Long-term follow-up of young adults with familial hypercholesterolemia after participation in clinical trials during childhood

摘要: Background There are little long-term data on patients with familial hypercholesterolemia (FH) who initiated lipid-lowering therapy during childhood. Objective To study outcomes in young adults FH participated clinical trials Methods Participants at least 1 of 6 that took place between 1999 and 2008 were interviewed 2011 or 2013. Frequency medical consultations, use therapy, lipid levels, side effects, diet, tobacco use, emotional issues investigated using information from interviews, blood samples records. Results Of the 118 individuals trials, 67 (57%) included. Median age was 25 years, median time before follow-up 10 years. Forty-eight (72%) participants statins follow-up, 8 (12%) also ezetimibe, 19 (28%) not any therapy. Mean LDL-cholesterol (LDL-C) 3.68 mmol/L statin users 6.08 mmol/L non-users ( P 2 years (4.10 5.17 mmol/L, respectively;  = .02). Statin had their last consultation more recently than (median 1.4 2.2 years, Conclusions Statins underused this population, most have reached treatment goal. Those recent consultations lower LDL-C levels often users. Therefore, yearly for seem warranted.