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Statins started in childhood for patients with familial hypercholesterolemia reduced progression of carotid thickening and cut the risk of cardiovascular disease 20 years later, according to authors of a long-term follow-up study.
There were no deaths and one cardiovascular event by the age of 39 years for patients in the observational study who had, as children, participated in a placebo-controlled, 2-year safety and efficacy study of pravastatin.
These positive effects on disease and a disease marker (carotid intima-media thickness) were observed even though only 20% of patients met LDL cholesterol goals, according to study senior authors John J.P. Kastelein, MD, PhD, and Barbara A. Hutten, PhD, of Amsterdam University Medical Centers in the Netherlands, and their colleagues.
“If corroborated, such findings would underscore the current pediatric guidelines, which recommend starting treatment from the age of 8 years or 10 years, with less-stringent targets than those for adults,” the investigators wrote in the report, which was published in the New England Journal of Medicine.
The study was based in part on follow-up visits with 184 of the original cohort of 214 patients with familial hypercholesterolemia in the 2-year pravastatin study, along with 77 of 95 unaffected siblings who had served as a control group in that study.
At the time of the 20-year follow-up, 79% of the familial hypercholesterolemia patients said they were using lipid-lowering medication, the investigators wrote.
The mean LDL cholesterol level at follow-up was 160.7 mg/dL for those with familial hypercholesterolemia, a drop of 32% when compared with the mean LDL cholesterol level at baseline in the original study, according to the investigators. By contrast, LDL cholesterol in the unaffected siblings was 121.9 mg/dL, up 24% from baseline.
Just 37 patients with familial hypercholesterolemia, or about 20%, reached the LDL cholesterol treatment target of less than 100 mg/dL, the investigators wrote.
At the start of the original trial, carotid intima-media thickness was greater in patients with familial hypercholesterolemia, compared with their unaffected siblings, with a mean difference of 0.012 mm (95% confidence interval, 0.002-0.021) after adjustment for age and sex.
Some 20 years later, the mean difference in thickness for patients with familial hypercholesterolemia and unaffected siblings, was 0.555 mm and 0.551 mm, respectively, for a mean difference of just 0.008 mm (95% CI, –0.009 to 0.026) after adjustments.
Data on cardiovascular events and deaths for affected parents was collected, as each child in the study had a parent with confirmed familial hypercholesterolemia, the investigators wrote.
A total of 7% of affected parents had died of MI before the age of 40 years, whereas there were no deaths from cardiovascular causes in all 214 patients with familial hypercholesterolemia from the original study.
Similarly, about a quarter of affected parents had a cardiovascular event before age 40, whereas there was only one event recorded in the patients in the study. That event, angina pectoris resulting in percutaneous coronary intervention, occurred in a patient who stopped taking the drug at the end of the trial, the investigators wrote.
The study was supported by a grant from the AMC Foundation. The study authors reported disclosures related to numerous pharmaceutical companies and government, nonprofit, or academic entities.
SOURCE: Kastelein JJP et al. N Engl J Med. 2019;381:1547-56.
Statins started in childhood for patients with familial hypercholesterolemia reduced progression of carotid thickening and cut the risk of cardiovascular disease 20 years later, according to authors of a long-term follow-up study.
There were no deaths and one cardiovascular event by the age of 39 years for patients in the observational study who had, as children, participated in a placebo-controlled, 2-year safety and efficacy study of pravastatin.
These positive effects on disease and a disease marker (carotid intima-media thickness) were observed even though only 20% of patients met LDL cholesterol goals, according to study senior authors John J.P. Kastelein, MD, PhD, and Barbara A. Hutten, PhD, of Amsterdam University Medical Centers in the Netherlands, and their colleagues.
“If corroborated, such findings would underscore the current pediatric guidelines, which recommend starting treatment from the age of 8 years or 10 years, with less-stringent targets than those for adults,” the investigators wrote in the report, which was published in the New England Journal of Medicine.
The study was based in part on follow-up visits with 184 of the original cohort of 214 patients with familial hypercholesterolemia in the 2-year pravastatin study, along with 77 of 95 unaffected siblings who had served as a control group in that study.
At the time of the 20-year follow-up, 79% of the familial hypercholesterolemia patients said they were using lipid-lowering medication, the investigators wrote.
The mean LDL cholesterol level at follow-up was 160.7 mg/dL for those with familial hypercholesterolemia, a drop of 32% when compared with the mean LDL cholesterol level at baseline in the original study, according to the investigators. By contrast, LDL cholesterol in the unaffected siblings was 121.9 mg/dL, up 24% from baseline.
Just 37 patients with familial hypercholesterolemia, or about 20%, reached the LDL cholesterol treatment target of less than 100 mg/dL, the investigators wrote.
At the start of the original trial, carotid intima-media thickness was greater in patients with familial hypercholesterolemia, compared with their unaffected siblings, with a mean difference of 0.012 mm (95% confidence interval, 0.002-0.021) after adjustment for age and sex.
Some 20 years later, the mean difference in thickness for patients with familial hypercholesterolemia and unaffected siblings, was 0.555 mm and 0.551 mm, respectively, for a mean difference of just 0.008 mm (95% CI, –0.009 to 0.026) after adjustments.
Data on cardiovascular events and deaths for affected parents was collected, as each child in the study had a parent with confirmed familial hypercholesterolemia, the investigators wrote.
A total of 7% of affected parents had died of MI before the age of 40 years, whereas there were no deaths from cardiovascular causes in all 214 patients with familial hypercholesterolemia from the original study.
Similarly, about a quarter of affected parents had a cardiovascular event before age 40, whereas there was only one event recorded in the patients in the study. That event, angina pectoris resulting in percutaneous coronary intervention, occurred in a patient who stopped taking the drug at the end of the trial, the investigators wrote.
The study was supported by a grant from the AMC Foundation. The study authors reported disclosures related to numerous pharmaceutical companies and government, nonprofit, or academic entities.
SOURCE: Kastelein JJP et al. N Engl J Med. 2019;381:1547-56.
Statins started in childhood for patients with familial hypercholesterolemia reduced progression of carotid thickening and cut the risk of cardiovascular disease 20 years later, according to authors of a long-term follow-up study.
There were no deaths and one cardiovascular event by the age of 39 years for patients in the observational study who had, as children, participated in a placebo-controlled, 2-year safety and efficacy study of pravastatin.
These positive effects on disease and a disease marker (carotid intima-media thickness) were observed even though only 20% of patients met LDL cholesterol goals, according to study senior authors John J.P. Kastelein, MD, PhD, and Barbara A. Hutten, PhD, of Amsterdam University Medical Centers in the Netherlands, and their colleagues.
“If corroborated, such findings would underscore the current pediatric guidelines, which recommend starting treatment from the age of 8 years or 10 years, with less-stringent targets than those for adults,” the investigators wrote in the report, which was published in the New England Journal of Medicine.
The study was based in part on follow-up visits with 184 of the original cohort of 214 patients with familial hypercholesterolemia in the 2-year pravastatin study, along with 77 of 95 unaffected siblings who had served as a control group in that study.
At the time of the 20-year follow-up, 79% of the familial hypercholesterolemia patients said they were using lipid-lowering medication, the investigators wrote.
The mean LDL cholesterol level at follow-up was 160.7 mg/dL for those with familial hypercholesterolemia, a drop of 32% when compared with the mean LDL cholesterol level at baseline in the original study, according to the investigators. By contrast, LDL cholesterol in the unaffected siblings was 121.9 mg/dL, up 24% from baseline.
Just 37 patients with familial hypercholesterolemia, or about 20%, reached the LDL cholesterol treatment target of less than 100 mg/dL, the investigators wrote.
At the start of the original trial, carotid intima-media thickness was greater in patients with familial hypercholesterolemia, compared with their unaffected siblings, with a mean difference of 0.012 mm (95% confidence interval, 0.002-0.021) after adjustment for age and sex.
Some 20 years later, the mean difference in thickness for patients with familial hypercholesterolemia and unaffected siblings, was 0.555 mm and 0.551 mm, respectively, for a mean difference of just 0.008 mm (95% CI, –0.009 to 0.026) after adjustments.
Data on cardiovascular events and deaths for affected parents was collected, as each child in the study had a parent with confirmed familial hypercholesterolemia, the investigators wrote.
A total of 7% of affected parents had died of MI before the age of 40 years, whereas there were no deaths from cardiovascular causes in all 214 patients with familial hypercholesterolemia from the original study.
Similarly, about a quarter of affected parents had a cardiovascular event before age 40, whereas there was only one event recorded in the patients in the study. That event, angina pectoris resulting in percutaneous coronary intervention, occurred in a patient who stopped taking the drug at the end of the trial, the investigators wrote.
The study was supported by a grant from the AMC Foundation. The study authors reported disclosures related to numerous pharmaceutical companies and government, nonprofit, or academic entities.
SOURCE: Kastelein JJP et al. N Engl J Med. 2019;381:1547-56.
FROM THE NEW ENGLAND JOURNAL OF MEDICINE