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Long-term statin therapy benefited children with familial hypercholesterolemia
Patients with familial hypercholesterolemia who had long-term statin therapy initiated in childhood had normalized progression of carotid intima-media thickness, according to a research letter published in JAMA.
Guidelines for familial hypercholesterolemia (FH) treatment recommend beginning statin therapy as early as age 8 years, but long-term safety and efficacy data for statin therapy initiated in childhood were unavailable until now, D. Meeike Kusters, MD, and colleagues wrote.
Kusters, of the department of vascular medicine at Academic Medical Center, Amsterdam, and colleagues performed a cohort study of 214 children with heterozygous FH aged 8 to 18 years.
The children with FH were enrolled between 1997 and 1999 in a 2-year, double blind, placebo-controlled trial of pravastatin (Pravachol, Bristol-Myers Squibb). At the end of the study in 2001, the children with FH were given pravastatin 20 mg/day to 40 mg/day and followed until March 2011, along with 95 of their unaffected siblings. Some of the children with FH switched to other statins during the study.
Ten-year follow-up was achieved in 194 children with FH (163 of whom were still using lipid-lowering medications) and 83 siblings, all of whom were aged 18 to 30 years in 2011.
At 10 years, mean carotid intima-media thickness was greater in those with FH compared with unaffected siblings (FH group, 0.48 mm; 95% CI, 0.472-0.489; sibling group, 0.469 mm; 95% CI, 0.459-0.48), Kusters and colleagues wrote.
However, there was no difference between the groups in progression of carotid intima-media thickness from baseline to 10 years (FH group, 0.039 mm; 95% CI, 0.032-0.046; sibling group, 0.037 mm; 95% CI, 0.032-0.042), according to the researchers.
Among those with FH, age at initiation of statin therapy was associated with carotid intima-media thickness at follow-up (regression coefficient, 0.003 mm; standard error, 0.001 mm; P=.009), Kusters and colleagues wrote.
There were no differences between the groups in laboratory safety parameters, growth, maturation and educational level, the researchers found. No serious adverse events were reported during follow-up, although the study “lacks statistical power to detect rare events,” they wrote.
Given that at 10 years, the mean total cholesterol and LDL levels in the FH group remained higher than recommended treatment standards and their mean carotid intima-media thickness remained higher than that of unaffected siblings, “more robust lipid-lowering therapy or earlier initiation of statins may be required to completely restore arterial wall morphology and avert [CV] events later in life in this high-risk population,” Kusters and colleagues wrote.
Disclosure: The study was funded by the Dutch Heart Foundation. One researcher reports receiving lecture grants from Aegerion, Amgen, AstraZeneca, Eli Lilly, Genzyme, Isis, Merck Sharp & Dohme, Novartis, Pfizer, Regeneron, Roche and Sanofi.