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

被引：9

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

来源：

ATHEROSCLEROSIS | 2024年 / 392卷

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.

引用

页数：13

共 27 条

[1] Clinical practice recommendations for primary hyperoxaluria: an expert consensus statement from ERKNet and OxalEurope
Groothoff, Jaap W.
Metry, Ella
Deesker, Lisa
Garrelfs, Sander
Acquaviva, Cecile
Almardini, Reham
Beck, Bodo B.
Boyer, Olivia
Cerkauskiene, Rimante
Ferraro, Pietro Manuel
Groen, Luitzen A.
Gupta, Asheeta
Knebelmann, Bertrand
Mandrile, Giorgia
Moochhala, Shabbir S.
Prytula, Agnieszka
Putnik, Jovana
Rumsby, Gill
Soliman, Neveen A.
Somani, Bhaskar
Bacchetta, Justine
NATURE REVIEWS NEPHROLOGY, 2023, 19 (03) : 194 - 211
[2] Clinical practice recommendations for primary hyperoxaluria: an expert consensus statement from ERKNet and OxalEurope
Jaap W. Groothoff
Ella Metry
Lisa Deesker
Sander Garrelfs
Cecile Acquaviva
Reham Almardini
Bodo B. Beck
Olivia Boyer
Rimante Cerkauskiene
Pietro Manuel Ferraro
Luitzen A. Groen
Asheeta Gupta
Bertrand Knebelmann
Giorgia Mandrile
Shabbir S. Moochhala
Agnieszka Prytula
Jovana Putnik
Gill Rumsby
Neveen A. Soliman
Bhaskar Somani
Justine Bacchetta
Nature Reviews Nephrology, 2023, 19 : 194 - 211
[3] Clinical Practice Recommendations on Kidney Management in Methylmalonic Acidemia: an Expert Consensus Statement From ERKNet and MetabERN
Servais, Aude
Zacchia, Miriam
Dehoux, Laurene
Shroff, Rukshana
Brassier, Anais
Taurisano, Roberta
Koelker, Stefan
Oh, Jun
Ariceta, Gema
Stojanovic, Jelena
Hoerster, Friederike
Strologo, Dello
Spada, Marco
Schiff, Manuel
Dionisi-Vici, Carlo
KIDNEY INTERNATIONAL REPORTS, 2024, 9 (12): : 3362 - 3374
[4] Recommendations for the management of patients with homozygous familial hypercholesterolaemia: Overview of a new European Atherosclerosis Society consensus statement
Bruckert, Eric
ATHEROSCLEROSIS SUPPLEMENTS, 2014, 15 (02) : 26 - 32
[5] Clinical course of a 15-year-LDL Apheresis Therapy in Children with homozygous familial Hypercholesterolaemia (FH)
Kurt, B.
Soufi, M.
Klaus, G.
Schaefer, J.
INTERNIST, 2016, 57 : S50 - S50
[6] 2023 Update on European Atherosclerosis Society Consensus Statement on Homozygous Familial Hypercholesterolaemia: new treatments and clinical guidance
Cuchel, Marina
Raal, Frederick J.
Hegele, Robert A.
Al-Rasadi, Khalid
Arca, Marcello
Averna, Maurizio
Bruckert, Eric
Freiberger, Tomas
Gaudet, Daniel
Harada-Shiba, Mariko
Hudgins, Lisa C.
Kayikcioglu, Meral
Masana, Luis
Parhofer, Klaus G.
Roeters van Lennep, Jeanine E.
Santos, Raul D.
Stroes, Erik S. G.
Watts, Gerald F.
Wiegman, Albert
Stock, Jane K.
Tokgoezoglu, Lale S.
Catapano, Alberico L.
Ray, Kausik K.
EUROPEAN HEART JOURNAL, 2023, 44 (25) : 2277 - 2291
[7] Practice of lipoprotein apheresis and short-term efficacy in children with homozygous familial hypercholesterolemia: Data from an international registry
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
Shahrani, Awad
Wiegman, Albert
Groothoff, Jaap W.
ATHEROSCLEROSIS, 2020, 299 : 24 - 31
[8] Clinical practice recommendations for management of Diabetes Mellitus in Arab region: An expert consensus statement from Arab Diabetes Forum (ADF)
Elsherif, Ibrahim
Jammah, Anwar Ali
Ibrahim, Athar Reda
Alawadi, Fatheya
Sadek, Ireny Samy
Rahman, Abbas Mahdi
el Sharify, Ghalab
AlFeky, Altayeb
Aldossari, Khaled
Roushdy, Eman
ElBarbary, Nancy Samir
BenRajab, Faten
Elghweiry, Awad
Farah, Sirelkhatim Ismaeil Sirri
Hajjaji, Issam
AlShammary, Afaf
Abdulkareem, Faris
AbdelRahim, Aly
Orabi, Abbass
PRIMARY CARE DIABETES, 2024, 18 (05) : 471 - 478
[9] Defining severe familial hypercholesterolaemia and the implications for clinical management: a consensus statement from the International Atherosclerosis Society Severe Familial Hypercholesterolemia Panel
Santos, Raul D.
Gidding, Samuel S.
Hegele, Robert A.
Cuchel, Marina A.
Barter, Philip J.
Watts, Gerald F.
Baum, Seth J.
Catapano, Alberico L.
Chapman, M. John
Defesche, Joep C.
Folco, Emanuela
Freiberger, Tomas
Genest, Jacques
Hovingh, G. Kees
Harada-Shiba, Mariko
Humphries, Steve E.
Jackson, Ann S.
Mata, Pedro
Moriarty, Patrick M.
Raal, Frederick J.
Al-Rasadi, Khalid
Ray, Kausik K.
Reiner, Zelijko
Sijbrands, Eric J. G.
Yamashita, Shizuya
LANCET DIABETES & ENDOCRINOLOGY, 2016, 4 (10): : 850 - 861
[10] Lipoprotein Apheresis: Utility, Outcomes, and Implementation in Clinical Practice: A Scientific Statement From the American Heart Association
Gianos, Eugenia
Duell, P. Barton
Toth, Peter P.
Moriarty, Patrick M.
Thompson, Gilbert R.
Brinton, Eliot A.
Hudgins, Lisa C.
Nametka, Mary
Byrne, Kathleen H.
Raghuveer, Geetha
Nedungadi, Prashant
Sperling, Laurence S.
ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY, 2024, 44 (12) : e304 - e321

← 1 2 3 →