Underrepresentation of non-white children in trials of statins in children with heterozygous familial hypercholesterolemia

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7 Scopus citations


Background: The elimination of disparities in cardiovascular health is a major focus of the Healthy People 2010 national public health agenda. However, identifying and addressing such disparities within the realm of pediatrics in general, and preventive cardiology in particular, has not received recent attention. In published pediatric clinical trials of statins in heterozygous familial hypercholesterolemia that report race, minority children are underrepresented. Objectives: The purpose of this analysis was 3-fold: 1) to obtain and report on the racial composition of statin trials in children with heterozygous familial hypercholesterolemia; 2) to explore the hypothesis that founder effects among populations of White children may have facilitated or favored their inclusion in statin trials; and 3) to determine whether the selective lipid screening guidelines based on family history may inadvertently identify fewer minority children who would otherwise qualify for investigative trials. Design: We conducted a Medline search to identify all pediatric familial hypercholesterolemia statin trials. We contacted the corresponding authors to obtain race/ethnicity data and to obtain information about the presence of founder effects in the populations studied. We conducted a second literature search for evidence that selective, family medical history-based screening of children for hypercholesterolemia, as proposed by the National Cholesterol Education Program, might fail to identify minority children who would otherwise qualify for inclusion in these studies. Results: Ninety-two percent of the 885 children enrolled in statin trials were White. A predominance of White children was found even in studies from countries with a sizable population of non-White children and where founder effects have not been described. Strong but indirect evidence from both the adult literature and the pediatric literature suggests that the family history-based selective screening engenders healthcare disparities for minority and disadvantaged children. Conclusions: Non-White children are underrepresented in international clinical trials of statins. Both ethical and pharmacogenomic arguments exist to justify efforts to correct this. Our findings suggest that intensive efforts will be required to arrive at a fair representation of minority children in studies of pediatric heterozygous familial hypercholesterolemia.

Original languageEnglish (US)
Pages (from-to)166-171
Number of pages6
JournalEthnicity and Disease
Issue number2
StatePublished - Mar 2009


  • Cholesterol
  • Familial hypercholesterolemia
  • Guidelines
  • HMG-CoA reductase inhibitors
  • Health services accessibility
  • Minority groups
  • Statins

ASJC Scopus subject areas

  • Epidemiology


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