alexa Long-term safety and efficacy of low-density lipoprotein apheresis in childhood for homozygous familial hypercholesterolemia.
Bioinformatics & Systems Biology

Bioinformatics & Systems Biology

Journal of Glycomics & Lipidomics

Author(s): Hudgins LC, Kleinman B, Scheuer A, White S, Gordon BR

Abstract Share this page

Abstract Untreated pediatric patients with homozygous familial hypercholesterolemia usually have myocardial infarctions, heart failure, or death by the teenage years. Low-density lipoprotein (LDL) apheresis effectively lowers LDL cholesterol in the short term, but there is little published information on the long-term safety and efficacy of this treatment in children. An analysis was performed of a registry of all 29 patients who began LDL apheresis before 18 years of age at 15 sites during the 11 years since approval by the United States Food and Drug Administration. A chart review of 9 patients treated at The Rogosin Institute was also performed to obtain additional details about lipid lowering, adverse events, and cardiovascular status. Of the 29 patients, 20 are currently treated, with a mean age at the start of treatment of 9 +/- 4 years (range 3 to 15) and a mean treatment duration of 6 +/- 4 years (range 2 to 21). The baseline LDL cholesterol (521 +/- 126 mg/dl) is acutely lowered by 75\% and chronically lowered by 48\% with biweekly sessions. Systemic adverse events have been uncommon. Atherosclerotic disease of the coronary arteries and/or aorta or aortic valve was evident by angiography and/or echocardiography in 12 patients (60\%) at baseline and progressed to more severe, symptomatic disease in 6 (30\%). In conclusion, LDL apheresis is well tolerated for decades by even very young pediatric patients with homozygous familial hypercholesterolemia. It effectively lowers LDL cholesterol, but target LDL levels are not achieved, and some patients will show progression of cardiovascular disease. This article was published in Am J Cardiol and referenced in Journal of Glycomics & Lipidomics

Relevant Expert PPTs

Relevant Speaker PPTs

Recommended Conferences

Relevant Topics

Peer Reviewed Journals
Make the best use of Scientific Research and information from our 700 + peer reviewed, Open Access Journals
International Conferences 2017-18
Meet Inspiring Speakers and Experts at our 3000+ Global Annual Meetings

Contact Us

© 2008-2017 OMICS International - Open Access Publisher. Best viewed in Mozilla Firefox | Google Chrome | Above IE 7.0 version