Long-term Statin Treatment in Children with Familial Hypercholesterolemia: More Insight into Tolerability and Adherence

Overview

Journal Paediatr Drugs

Specialties Pediatrics
Pharmacology

Date 2015 Feb 4

PMID 25644328

Citations 18

Authors

Marjet J A M Braamskamp

D Meeike Kusters

Hans J Avis

Ellen M A Smets

Frits A Wijburg

John J P Kastelein

Albert Wiegman

Barbara A Hutten

Affiliations

Soon will be listed here.

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.

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References

Benner J, Glynn R, Mogun H, Neumann P, Weinstein M, Avorn J . Long-term persistence in use of statin therapy in elderly patients. JAMA. 2002; 288(4):455-61. DOI: 10.1001/jama.288.4.455. View

Jackevicius C, Mamdani M, Tu J . Adherence with statin therapy in elderly patients with and without acute coronary syndromes. JAMA. 2002; 288(4):462-7. DOI: 10.1001/jama.288.4.462. View

Thompson P, Clarkson P, Karas R . Statin-associated myopathy. JAMA. 2003; 289(13):1681-90. DOI: 10.1001/jama.289.13.1681. View

Marks D, Thorogood M, Neil H, Humphries S . 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. View

Wiegman A, Hutten B, de Groot E, Rodenburg J, Bakker H, Buller H . Efficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized controlled trial. JAMA. 2004; 292(3):331-7. DOI: 10.1001/jama.292.3.331. View

FRIEDEWALD W, Levy R, Fredrickson D . Estimation of the concentration of low-density lipoprotein cholesterol in plasma, without use of the preparative ultracentrifuge. Clin Chem. 1972; 18(6):499-502. View

Kovacs M, Goldston D, Obrosky D, Bonar L . Psychiatric disorders in youths with IDDM: rates and risk factors. Diabetes Care. 1997; 20(1):36-44. DOI: 10.2337/diacare.20.1.36. View

Senior V, Marteau T, Weinman J . Self-reported adherence to cholesterol-lowering medication in patients with familial hypercholesterolaemia: the role of illness perceptions. Cardiovasc Drugs Ther. 2005; 18(6):475-81. DOI: 10.1007/s10557-004-6225-z. View

Osterberg L, Blaschke T . Adherence to medication. N Engl J Med. 2005; 353(5):487-97. DOI: 10.1056/NEJMra050100. View

10.

Baigent C, Keech A, Kearney P, Blackwell L, Buck G, Pollicino C . Efficacy and safety of cholesterol-lowering treatment: prospective meta-analysis of data from 90,056 participants in 14 randomised trials of statins. Lancet. 2005; 366(9493):1267-78. DOI: 10.1016/S0140-6736(05)67394-1. View

11.

Perreault S, Blais L, Dragomir A, Bouchard M, Lalonde L, Laurier C . Persistence and determinants of statin therapy among middle-aged patients free of cardiovascular disease. Eur J Clin Pharmacol. 2005; 61(9):667-74. DOI: 10.1007/s00228-005-0980-z. View

12.

Zindani G, Streetman D, Streetman D, Nasr S . Adherence to treatment in children and adolescent patients with cystic fibrosis. J Adolesc Health. 2006; 38(1):13-7. DOI: 10.1016/j.jadohealth.2004.09.013. View

13.

Bruckert E, Hayem G, Dejager S, Yau C, Begaud B . Mild to moderate muscular symptoms with high-dosage statin therapy in hyperlipidemic patients--the PRIMO study. Cardiovasc Drugs Ther. 2006; 19(6):403-14. DOI: 10.1007/s10557-005-5686-z. View

14.

Kashani A, Phillips C, Foody J, Wang Y, Mangalmurti S, Ko D . Risks associated with statin therapy: a systematic overview of randomized clinical trials. Circulation. 2006; 114(25):2788-97. DOI: 10.1161/CIRCULATIONAHA.106.624890. View

15.

McCrindle B, Urbina E, Dennison B, Jacobson M, Steinberger J, Rocchini A . 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.... Circulation. 2007; 115(14):1948-67. DOI: 10.1161/CIRCULATIONAHA.107.181946. View

16.

Bouchard M, Dragomir A, Blais L, Berard A, Pilon D, Perreault S . Impact of adherence to statins on coronary artery disease in primary prevention. Br J Clin Pharmacol. 2007; 63(6):698-708. PMC: 2000596. DOI: 10.1111/j.1365-2125.2006.02828.x. View

17.

Nachega J, Hislop M, Nguyen H, Dowdy D, Chaisson R, Regensberg L . Antiretroviral therapy adherence, virologic and immunologic outcomes in adolescents compared with adults in southern Africa. J Acquir Immune Defic Syndr. 2009; 51(1):65-71. PMC: 2674125. DOI: 10.1097/QAI.0b013e318199072e. View

18.

Horne R, Parham R, Driscoll R, Robinson A . Patients' attitudes to medicines and adherence to maintenance treatment in inflammatory bowel disease. Inflamm Bowel Dis. 2008; 15(6):837-44. DOI: 10.1002/ibd.20846. View

19.

Ho P, Bryson C, Rumsfeld J . Medication adherence: its importance in cardiovascular outcomes. Circulation. 2009; 119(23):3028-35. DOI: 10.1161/CIRCULATIONAHA.108.768986. View

20.

Perreault S, Ellia L, Dragomir A, Cote R, Blais L, Berard A . Effect of statin adherence on cerebrovascular disease in primary prevention. Am J Med. 2009; 122(7):647-55. DOI: 10.1016/j.amjmed.2009.01.032. View