An update on lipid apheresis for familial hypercholesterolemia

Abstract

Familial hypercholesterolemia (FH) is an inherited metabolic defect leading to increased total cholesterol and low-density cholesterol (LDL) from birth onwards. Homozygous FH, presenting with clear clinical features, has a prevalence of ~ 1 per million. Prevalence of heterozygous FH is 1/500 European population. Atherosclerotic burden depends on the degree and duration of high LDL exposure. In severe cases, early detection is critical, and aggressive lipid-lowering therapies should begin in early childhood to reduce coronary heart disease risk. Pediatric therapeutic concepts correspond to adults and are orientated at LDL plasma concentration. Mean LDL plasma target value during treatment is < 135 mg/dL. Medication in childhood consists of ezetemibe, statins, resins, and PCSK-9 inhibitors, with consideration for age restrictions. Only a minority achieve the treatment target with drug therapy alone. Therapeutic apheresis for the treatment of hypercholesterolemia selectively removes lipoproteins from blood (lipid apheresis (LA)). LA has a long tradition in adult medicine and is also safely used in children by a variety of methods, if customized to special pediatric needs. LA reduces cholesterol levels independently of residual LDL-receptor function and not only achieves reduction or disappearance of xanthomas but also inhibits progression of or mitigates aortic valve stenosis and supravalvular aortic stenosis as well as coronary artery and other atherosclerotic lesions. Cardiovascular prognosis of patients with otherwise untreatable FH depends largely on timely use of LA. Taking into account LA as a lifelong treatment, starting early in childhood, it is important to accommodate therapy modalities, such as treatment frequency and point of time, into the life of the individual.

Direct adsorption of LDL cholesterol - one center experience

“…We believe that there is insufficient awareness of the existence of familial HoFH [12][13][14][15]. Patients with FH have rapid progression of atherosclerosis with a high incidence of MACE.…”

Dyslipidemia: A Narrative Review on Pharmacotherapy

“…The most commonly used apheresis methods for removing lipoproteins from the blood are (1) double filtration; (2) LDL-C adsorption due to binding based on immunoaffinity; (3) LDL-C adsorption due to binding based on immunoaffinity; (4) heparin-induced precipitation; and (5) direct adsorption of lipoproteins (DALI) [ 182 ].…”

Facile Preparation of β-Cyclodextrin-Modified Polysulfone Membrane for Low-Density Lipoprotein Adsorption via Dopamine Self-Assembly and Schiff Base Reaction

“…In situations where conventional approaches fall short, LDL apheresis is highly recommendable owing to its remarkable efficacy in lowering LDL cholesterol and achieving therapeutic objectives. Presently, six prevalent LDL apheresis technologies are accessible, with four relying on the adsorption principle—dextran sulfate adsorption, dextran sulfate direct perfusion, polyacrylate-coated polyacrylamide direct perfusion and immune-adsorption [ 16 , 17 , 18 ]—which underscores the pivotal role of the adsorption material as a determining factor in the therapeutic efficacy of LDL-apheresis systems. Consequently, considerable focus has been directed towards exploring cost-effective and efficient adsorbents, resulting in the emergence of diverse LDL ligands, including antibodies [ 19 , 20 ], heparin [ 21 , 22 , 23 , 24 ], chondroitin sulfate [ 25 , 26 ], amino acid [ 27 ], phospholipids [ 28 , 29 ], chitosan derivatives [ 30 , 31 ] and others.…”