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Statin therapy in children with FH reduces CV risk
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Patients with familial hypercholesterolemia who initiated statin therapy during childhood had a slower progression of carotid intima-media thickness and a reduced risk for CVD in adulthood, according to a study with 20-year follow-up data published in The New England Journal of Medicine.
Statin therapy in children
Ilse K. Luirink, MD, PhD candidate at Amsterdam University Medical Centers, and colleagues analyzed data from 214 patients with familial hypercholesterolemia (FH) who were assigned statin therapy at age 8 to 18 years in a single-center, double-blind, placebo-controlled trial that originally evaluated the efficacy and safety of pravastatin at 2 years. Most patients with FH (98%) were genetically confirmed as having the condition. Follow-up in the original study was conducted at a pediatric lipid clinic until patients transitioned to a general practitioner or adult lipid clinic.
The control group consisted of 95 siblings who were unaffected by FH.
In the current study, researchers contacted all patients and their siblings 20 years after the original trial was initiated. Patients who agreed to participate in this follow-up attended a single hospital visit for a physical examination, measurement of carotid intima-media thickness and a fasting blood sample. Questionnaires were sent before this visit to collect information on lifestyle habits, medical history, family history and medication use. A phone interview was conducted for patients who could not visit the hospital or did not complete the questionnaire.
Prespecified outcomes in this study included CVD and CV death. CVD was defined as the presence of angina pectoris, MI, stroke or peripheral artery disease, or a coronary revascularization procedure.
Of the original cohort, 86% of those with FH and 81% of siblings were seen in follow-up. Data on CV events were available for 95% of patients with FH, whereas data on CV death was obtained for 100% of these patients.
Patients with FH had a decrease in mean LDL from 237.3 mg/dL to 160.7 mg/dL, which represented a decrease of 32% from baseline. Twenty percent of patients with FH achieved LDL treatment goals of less than 100 mg/dL.
During follow-up, patients with FH had a mean progression of carotid intima-media thickness of 0.0056 mm per year compared with 0.0057 mm per year in siblings (mean difference adjusted for sex, –0.0001 mm per year; 95% CI, –0.001 to 0.0008).
CV events, death at 20 years
The cumulative incidence of CV events at age 39 years was lower in patients with FH vs. siblings (1% vs. 26%). The cumulative incidence of CV death at 39 years of age was also lower in patients with FH (0% vs. 7%).
“We deduced that starting statin treatment in childhood had beneficial effects on atherosclerotic vascular disease in participants in the present study, even when guideline goals were not always achieved,” Luirink and colleagues wrote. “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.” – by Darlene Dobkowski
Disclosures: Luirink reports no relevant financial disclosures. Please see the study for all other authors’ relevant financial disclosures.
Perspective
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Amy L. Peterson, MD
These findings provide the first look at long-term outcomes in individuals with FH who are now adults but have been treated since childhood. These data demonstrate that long-term treatment of FH from childhood can effectively improve carotid intima-media thickness and, even more importantly, lower the risk for CV events. Also importantly, in this cohort there were no long-term adverse events of statin therapy. Previously, outcome data from statin therapy started in childhood had been limited to short- and intermediate-term follow-up intervals, too short to address both long-term safety questions or to report CV outcomes.
To clinicians, these findings should reinforce that a FH diagnosis should ideally occur in childhood in order to allow the opportunity for treatment of the child and cascade screening of potentially affected family members.
Additional long-term studies are needed to continue to evaluate statin efficacy and safety over long periods of time, and, for children, to identify which populations of children would derive most benefit from early statin treatment.
Despite the growing body of evidence supporting treatment of FH with statins from childhood, many health care providers (primary care providers, but also cardiologists and even lipid experts) are reluctant to recommend testing children’s cholesterol levels out of concern both for long-term safety with statins but also lack of familiarity with current recommendations. This study adds to that body of evidence and hopefully will help persuade some providers to change their practice.
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