Top Things to Know: Lipoprotein Apheresis – Utility, Outcomes, and Implementation in Clinical Practice
Prepared by Bernardo Acevedo-Mendez, MD, FACP; Assistant Professor of Medicine, Zucker School of Medicine at Hofstra/Northwell
- Untreated patients living with homozygous familial hypercholesterolemia (FH) commonly have lipoprotein cholesterol (LDL-C) levels of 450-1100 mg/dL, resulting in a mean age of first atherosclerotic cardiovascular disease (ASCVD) event at 12 years and death at age 18 years. Patients with untreated heterozygous familial hypercholesterolemia (FH) may have a first ASCVD event before the age of 30 years, with 50% risk of heart attack in men by age 50 years and in women by age 65 years.
- FH remains underdiagnosed and undertreated. Among those who qualify for lipoprotein apheresis (LA), only ~2-3% are receiving treatment.
- LA is a Food and Drug Administration (FDA) approved adjunctive therapeutic option for treating refractory high levels of low-density lipoprotein cholesterol and lipoprotein (a) [Lp(a)] in children and adults with clinical heterozygous and homozygous (FH) who meet the criteria.
- LA is currently the only FDA approved treatment for Lp(a) lowering, but only in patients with clinical FH and LDL-C > 100 mg/dL and ASCVD. In other parts of the world, it is approved for LDL or Lp(a) lowering and used more commonly in countries such as Germany and the United Kingdom.
- The most common LA devices remove apolipoprotein B (ApoB) containing lipoproteins from the bloodstream using a system that targets the positive isoelectric charge of ApoB.
- In addition to lowering atherogenic lipoproteins, LA may reduce serum levels of pro-inflammatory and pro-thrombotic factors, reduces blood viscosity, and acutely increases microvascular myocardial perfusion, which may have beneficial effects on endothelial function.
- Treatment with LA may acutely lower LDL-C concentration by 75-85%.
- Other possible clinical benefits provided by LA include decreased angina, increased walking distance, plaque stabilization and regression, and regression of xanthomas.
- LA is also potentially beneficial for reversing kidney disease in a portion of patients with refractory focal segmental glomerulosclerosis and may be beneficial in the treatment of pre-eclampsia.
- Prospective and retrospective observational data suggest that LA may lower major adverse cardiovascular events by 75-95%. Randomized controlled trials or larger prospective studies are warranted to further substantiate cardiovascular benefits.
Citation
Gianos E, Duell PB, Toth PP, Moriarty PM, Thompson GR, Brinton EA, Hudgins LC, Nametka M, Byrne KH, Raghuveer G, Nedungadi P, Sperling LS; on behalf of the American Heart Association Council on Arteriosclerosis, Thrombosis and Vascular Biology; Council on Cardiovascular and Stroke Nursing; Council on Clinical Cardiology; Council on Lifelong Congenital Heart Disease and Heart Health in the Young; and Council on Peripheral Vascular Disease. Lipoprotein apheresis: utility, outcomes, and implementation in clinical practice: a scientific statement from the American Heart Association. Arterioscler Thromb Vasc Biol. Published online October 7, 2024. doi: 10.1161/ATV.0000000000000177