- •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.
There are little long-term data on patients with familial hypercholesterolemia (FH) who initiated lipid-lowering therapy during childhood.
To study long-term outcomes in young adults with FH who participated in clinical trials on lipid-lowering therapy during childhood.
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.
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).
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.
To read this article in full you will need to make a payment
Purchase one-time access:Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
One-time access price info
- For academic or personal research use, select 'Academic and Personal'
- For corporate R&D use, select 'Corporate R&D Professionals'
Subscribe:Subscribe to Journal of Clinical Lipidology
Already a print subscriber? Claim online access
Already an online subscriber? Sign in
Register: Create an account
Institutional Access: Sign in to ScienceDirect
- Familial hypercholesterolemias: prevalence, genetics, diagnosis and screening recommendations from the National Lipid Association Expert Panel on Familial Hypercholesterolemia.J Clin Lipidol. 2011; 5: S9-17
- Familial hypercholesterolemia in the Danish general population: prevalence, coronary artery disease, and cholesterol-lowering medication.J Clin Endocrinol Metab. 2012; 97: 3956-3964
- Familial hypercholesterolaemia is underdiagnosed and undertreated in the general population: guidance for clinicians to prevent coronary heart disease: Consensus Statement of the European Atherosclerosis Society.Eur Heart J. 2013; 34: 3478-3490
- Integrated guidance on the care of familial hypercholesterolaemia from the International FH Foundation.Eur J Prev Cardiol. 2015; 22: 849-854
- Reductions in all-cause, cancer, and coronary mortality in statin-treated patients with heterozygous familial hypercholesterolaemia: a prospective registry study.Eur Heart J. 2008; 29: 2625-2633
- Efficacy of statins in familial hypercholesterolaemia: a long term cohort study.BMJ. 2008; 337: a2423
- Efficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized controlled trial.JAMA. 2004; 292: 331-337
- Efficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized, double-blind, placebo-controlled trial with simvastatin.Circulation. 2002; 106: 2231-2237
- Efficacy and safety of atorvastatin in children and adolescents with familial hypercholesterolemia or severe hyperlipidemia: a multicenter, randomized, placebo-controlled trial.J Pediatr. 2003; 143: 74-80
- Efficacy and safety of rosuvastatin therapy for children with familial hypercholesterolemia.J Am Coll Cardiol. 2010; 55: 1121-1126
- Statins for children with familial hypercholesterolemia.Cochrane Database Syst Rev. 2014; : CD006401
- Expert panel on integrated guidelines for cardiovascular health and risk reduction in children and adolescents: summary report.Pediatrics. 2011; 128: S213-S256
- Treatment of familial hypercholesterolaemia in children and adolescents in the last three decades.Cardiol Young. 2014; 24: 437-441
- Ten-year follow-up after initiation of statin therapy in children with familial hypercholesterolemia.JAMA. 2014; 312: 1055-1057
- Colesevelam hydrochloride: efficacy and safety in pediatric subjects with heterozygous familial hypercholesterolemia.J Pediatr. 2010; 156: 231-236
- Efficacy and safety of coadministration of ezetimibe and simvastatin in adolescents with heterozygous familial hypercholesterolemia.J Am Coll Cardiol. 2008; 52: 1421-1429
- Plant sterol ester-enriched spread lowers plasma total and LDL cholesterol in children with familial hypercholesterolemia.Am J Clin Nutr. 2002; 76: 338-344
- Guidelines for the diagnosis and management of heterozygous familial hypercholesterolemia.Atherosclerosis. 2004; 173: 55-68
- Reproducibility and validity of a short food questionnaire for the assessment of dietary habits.Nutr Metab Cardiovasc Dis. 2002; 12: 60-70
- [Assessment of dietary habits and life style].Tidsskr Nor Laegeforen. 2011; 131: 454
- Two years after molecular diagnosis of familial hypercholesterolemia: majority on cholesterol-lowering treatment but a minority reaches treatment goal.PLoS One. 2010; 5: e9220
- Diagnosis of familial hypercholesterolemia in general practice using clinical diagnostic criteria or genetic testing as part of cascade genetic screening.Community Genet. 2008; 11: 26-35
- Evaluation of cholesterol lowering treatment of patients with familial hypercholesterolemia: a large cross-sectional study in The Netherlands.Atherosclerosis. 2010; 209: 189-194
- Long-term statin treatment in children with familial hypercholesterolemia: more insight into tolerability and adherence.Paediatr Drugs. 2015; 17: 159-166
- Risk of fatal coronary heart disease in familial hypercholesterolaemia. Scientific Steering Committee on behalf of the Simon Broome Register Group.BMJ. 1991; 303: 893-896
- Measurement of arterial wall thickness as a surrogate marker for atherosclerosis.Circulation. 2004; 109: III33-III38
- Arterial intima-media thickness in children heterozygous for familial hypercholesterolaemia.Lancet. 2004; 363: 369-370
- Markers of atherosclerotic development in children with familial hypercholesterolemia: a literature review.Atherosclerosis. 2014; 235: 299-309
- Statin treatment in children with familial hypercholesterolemia: the younger, the better.Circulation. 2007; 116: 664-668
- Familial hypercholesterolaemia in children and adolescents: gaining decades of life by optimizing detection and treatment.Eur Heart J. 2015; 36: 2425-2437
- Statin-associated muscle symptoms: impact on statin therapy-European Atherosclerosis Society Consensus Panel Statement on Assessment, Aetiology and Management.Eur Heart J. 2015; 36: 1012-1022
- 2013 AHA/ACC guideline on lifestyle management to reduce cardiovascular risk: a report of the American College of Cardiology/American Heart Association Task Force on Practice Guidelines.J Am Coll Cardiol. 2014; 63: 2960-2984
Norwegian Institute of Public Health. Røyking og snusbruk i Noreg—Folkehelserapporten 2014. Available at: http://www.fhi.no/publikasjoner-og-haandboker/folkehelserapporten. Accessed June 30, 2014.
- Quality of life, anxiety and concerns among statin-treated children with familial hypercholesterolaemia and their parents.Acta Paediatr. 2003; 92: 1096-1101
- Predictive genetic testing for cardiovascular diseases: impact on carrier children.Am J Med Genet A. 2008; 146A: 3136-3146
- Strategies of adherence promotion in the management of pediatric chronic conditions.J Dev Behav Pediatr. 2013; 34: 716-729
- Non-adherence to statin therapy: a major challenge for preventive cardiology.Expert Opin Pharmacother. 2009; 10: 2973-2985
Published online: August 28, 2015
Accepted: August 22, 2015
Received: April 10, 2015
© 2015 National Lipid Association. Published by Elsevier Inc. All rights reserved.