Familial hypercholesterolemia “is a serious condition that needs to be detected and treated early in life. Unless treated in childhood it is likely the patient will not receive treatment until some cardiovascular event occurs in early- to mid-adult life (if they survive the event),” senior author Dr. Evan A. Stein, from the Metabolic and Atherosclerosis Research Center in Cincinnati, Ohio, told Reuters Health by email.
In a placebo-controlled trial, Dr. Stein and his colleagues analyzed the efficacy and safety of rosuvastatin in 177 children ages 10 to 17 years with familial hypercholesterolemia.
During the 12-week double-blind phase of the trial, LDL cholesterol declined from baseline by 38% with rosuvastatin 5 mg, 45% with rosuvastatin 10 mg, and 50% for rosuvastatin 20 mg, compared with only 1% in the placebo group.
Response patterns were similar for total cholesterol and apolipoprotein B, but changes in triglycerides, HDL cholesterol, and apolipoprotein A-I did not differ significantly between the rosuvastatin and placebo groups.
Twelve percent, 41%, and 41% of the patients treated with rosuvastatin 5, 10, and 20 mg, respectively, achieved the LDL cholesterol target of less than 110 mg/dL. Later, during a 40-week open-label extension phase, 40% of participants reached this goal.
Just over two-thirds of the children (68%) met the secondary LDL cholesterol goal of less than 130 mg/dL during the open-label phase.
Roughly 90% of patients in the double-blind phase and roughly 60% in the open-label phase were at least 80% compliant with the medication regimen.
Adverse events occurred with similar frequency in all groups, and no hepatic, skeletal muscle, or renal adverse events led to permanent treatment discontinuation. In each group, at least 50% of children had adverse events, however, with the rate reaching 64% with the highest rosuvastatin dose. Headache was the most common complaint.
“This trial, along with other studies in nearly 1000 pediatric patients, confirms that LDL cholesterol lowering with statins is well tolerated in adolescents with familial hypercholesterolemia and, despite significant LDL cholesterol reductions, highlights the difficulty in achieving optimal goals in these patients,” the investigators conclude.
Dr. Stein said that together with Dr. John Kastelein and other investigators in Europe and Canada, he and his colleagues have just launched a 2-year trial in 6- to 17-year-olds with familial hypercholesterolemia. The researchers plan to monitor the kids with annual carotid ultrasonography to see whether reducing LDL cholesterol to 110 mg/dl or below (by titrating the rosuvastatin dose up to 20 mg) will result in carotid intima media thickness consistent with that in children without the condition.
Familial hypercholesterolemia “is one of the most common genetic disorders in our society, far more common than Type I diabetes or AIDS, is far easier and less expensive to treat and much more likely if treated to completely prevent the associated CVD events,” Dr. Stein said.
The study was supported by AstraZeneca, which markets rosuvastatin as Crestor.
J Am Coll Cardiol 2010;55:1121-1126.