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A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
Published on: September 15, 2018
Benefits of the MEDPED treatment support program for patients with familial hypercholesterolemia
Susan Huckendubler Stephenson1, Stacey Larrinaga-Shum, Paul N Hopkins
1Department of Internal Medicine, Division of Cardiovascular Genetics, University of Utah School of Medicine, 420 Chipeta Way, Room 1160, Salt Lake City, Utah 84108 USA.
Background:
Patients with heterozygous familial hypercholesterolemia (FH) patients often have difficultly achieving National Cholesterol Education Program (NCEP) goals. Herein we present an evaluation of a centrally located, nationwide treatment support program that remotely attempted to educate and guide FH patients by monitoring their serum low-density lipoprotein (LDL) cholesterol levels and giving appropriate treatment recommendations through mail contact. All subjects were FH patients registered with the Make Early Diagnosis to Prevent Early Deaths (MEDPED) program.
Methods:
In this descriptive evaluation, we compared self-reported lipid levels in 386 FH patients participating in our treatment support program with 295 non-participants who had responded to questionnaires. Participants were recruited into the treatment support program if they had not reached their LDL cholesterol goal after at least 1 year of follow-up and were unable to receive specialized lipid care due to location.
Results:
Participants who continued for a longer term in the treatment support program achieved greater total cholesterol reductions (14%) than the comparison group (7%, P=.004). Reductions in total and LDL cholesterol were highly correlated with more aggressive use of statin medications (P <.0001).
Conclusions:
These results demonstrate the potential benefits and limitations of a centralized program operating remotely to encourage appropriate treatment of severe hypercholesterolemia.
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