Universität zu Köln

Luirink, Ilse K., Hutten, Barbara A., Greber-Platzer, Susanne, Kolovou, Genovefa D., Dann, Eldad J., de Ferranti, Sarah D., Taylan, Christina, Bruckert, Eric, Saheb, Samir, Oh, Jun, Driemeyer, Joenna, Farnier, Michel, Pape, Lars, Schmitt, Claus P., Novoa, Francisco J., Maeser, Martin, Masana, Luis ORCID: 0000-0002-0789-4954, Shahrani, Awad, Wiegman, Albert and Groothoff, Jaap W. (2020). Practice of lipoprotein apheresis and short-term efficacy in children with homozygous familial hypercholesterolemia: Data from an international registry. Atherosclerosis, 299. S. 24 - 32. CLARE: ELSEVIER IRELAND LTD. ISSN 1879-1484

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Abstract

Background and aims: Homozygous familial hypercholesterolemia (hoFH) may cause life-threatening atherosclerotic cardiovascular disease in childhood. Lipoprotein apheresis (LA) is considered a pivotal treatment option, but data on its efficacy, safety and optimal performance are limited. We therefore established an international registry on the execution and outcomes of LA in HoFH children. Here we report LA policies and short-term outcomes. Methods: We approached centers worldwide, involved in LA in children with hoFH for participation. We collected information on clinical and treatment characteristics on patients aged 0-19 years between November 2016 and November 2018. Results: We included 50 children, treated at 15 sites. Median (IQR) LDL-C levels at diagnosis, on medication and on LA were 19.2 (16.2-22.1), 14.4 (10.8-16.7) mmol/L and 4.6 mmol/L, respectively. Median (IQR) time between diagnosis and start of LA was 2.8 (1.0-4.7) years. Six (12%) patients developed cardiovascular disease during that period. Most children received LA either weekly (43%) or biweekly (37%). Seven (17%) patients reached mean LDL-C levels < 3.5 mmol/L, all of them treated at least weekly. Xanthomas were present in 42 (84%) patients at diagnosis and disappeared completely in 19 (45%) on LA. Side effects of LA were minor. There were significant differences in LA conduction between sites in terms of frequency, responsible medical specialities and vascular access. Conclusions: LA is a safe treatment and may effectively lower LDL-C in children with HoFH. However, there is room for improvement with respect to time of onset and optimization of LA therapy in terms of frequency and execution.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Luirink, Ilse K. UNSPECIFIED UNSPECIFIED UNSPECIFIED Hutten, Barbara A. UNSPECIFIED UNSPECIFIED UNSPECIFIED Greber-Platzer, Susanne UNSPECIFIED UNSPECIFIED UNSPECIFIED Kolovou, Genovefa D. UNSPECIFIED UNSPECIFIED UNSPECIFIED Dann, Eldad J. UNSPECIFIED UNSPECIFIED UNSPECIFIED de Ferranti, Sarah D. UNSPECIFIED UNSPECIFIED UNSPECIFIED Taylan, Christina UNSPECIFIED UNSPECIFIED UNSPECIFIED Bruckert, Eric UNSPECIFIED UNSPECIFIED UNSPECIFIED Saheb, Samir UNSPECIFIED UNSPECIFIED UNSPECIFIED Oh, Jun UNSPECIFIED UNSPECIFIED UNSPECIFIED Driemeyer, Joenna UNSPECIFIED UNSPECIFIED UNSPECIFIED Farnier, Michel UNSPECIFIED UNSPECIFIED UNSPECIFIED Pape, Lars UNSPECIFIED UNSPECIFIED UNSPECIFIED Schmitt, Claus P. UNSPECIFIED UNSPECIFIED UNSPECIFIED Novoa, Francisco J. UNSPECIFIED UNSPECIFIED UNSPECIFIED Maeser, Martin UNSPECIFIED UNSPECIFIED UNSPECIFIED Masana, Luis UNSPECIFIED orcid.org/0000-0002-0789-4954 UNSPECIFIED Shahrani, Awad UNSPECIFIED UNSPECIFIED UNSPECIFIED Wiegman, Albert UNSPECIFIED UNSPECIFIED UNSPECIFIED Groothoff, Jaap W. UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-338831
DOI:	10.1016/j.atherosclerosis.2020.01.031
Journal or Publication Title:	Atherosclerosis
Volume:	299
Page Range:	S. 24 - 32
Date:	2020
Publisher:	ELSEVIER IRELAND LTD
Place of Publication:	CLARE
ISSN:	1879-1484
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language LDL-APHERESIS; ATHEROSCLEROSIS; IMPACT Multiple languages Cardiac & Cardiovascular Systems; Peripheral Vascular Disease Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/33883