PS3-48: Assessing Appropriate Treatment of Familial Hypercholesterolemia in Children.

Underland L, Copeland L, McNeal C, Zachariah J.

Abstract

Background/Aims In 2008, the American Academy of Pediatrics promulgated guidelines to identify and treat children with life-threatening elevations in low density lipoprotein cholesterol, aka familial hypercholesterolemia (FH). FH is fairly common with heterozygotes characterizing roughly 1/500 births. Primary treatments are lifestyle modification and antilipemics. The 2011 expansion of NHLBI guidelines to include universal pediatric screening fueled the controversy of medicating kids with predictions of "an epidemic of…lipid-lowering drug therapy," implying some are wrongly treated. While debate abounds, there are no data on the effects of the 2008 guidelines. Datasets large enough to have significant numbers of FH kids or robustly populated with relevant data are rare. The HMORN Virtual Data Warehouse (VDW) is an exception. We hypothesized that (a) the 2008 guidelines have not influenced medication use or FH identification, and (b) common healthcare disparities correlate with likelihood of therapy. Methods This exploratory study of children 2-20 years old in the Scott and White VDW will use a time-series design to study the cumulative proportion treated and yearly rate of new treatment, comparing 2005-07 vs. 2009-11. Over the same periods, we will also identify temporal trends in FH diagnosis and its sociodemographic correlates. Results We use standardized data definitions and data abstraction methods developed as part of the Cardiovascular Research Network (CVRN) VDW. Data abstracted include pharmacy claims; ICD-9 diagnosis; and predictors: age, sex, race, obesity, socioeconomic status, geographic region, urban vs. rural practice area, family history of cardiovascular disease, and provider characteristics. Primary analysis will compare the proportion treated with lipid-lowering therapy pre-2008 vs. post-2008 using multivariable adjusted regression models (logistic for proportions or Poisson for rates) to identify correlates of therapy or diagnosis and association with implementation of the 2008 guidelines as evidence of change in indications. Secondary outcomes will be treated similarly. Conclusions The CVRN VDW represents a unique resource to ascertain if pediatric lipid treatment is being rationally applied consistent with published guidelines. This pilot study may identify VDW construction and population issues that interfere with addressing this data gap.