title: Systematic Review of Low-Density Lipoprotein Cholesterol Apheresis for the Treatment of Familial Hypercholesterolemia.
abstract: Apheresis is an important treatment for reducing low-density lipoprotein cholesterol (LDL-C) in patients with familial hypercholesterolemia (FH). We systematically reviewed the current literature surrounding LDL-C apheresis for FH.
  
  
  
  Electronic databases were searched for publications of LDL-C apheresis in patients with FH. Inclusion criteria include articles in English published in 2000-2013 that provide descriptions of practice patterns, efficacy/effectiveness, and costs related to LDL-C apheresis in patients with FH. Data were stratified by country and FH genotype where possible. Thirty-eight studies met the inclusion criteria: 8 open-label clinical trials, 11 observational studies, 17 reviews/guidelines, and 2 health technology assessments. The prevalence of FH was not well characterized by country, and underdiagnosis was a barrier to FH treatment. Treatment guidelines varied by country, with some guidelines recommending LDL-C apheresis as first-line treatment in patients with homozygous FH and after drug therapy failure in patients with heterozygous FH. Additionally, guidelines typically recommended weekly or biweekly LDL-C apheresis treatments conducted at apheresis centers that may last 2 to >3 hours per session. Studies reported a range for mean LDL-C reduction after apheresis: 57-75% for patients with homozygous FH and 58-63% for patients with heterozygous FH. Calculated annual costs (in US$2015) may reach US$66 374 to US$228 956 per patient for weekly treatment.
  
  
  
  LDL-C apheresis treatment may be necessary for patients with FH when drug therapy is inadequate in reducing LDL-C to target levels. While apheresis reduces LDL-C, high per-session costs and the frequency of guideline-recommended treatment result in substantial annual costs, which are barriers to the optimal treatment of FH.
authors.affiliation: ICON Plc, Los Angeles, CA.
authors.forename: Anthony
authors.initials: A
authors.lastname: Wang
authors.affiliation: Seattle Genetics, Bothell, WA.
authors.forename: Akshara
authors.initials: A
authors.lastname: Richhariya
authors.affiliation: Amgen Inc., Thousand Oaks, CA sgandra@amgen.com.
authors.forename: Shravanthi R
authors.initials: SR
authors.lastname: Gandra
authors.affiliation: ICON Plc, Los Angeles, CA.
authors.forename: Brian
authors.initials: B
authors.lastname: Calimlim
authors.affiliation: Health Net, Woodland Hills, CA.
authors.forename: Lisa
authors.initials: L
authors.lastname: Kim
authors.affiliation: Amgen Inc., Thousand Oaks, CA.
authors.forename: Ruben G W
authors.initials: RG
authors.lastname: Quek
authors.affiliation: Precision for Medicine, Los Angeles, CA.
authors.forename: Robert J
authors.initials: RJ
authors.lastname: Nordyke
authors.affiliation: University of Illinois College of Medicine, Peoria, IL Ciccarone Center for the Prevention of Cardiovascular Disease, Johns Hopkins University School of Medicine, Baltimore, MD.
authors.forename: Peter P
authors.initials: PP
authors.lastname: Toth
mesh_terms: D001781:Blood Component Removal / Q000191:economics / Q000379:methods; D008078:Cholesterol, LDL / Q000097:blood*; D003362:Cost-Benefit Analysis; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000628:therapy*
chemical_list: D008078:Cholesterol, LDL
keywords: apheresis; familial hypercholesterolemia; low‐density lipoprotein cholesterol