Treatment Outcomes in Children and Adolescents with Hypercholesterolemia

A recent study (MM Mendelson et al. J Pediatr 2016; 178: 149-55) provides some useful data indicating that statin therapy for children and adolescents is typically effective based on cholesterol reduction levels.

This observational study prospectively collected data from 2010-2014 among 1521 pediatric patients seen for a lipid disorder.  In this cohort, 1260 patients (83%) did not receive statin therapy during the study period.  Ultimately, 97 patients (6% of clinic cohort) had received statin therapy and had adequate data for evaluation.

  • 70 patients received simvastatin: 1 at 5 mg/day, 26 at 10 mg/day, and 43 for 20 mg/day.
  • 24 patients received atorvastatin: 22 at 10 mg/day and 2 at 20 mg/day
  • 3 patients received pravastatin: 2 at 10 mg/day and 1 at 20 mg/day

Primary outcome for therapy: LDL-C <130 for patients without high risk factors and <110 for patients with high risk condition(s) (eg. diabetes mellitus, end-stage renal disease, heart transplant, Kawasaki disease with aneurysms)

Key findings:

  • Median baseline LDL-C was 215.
  • LDL-C decreased by 37% on average (83 mg/dL) within the first 60 days of therapy
  • Achieved primary outcome: 60% at 1 year,  73% at 2 years, and 87% at 3 years
  • No patients presented with relevant hepatic or myopathic side effects. 2 of 97 had transient epiosde of ALT > 3 x ULN.

Discussion:

  • Overall, the reported outcomes in this select cohort were at least as good as outcomes reported in studies of adults in the general population.  This may be due to parental supervision or perhaps due to a better physiologic response. In addition, as this was an observational study, poorly adherent patients may be lost to follow-up and would not be accounted for.
  • Currently statin therapy is recommended if lifestyle modifications are not sufficient to lower LDL-C.  Thus, “it is estimated that more than 700,000 US children and adolescents may be eligible for statin therapy according to the 2011 NHLBI guidelines.”

My take: Since cholesterol and LDL-C are biomarkers of treatment, the long-term benefit (& possible risks) of statin therapy remains unclear .  However, more data on meaningful endpoints like heart attacks and strokes could take decades.  Until then, the best evidence available suggests that the potential benefit of statin therapy could be quite substantial.

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