Universität zu Köln

Klaus, Gunter, Taylan, Christina, Buescher, Rainer, Schmitt, Claus Peter, Pape, Lars, Oh, Jun, Driemeyer, Joenna, Galiano, Matthias, Koenig, Jens, Schuerfeld, Carsten, Spitthover, Ralf, Schaefer, Juergen R., Weber, Lutz T., Heibges, Andreas and Klingel, Reinhard (2018). Multimodal lipid-lowering treatment in pediatric patients with homozygous familial hypercholesterolemia-target attainment requires further increase of intensity. Pediatr. Nephrol., 33 (7). S. 1199 - 1209. NEW YORK: SPRINGER. ISSN 1432-198X

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Abstract

Background Familial hypercholesterolemia (FH) causes premature cardiovascular disease (CVD). Lipoprotein apheresis (LA) is recommended as first-line lipid-lowering treatment (LLT) for homozygous (ho) FH. Methods Efficacy of multimodal LLT including lifestyle counseling, drug treatment, and LAwas analyzed in 17 pediatric hoFH or compound heterozygous (c-het) FH patients, who commenced chronic LA in Germany before the age of 18. Results At time of diagnosis, mean low-density lipoprotein cholesterol (LDL-C) concentration was 19.6 mmol/ l (756 mg/ dl). Multimodal LLT resulted in 73% reduction of mean LDL-C concentration including a 62% contribution of LA. Only three children (18%) achieved mean LDL-C concentrations below the recommended pediatric target of 3.5 mmol/ l (135 mg/ dl). In 13 patients (76%) during chronic LA, neither cardiovascular events occurred nor was CVD progression detected clinically or by routine imaging techniques. In four patients (24%), cardiovascular events documented progression of CVD despite weekly LA, including one death due to coronary and cerebrovascular CVD which was not stabilized after commencing LA. Based on the mutational status, only 6 out of the 17 children were candidates for proprotein convertase subtilisin-kexin type 9 (PCSK9) inhibition. Two already responded with further LDL-C decrease by 40%. Conclusions Next to drug therapy, regular LA is an essential component of LLT for approaching LDL-C targets in children with hoFH or c-hetFH, which was successful only in a minority of children. Progression of CVD morbidity and resulting mortality remain unresolved issues. Early and intensified multimodal LLT guided by risk factors beyond LDL-C concentration is needed to improve outcome.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Klaus, Gunter UNSPECIFIED UNSPECIFIED UNSPECIFIED Taylan, Christina UNSPECIFIED UNSPECIFIED UNSPECIFIED Buescher, Rainer UNSPECIFIED UNSPECIFIED UNSPECIFIED Schmitt, Claus Peter UNSPECIFIED UNSPECIFIED UNSPECIFIED Pape, Lars UNSPECIFIED UNSPECIFIED UNSPECIFIED Oh, Jun UNSPECIFIED UNSPECIFIED UNSPECIFIED Driemeyer, Joenna UNSPECIFIED UNSPECIFIED UNSPECIFIED Galiano, Matthias UNSPECIFIED UNSPECIFIED UNSPECIFIED Koenig, Jens UNSPECIFIED UNSPECIFIED UNSPECIFIED Schuerfeld, Carsten UNSPECIFIED UNSPECIFIED UNSPECIFIED Spitthover, Ralf UNSPECIFIED UNSPECIFIED UNSPECIFIED Schaefer, Juergen R. UNSPECIFIED UNSPECIFIED UNSPECIFIED Weber, Lutz T. UNSPECIFIED UNSPECIFIED UNSPECIFIED Heibges, Andreas UNSPECIFIED UNSPECIFIED UNSPECIFIED Klingel, Reinhard UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-182285
DOI:	10.1007/s00467-018-3906-6
Journal or Publication Title:	Pediatr. Nephrol.
Volume:	33
Number:	7
Page Range:	S. 1199 - 1209
Date:	2018
Publisher:	SPRINGER
Place of Publication:	NEW YORK
ISSN:	1432-198X
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language DENSITY-LIPOPROTEIN APHERESIS; CARDIOVASCULAR EVENTS; FOLLOW-UP; ATHEROSCLEROSIS SOCIETY; REDUCING LIPIDS; LDL-APHERESIS; EFFICACY; SAFETY; DISEASE; COHORT Multiple languages Pediatrics; Urology & Nephrology Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/18228