Role of lipid apheresis in changing times

Abstract

During the last decades, LDL-apheresis was established as an extracorporeal treatment option for patients with severe heterozygous or homozygous familial hypercholesterolemia (FH) that is resistant to conventional treatment strategies such as diet, drugs, and changes in lifestyle. Nearly half a century ago, the first LDL-apheresis treatment was performed by plasma exchange in a child with homozygous FH.At the beginning of the 1970s, the clinical advantage of regular extracorporeal LDL-elimination was demonstrated in siblings suffering from homozygous FH. These findings encouraged researchers especially from Germany and Japan to develop extracorporeal devices to selectively eliminate LDL-cholesterol in the 1980s.Although the selectivity of the currently available LDL-apheresis devices is different, the efficacy of LDL-elimination during a single treatment is rather similar and ranges between 55 and 65 % of the pretreatment LDL plasma concentration.In the 1990s, the patients regularly treated by extracorporeal LDL-elimination, diet, and drugs were included in regression studies assessed by angiography. It was shown that the combined treatment with LDL-apheresis, diet, and drugs resulted in less progression of coronary lesions than drugs and/or diet alone. However, although a tendency was evident, results did not reach criteria for significance.During the last decade, apheresis registries were established to collect data on efficiency, safety, and clinical outcome of regular long-term LDL-apheresis. The evaluation of registry data will certainly permit further insights in the therapeutic benefit of this expensive and time-consuming therapeutic approach.Furthermore, the future of LDL-apheresis will depend upon the availability of highly efficient new drugs and molecular genetic approaches such as RNA silencing of the apoB gene, whereas the liver transplantation and gene therapy of the LDL-receptor deficiency will not replace LDL-apheresis in severe familial hypercholesterolemia in the near future.

Management of patients with familial hypercholesterolaemia

“…65,66 Although apheresis might increase longevity in patients with HoFH and decrease CVD morbidity in patients with HeFH who are refractory or intolerant to statins, 67 no hard efficacy data from double-blind, randomized trials exists and, owing to several reasons (primarily ethical), such studies will probably not be performed. 68 In very young patients with HoFH, venous access can be difficult to find, which can lead to treatment postponement for 1-2 years: the youngest patient with HoFH treated with statin therapy only underwent apheresis at the age of 3 years; 69 as of 2015, 12 years later, the patient remains alive and with no cardiovascular events. Although apheresis is well tolerated for decades by even very young patients with HoFH and lowers LDL-C effectively, the target LDL-C level is often not achieved with this technique.…”

Lipoprotein apheresis: an update

“…Clearly, liver transplantation and gene ther apy of the LDLreceptor deficiency will not replace lipoprotein apheresis in severe familial hypercholesterolemia in the near future [86].…”

Hipercolesterolemia familiar homocigota: adaptación a España del documento de posición del grupo de consenso sobre hipercolesterolemia familiar de la Sociedad Europea de Arteriosclerosis. Documento de Consenso de la Sociedad Española de Arteriosclerosis (SEA) y la Fundación Hipercolesterolemia Familiar (FHF)

“…Sin embargo, las curvas de rebote tras el tratamiento son más o menos paralelas. Véase Schuff-Werner et al 51 .…”