Highlights
- •Ten-year follow-up of 67 young adults with familial hypercholesterolemia (FH).
- •One in 4 did not use statins and only 9% had an LDL-cholesterol (LDL-C) ≤2.5 mmol/L.
- •LDL-C was significantly lower when time since last consultation was ≤2 years.
- •Yearly consultations for young adults with FH seem warranted.
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.
Keywords
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Article info
Publication history
Published online: August 28, 2015
Accepted:
August 22,
2015
Received:
April 10,
2015
Identification
Copyright
© 2015 National Lipid Association. Published by Elsevier Inc. All rights reserved.
