TY - JOUR
T1 - Practice of lipoprotein apheresis and short-term efficacy in children with homozygous familial hypercholesterolemia: Data from an international registry
AU - Luirink, Ilse K.
AU - Hutten, Barbara A.
AU - Greber-Platzer, Susanne
AU - Kolovou, Genovefa D.
AU - Dann, Eldad J.
AU - de Ferranti, Sarah D.
AU - Taylan, Christina
AU - Bruckert, Eric
AU - Saheb, Samir
AU - Oh, Jun
AU - Driemeyer, Joenna
AU - Farnier, Michel
AU - Pape, Lars
AU - Schmitt, Claus P.
AU - Novoa, Francisco J.
AU - Maeser, Martin
AU - Masana, Luis
AU - Shahrani, Awad
AU - Wiegman, Albert
AU - Groothoff, Jaap W.
PY - 2020/4
Y1 - 2020/4
N2 - 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.
AB - 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.
KW - Children
KW - Familial hypercholesterolemia
KW - Homozygous
KW - Lipoprotein spheresis
UR - http://www.scopus.com/inward/record.url?scp=85081927308&partnerID=8YFLogxK
U2 - https://doi.org/10.1016/j.atherosclerosis.2020.01.031
DO - https://doi.org/10.1016/j.atherosclerosis.2020.01.031
M3 - Article
C2 - 32199148
SN - 0021-9150
VL - 299
SP - 24
EP - 31
JO - Atherosclerosis
JF - Atherosclerosis
ER -