Universität zu Köln

Taylan, Christina, Schlune, Andrea, Meissner, Thomas, Azukaitis, Karolis, ten Cate, Floris E. A. Udink and Weber, Lutz T. (2016). Disease control via intensified lipoprotein apheresis in three siblings with familial hypercholesterolemia. J. Clin. Lipidol., 10 (6). S. 1303 - 1311. NEW YORK: ELSEVIER SCIENCE INC. ISSN 1876-4789

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Abstract

BACKGROUND: Familial hypercholesterolemia (FH), the prevalent monogenic form of hypercholesterolemia, carries the risk of premature coronary heart disease. Lipoprotein-apheresis is established in children with severe dyslipidemia. We present 3 siblings with a negative/negative residual low density lipoprotein (LDL) receptor mutation (p.Trp577Arg), unresponsive to drug treatment. OBJECTIVE: Intensified lipoprotein-apheresis is well tolerated and results in permanently low lipid values without harming the health-related quality of life in children. METHODS: Three homozygous FH siblings, aged 7-13 years, had been treated with statins and ezetimibe for 12 months but still showed highly elevated low-density lipoprotein cholesterol (LDL-C) plasma concentrations. They were started on double-filtration plasmapheresis that was subsequently intensified according to plasma lipid levels. RESULTS: Each lipoprotein apheresis session reduced LDL-C concentration by 66% to 70%. Treated plasma volume was doubled after 6 months due to a sustained rebound of LDL-C between sessions. However, the rebound remained unchanged. Only an increase in frequency of sessions to every 3 to 4 days resulted in acceptable pre-treatment LDL-C concentrations (Cmax). Neither cessation of statins nor reduction of plasma exchange volume to 1.5 fold in follow-up influenced Cmax. Intensified therapy did not harm health-related quality of life as assessed by PedsQL and was well tolerated. CONCLUSIONS: In pediatric FH patients unresponsive to drug treatment, intensified lipoprotein apheresis can normalize plasma lipid levels. Apparently, treatment frequency rather than volume has greater influence on its efficacy. The potential burden of intensified therapy to daily life has to be regarded. Serum lipid levels in FH should be normalized to minimize cardiovascular risk. (C) 2016 National Lipid Association. All rights reserved.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Taylan, Christina UNSPECIFIED UNSPECIFIED UNSPECIFIED Schlune, Andrea UNSPECIFIED UNSPECIFIED UNSPECIFIED Meissner, Thomas UNSPECIFIED UNSPECIFIED UNSPECIFIED Azukaitis, Karolis UNSPECIFIED UNSPECIFIED UNSPECIFIED ten Cate, Floris E. A. Udink UNSPECIFIED UNSPECIFIED UNSPECIFIED Weber, Lutz T. UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-253951
DOI:	10.1016/j.jacl.2016.08.006
Journal or Publication Title:	J. Clin. Lipidol.
Volume:	10
Number:	6
Page Range:	S. 1303 - 1311
Date:	2016
Publisher:	ELSEVIER SCIENCE INC
Place of Publication:	NEW YORK
ISSN:	1876-4789
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language QUALITY-OF-LIFE; INTIMA-MEDIA THICKNESS; STAGE RENAL-DISEASE; LDL-APHERESIS; CARDIOVASCULAR-DISEASE; REFERENCE VALUES; FOLLOW-UP; CHILDREN; DIAGNOSIS; ADULTS Multiple languages Pharmacology & Pharmacy Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/25395