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

Braamskamp MJ, Kusters DM, Avis HJ, Smets EM, Wijburg FA, Kastelein JJ, Wiegman A, Hutten BA - Paediatr Drugs (2015)

Bottom Line: In adults, these agents have been proven to be safe and well tolerated; however, non-adherence is a significant clinical issue.None of the patient characteristics were significantly associated with adherence.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.

View Article: PubMed Central - PubMed

Affiliation: Department of Vascular Medicine, Academic Medical Center, Meibergdreef 9, 1105 AZ, Amsterdam, The Netherlands, j.a.braamskamp@amc.uva.nl.

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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Related in: MedlinePlus

Flow chart of familial hypercholesterolemia (FH) patients enrolled in the study
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Related In: Results  -  Collection


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Fig1: Flow chart of familial hypercholesterolemia (FH) patients enrolled in the study

Mentions: From the original patient cohort of 214 children, nine subjects (4.2 %) were not available for follow-up because they refused consent (n = 5) or had moved abroad (n = 2), and one male adolescent had died after a motorcycle accident (Fig. 1). One male was excluded, because he had not completed the questionnaire. Of the remaining 205 subjects, the mean age [±standard deviation (SD)] was 24.0 (±3.2) years and 45.9 % were male. Furthermore, 76 patients (37.1 %) had a family history with CVD in a first-degree relative. Demographic characteristics and lipid profiles at the start of the randomized trial and after follow-up are depicted in Table 1.Fig. 1


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

Braamskamp MJ, Kusters DM, Avis HJ, Smets EM, Wijburg FA, Kastelein JJ, Wiegman A, Hutten BA - Paediatr Drugs (2015)

Flow chart of familial hypercholesterolemia (FH) patients enrolled in the study
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

Show All Figures
getmorefigures.php?uid=PMC4372689&req=5

Fig1: Flow chart of familial hypercholesterolemia (FH) patients enrolled in the study
Mentions: From the original patient cohort of 214 children, nine subjects (4.2 %) were not available for follow-up because they refused consent (n = 5) or had moved abroad (n = 2), and one male adolescent had died after a motorcycle accident (Fig. 1). One male was excluded, because he had not completed the questionnaire. Of the remaining 205 subjects, the mean age [±standard deviation (SD)] was 24.0 (±3.2) years and 45.9 % were male. Furthermore, 76 patients (37.1 %) had a family history with CVD in a first-degree relative. Demographic characteristics and lipid profiles at the start of the randomized trial and after follow-up are depicted in Table 1.Fig. 1

Bottom Line: In adults, these agents have been proven to be safe and well tolerated; however, non-adherence is a significant clinical issue.None of the patient characteristics were significantly associated with adherence.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.

View Article: PubMed Central - PubMed

Affiliation: Department of Vascular Medicine, Academic Medical Center, Meibergdreef 9, 1105 AZ, Amsterdam, The Netherlands, j.a.braamskamp@amc.uva.nl.

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.

Show MeSH
Related in: MedlinePlus