Clinical practice recommendations on lipoprotein apheresis for children with homozygous familial hypercholesterolaemia: An expert consensus statement from ERKNet and ESPN

被引:10
作者
Reijman, M. Doortje [1 ]
Kusters, D. Meeike [1 ]
Groothoff, Jaap W. [1 ]
Arbeiter, Klaus [2 ]
Dann, Eldad J. [3 ]
de Boer, Lotte M. [1 ]
de Ferranti, Sarah D. [4 ,5 ]
Gallo, Antonio [6 ]
Greber-Platzer, Susanne [7 ]
Hartz, Jacob [4 ,5 ]
Hudgins, Lisa C. [8 ]
Ibarretxe, Daiana [9 ,10 ,11 ,12 ]
Kayikcioglu, Meral [13 ]
Klingel, Reinhard [14 ]
Kolovou, Genovefa D. [15 ]
Oh, Jun [16 ]
Planken, R. Nils [17 ,18 ]
Stefanutti, Claudia [19 ]
Taylan, Christina [20 ]
Wiegman, Albert [1 ,22 ]
Schmitt, Claus Peter [21 ]
机构
[1] Univ Amsterdam, Dept Paediat, Amsterdam UMC, Amsterdam Cardiovasc Sci,Amsterdam Gastroenterol E, Meibergdreef 9, Amsterdam, Netherlands
[2] Med Univ Vienna, Dept Paediat & Adolescent Med, Div Paediat Nephrol & Gastroenterol, Vienna, Austria
[3] Rambam Hlth Care Campus, Blood Bank & Apheresis Unit, Haifa, Israel
[4] Boston Childrens Hosp, Dept Cardiol, Boston, MA USA
[5] Harvard Med Sch, Dept Pediat, Boston, MA USA
[6] Sorbonne Univ, Hop Pitie Salpetriere, AP HP,INSERM,UMR 1166,Dept Nutr, Lipidol & Cardiovasc Prevent Unit,Dept Nutr, F-75013 Paris, France
[7] Med Univ Vienna, Dept Paediat & Adolescent Med, Clin Div Paediat Pulmonol Allergol & Endocrinol, Vienna, Austria
[8] Rogosin Inst, Weill Cornell Med Coll, New York, NY USA
[9] Hosp Univ St Joan, Vasc Med & Metab Unit UVASMET, Tarragona, Spain
[10] Univ Rovira & Virgili, Tarragona, Spain
[11] Inst Invest Sanitaria Pere Virgili IISPV CERCA, Tarragona, Spain
[12] Inst Salud Carlos III, Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain
[13] Ege Univ, Med Fac, Dept Cardiol, TR-35100 Izmir, Turkiye
[14] Apheresis Res Inst, Stadtwaldguertel 77, D-50935 Cologne, Germany
[15] Metropolitan Hosp, Dept Prevent Cardiol, 9 Ethn Makariou & 1, Athens 18547, Greece
[16] Univ Med Ctr Hamburg Eppendorf, Martinistr 52, D-20246 Hamburg, Germany
[17] Amsterdam UMC Locat AMC, Dept Radiol & Nucl Med, Meibergdreef 9, NL-1105 AZ Amsterdam, Netherlands
[18] Amsterdam Cardiovasc Sci, Atherosclerosis & Ischem Syndromes, Amsterdam, Netherlands
[19] Sapienza Univ Rome, Umberto I Hosp, Lipid Clin & Atherosderosis Prevent Ctr, Dept Mol Med, I-00161 Rome, Italy
[20] Univ Hosp Cologne, Univ Cologne, Childrens & Adolescents Hosp, Fac Med,Paediat Nephrol, Kerpener Str 62, D-50937 Cologne, Germany
[21] Heidelberg Univ, Ctr Pediat & Adolescent Med, Pediat Nephrol, Heidelberg, Germany
[22] Dept Paediat, Amsterdam UMC, Locat AMC, Meibergdreef 9, NL-1105 AZ Amsterdam, Netherlands
关键词
Homozygous familial hypercholesterolaemia; Cholesterol; LDL; Lipoprotein apheresis; Paediatrics; Consensus statement; DOUBLE FILTRATION PLASMAPHERESIS; LDL-APHERESIS; CARDIOVASCULAR-DISEASE; LIVER-TRANSPLANTATION; DEXTRAN SULFATE; FOLLOW-UP; PEDIATRIC-PATIENTS; LIPID APHERESIS; ELEVATED LIPOPROTEIN(A); ARTERIOVENOUS-FISTULAS;
D O I
10.1016/j.atherosclerosis.2024.117525
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Homozygous familial hypercholesterolaemia is a life-threatening genetic condition, which causes extremely elevated LDL-C levels and atherosclerotic cardiovascular disease very early in life. It is vital to start effective lipid-lowering treatment from diagnosis onwards. Even with dietary and current multimodal pharmaceutical lipid-lowering therapies, LDL-C treatment goals cannot be achieved in many children. Lipoprotein apheresis is an extracorporeal lipid-lowering treatment, which is used for decades, lowering serum LDL-C levels by more than 70% directly after the treatment. Data on the use of lipoprotein apheresis in children with homozygous familial hypercholesterolaemia mainly consists of case-reports and case-series, precluding strong evidence-based guidelines. We present a consensus statement on lipoprotein apheresis in children based on the current available evidence and opinions from experts in lipoprotein apheresis from over the world. It comprises practical statements regarding the indication, methods, treatment goals and follow-up of lipoprotein apheresis in children with homozygous familial hypercholesterolaemia and on the role of lipoprotein(a) and liver transplantation.
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页数:13
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