title: Long-term follow-up of young adults with familial hypercholesterolemia after participation in clinical trials during childhood.
abstract: BACKGROUND
  There are little long-term data on patients with familial hypercholesterolemia (FH) who initiated lipid-lowering therapy during childhood.
  
  
  OBJECTIVE
  To study long-term outcomes in young adults with FH who participated in clinical trials on lipid-lowering therapy during childhood.
  
  
  METHODS
  Participants in at least 1 of 6 clinical trials that took place between 1999 and 2008 were interviewed in 2011 or 2013. Frequency of medical consultations, use of lipid-lowering therapy, lipid levels, side effects, diet, tobacco use, and emotional issues were investigated using information from interviews, blood samples and medical records.
  
  
  RESULTS
  Of the 118 individuals who participated in the trials, 67 (57%) were included. Median age was 25 years, and median time before follow-up was 10 years. Forty-eight (72%) participants were using statins at follow-up, 8 (12%) were also using ezetimibe, and 19 (28%) were not using any lipid-lowering therapy. Mean LDL-cholesterol (LDL-C) was 3.68 mmol/L in statin users and 6.08 mmol/L in non-users (P < .001). Only 6 (9%) participants reached treatment goal, ie, an LDL-C ≤2.5 mmol/L. Participants who attended a consultation ≤2 years before follow-up had a significantly lower LDL-C compared with those who had a consultation >2 years before follow-up (4.10 and 5.17 mmol/L, respectively; P = .02). Statin users had their last consultation more recently than non-users (median 1.4 and 2.2 years, respectively; P = .02).
  
  
  CONCLUSIONS
  Statins are underused in this population, and most patients have not reached treatment goal. Those with recent consultations had lower LDL-C levels and were more often statin users. Therefore, yearly consultations for young adults with FH seem warranted.
authors.affiliation: Lipid Clinic, Division of Medicine, Oslo University Hospital, Oslo, Norway. Electronic address: glangsle@ous-hf.no.
authors.forename: Gisle
authors.initials: G
authors.lastname: Langslet
authors.affiliation: Lipid Clinic, Division of Medicine, Oslo University Hospital, Oslo, Norway.
authors.forename: Martin P
authors.initials: MP
authors.lastname: Bogsrud
authors.affiliation: Department of Nutrition, Institute of Basic Medical Sciences, University of Oslo, Oslo, Norway.
authors.forename: Ida
authors.initials: I
authors.lastname: Halvorsen
authors.affiliation: Lipid Clinic, Division of Medicine, Oslo University Hospital, Oslo, Norway.
authors.forename: Heidi
authors.initials: H
authors.lastname: Fjeldstad
authors.affiliation: Department of Nutrition, Institute of Basic Medical Sciences, University of Oslo, Oslo, Norway.
authors.forename: Kjetil
authors.initials: K
authors.lastname: Retterstøl
authors.affiliation: Department of Biostatistics, Oslo Centre for Biostatistics and Epidemiology, Institute of Basic Medical Sciences, University of Oslo, Oslo, Norway.
authors.forename: Marit B
authors.initials: MB
authors.lastname: Veierød
authors.affiliation: Lipid Clinic, Division of Medicine, Oslo University Hospital, Oslo, Norway.
authors.forename: Leiv
authors.initials: L
authors.lastname: Ose
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002986:Clinical Trials as Topic; D004032:Diet; D004644:Emotions; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000188:drug therapy* / Q000523:psychology; D000960:Hypolipidemic Agents / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D008055:Lipids / Q000097:blood; D008297:Male; D064424:Tobacco Use; D055815:Young Adult
chemical_list: D000960:Hypolipidemic Agents; D008055:Lipids
keywords: Familial hypercholesterolemia; LDL-Cholesterol; Statins; Treatment adherence; Young adults