39.9% for TS ≥2. There was no evidence of variations in the lipid-lowering efficacy of atorvastatin between the TS groups analyzed (1 vs ≥2) or in subjects aged <10 vs ≥10 years, and the treatment had no adverse effect on growth or maturation. Atorvastatin had a favorable safety and tolerability profile, and only 6 (2.2%) subjects discontinued because of adverse events.
Atorvastatin over 3 years was efficacious, had no impact on growth/maturation, and was well tolerated in children and adolescents with HeFH aged 6-15 years.
阿托伐他汀在年龄为 10-17 岁的杂合子家族性高胆固醇血症（HeFH）儿童/青少年中的疗效和安全性已在持续 1 年的试验中得到证实。然而，尚未评估 HeFH 儿童/青少年（包括 6 岁以上的儿童）使用阿托伐他汀超过 1 年的疗效/安全性。
评估阿托伐他汀治疗 HeFH 儿童/青少年（6-15 岁）超过 3 年的疗效和安全性，并评估其对生长和发育的影响。
共纳入 272 名 HeFH 且低密度脂蛋白胆固醇（LDL-C）≥4.0mmol/L（154mg/dL）的 6-15 岁儿童/青少年入组 3 年研究（NCT00827606）。根据 LDL-C 水平，受试者起始剂量为阿托伐他汀 5mg 或 10mg，最大剂量可增至 80mg。
在 36 个月/提前终止时，Tanner 分期（TS）1 期和≥2 期受试者的 LDL-C 自基线的平均百分比降幅分别为 43.8%和 39.9%。未发现降脂疗效在 TS 组之间（1 与≥2）或在<10 岁与≥10 岁的受试者之间存在差异，且治疗对生长或成熟无不良影响。阿托伐他汀具有良好的安全性和耐受性，仅有 6 名（2.2%）受试者因不良事件而停药。
阿托伐他汀治疗 HeFH 儿童/青少年（6-15 岁）超过 3 年有效，对生长/成熟无影响，且耐受性良好。
