Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Randomized Controlled Trial
. 2015 Apr;17(2):159-66.
doi: 10.1007/s40272-014-0116-y.

Long-term statin treatment in children with familial hypercholesterolemia: more insight into tolerability and adherence

Affiliations
Randomized Controlled Trial

Long-term statin treatment in children with familial hypercholesterolemia: more insight into tolerability and adherence

Marjet J A M Braamskamp et al. Paediatr Drugs. 2015 Apr.

Abstract

Background: Statins are currently the preferred pharmacological therapy in individuals with familial hypercholesterolemia (FH) with the aim to prevent premature atherosclerosis. In adults, these agents have been proven to be safe and well tolerated; however, non-adherence is a significant clinical issue.

Objectives: In this study, we evaluated tolerability and adherence to statin therapy in young adult FH patients 10 years after this was initiated in their childhood.

Methods: A questionnaire including items on medical history, adherence and reasons for discontinuation was sent to 214 young adult FH patients that initiated statin therapy at least 10 years ago. Tolerability was defined as 100% minus the percentage of patients that discontinued statin therapy due to side effects. Adherence was defined as the extent to which patients took their medication as prescribed by their physician. We labelled patients adherent if they took 80% or more of their pills in the month preceding our assessment.

Results: Follow-up was successful in 205 (95.8%) subjects (age 18-30 years). A history of side effects was reported by 40 (19.5%) of the patients, and mainly consisted of muscle complaints and gastrointestinal symptoms. Three patients (1.5%) discontinued statin therapy because of side effects. Rhadbomyolysis or other serious adverse events were not reported. In fact, 169 (82.4%) of 205 patients remained on statin treatment and 78.7% (148 out of 188) were adherent. None of the patient characteristics were significantly associated with adherence.

Conclusions: Individuals with FH who started statin therapy in childhood demonstrated good adherence during ten years of treatment. Furthermore, statin therapy was well tolerated; only a small minority discontinued therapy because of side effects and the side effects that were reported were mild in nature.

PubMed Disclaimer

Figures

Fig. 1
Fig. 1
Flow chart of familial hypercholesterolemia (FH) patients enrolled in the study
Fig. 2
Fig. 2
Number of patients that had ever experienced side effects in the last 10 years. Other: frequent urination (2×), weight reduction, hair loss, forgetfulness

Similar articles

Cited by

References

    1. Marks D, Thorogood M, Neil HAW, Humphries SE. A review on the diagnosis, natural history, and treatment of familial hypercholesterolaemia. Atherosclerosis. 2003;168(1):1–14. doi: 10.1016/S0021-9150(02)00330-1. - DOI - PubMed
    1. Goldstein JL, Hobbs HH, Brown MS. PART 12: LIPIDS Chapter 120: Familial Hypercholesterolemia Joseph L. Goldstein, Helen H. Hobbs, Michael S. Brown. In: Scriver CR, Beaudet AL, Sly WS, Valle D, editors. The Metabolic and Molecular Bases of Inherited Disease. 8th ed.; 2001. p. 2863–2913.
    1. Daniels SR. Screening and treatment of dyslipidemias in children and adolescents. Horm Res Paediatr. 2011;76(suppl 1):47–51. doi: 10.1159/000329163. - DOI - PubMed
    1. McCrindle BW, Urbina EM, Dennison BA, et al. Drug therapy of high-risk lipid abnormalities in children and adolescents: a scientific statement from the American Heart Association Atherosclerosis, Hypertension, and Obesity in Youth Committee, Council of Cardiovascular Disease in the Young, with the C. Circulation. 2007;115(14):1948–1967. doi: 10.1161/CIRCULATIONAHA.107.181946. - DOI - PubMed
    1. Kusters DM, Avis HJ, de Groot E, et al. Ten-year follow-up after initiation of statin therapy in children with familial hypercholesterolemia. JAMA. 2014;312(10):1055–1057. doi: 10.1001/jama.2014.8892. - DOI - PubMed

Publication types

Substances