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Atherosclerosis
journal homepage: www.elsevier.com/locate/atherosclerosis
Practice of lipoprotein apheresis and short-term efficacy in children with
homozygous familial hypercholesterolemia: Data from an international
registry
Ilse K. Luirink
a,b,c,∗∗
, Barbara A. Hutten
b
, Susanne Greber-Platzer
d
, Genovefa D. Kolovou
e
,
Eldad J. Dann
f
, Sarah D. de Ferranti
g
, Christina Taylan
h
, Eric Bruckert
i
, Samir Saheb
i
, Jun Oh
j
,
Joenna Driemeyer
j
, Michel Farnier
k
, Lars Pape
l
, Claus P. Schmitt
m
, Francisco J. Novoa
n
,
Martin Maeser
o
, Luis Masana
p
, Awad Shahrani
q
, Albert Wiegman
a
, Jaap W. Groothoff
a
a
Department of Paediatrics, Amsterdam University Medical Centers, Amsterdam, the Netherlands
b
Department of Clinical Epidemiology, Biostatistics and Bioinformatics, Amsterdam University Medical Centers, Amsterdam, the Netherlands
c
Department of Vascular Medicine, Amsterdam University Medical Centers, Amsterdam, the Netherlands
d
Department of Pediatrics, University of Vienna, Vienna, Austria
e
Cardiology Department and Lipoprotein Apheresis Unit, Onassis Cardiac Surgery Center, Athens, Greece
f
Blood Bank and Apheresis, Rambam Health Care Campus, Haifa, Israel
g
Department of Cardiology, Boston Children's Hospital, Boston, USA
h
Children's and Adolescents' Hospital, University Hospital of Cologne, Cologne, Germany
i
Groupe Hospitalier Pitié-Salpêtrière, Service D'Endocrinologie, Métabolisme et Prévention des Maladies Cardiovasculaires, Paris, France
j
University Children's Hospital, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
k
Lipid Clinic, Point Médical, Rond-point de La Nation, Dijon, France
l
Pediatric Nephrology, Hannover Medical School, Hannover, Germany
m
Division of Pediatric Nephrology, Center for Pediatrics and Adolescent Medicine, University of Heidelberg, Heidelberg, Germany
n
Endocrinology Department, University Hospital Insular de Gran Canaria, University Institute of Biomedical and Health Research of the University of Las Palmas de Gran
Canaria, Las Palmas, Spain
o
Landeskrankenhaus Feldkirch, Feldkirch, Austria
p
Unitat de Medicina Vascular i Metabolisme, Hospital Universitari Sant Joan, Universitat Rovira i Virgili, Spain
q
College of Medicine, King Saud Bin Abdulaziz University for Health Sciences, Riyadh, Saudi Arabia
HIGHLIGHTS
•
Lipoprotein apheresis (LA) is safe in children with hoFH with a paucity of side effects.
•
LA leads to an important reduction of LDL-C and xanthomas in children with hoFH.
•
Only a small number of children with hoFH reach treatment goals on LA.
•
There are important differences in LA conduction strategies between different sites.
ARTICLEINFO
Keywords:
Children
Familial hypercholesterolemia
Homozygous
Lipoprotein spheresis
ABSTRACT
Background and aims: Homozygous familial hypercholesterolemia (hoFH) may cause life-threatening athero-
sclerotic cardiovascular disease in childhood. Lipoprotein apheresis (LA) is considered a pivotal treatment op-
tion, but data on its efficacy, safety and optimal performance are limited. We therefore established an inter-
national 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 col-
lected 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
https://doi.org/10.1016/j.atherosclerosis.2020.01.031
Received 17 November 2019; Received in revised form 13 January 2020; Accepted 31 January 2020
∗
Corresponding author. Amsterdam University Medical Centers, Amsterdam, Meibergdreef 9, 1105, AZ, Amsterdam, the Netherlands.
E-mail address: i.k.luirink@amsterdamumc.nl (I.K. Luirink).
Atherosclerosis 299 (2020) 24–31
Available online 18 February 2020
0021-9150/ © 2020 Published by Elsevier B.V.
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