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

Marjet J A M Braamskamp1, D Meeike Kusters, Hans J Avis

  • 1Department of Vascular Medicine, Academic Medical Center, Meibergdreef 9, 1105 AZ, Amsterdam, The Netherlands, j.a.braamskamp@amc.uva.nl.

Paediatric Drugs
|February 4, 2015
PubMed

Insights

Young adults with familial hypercholesterolemia (FH) showed good adherence and tolerability to statin therapy initiated in childhood. Most patients continued treatment for ten years with mild side effects, indicating long-term treatment success.

Area of Science:

  • Cardiology
  • Pharmacology
  • Genetics

Background:

  • Familial hypercholesterolemia (FH) management aims to prevent premature atherosclerosis using statins.
  • While statins are effective and generally safe in adults, non-adherence is a significant challenge.

Purpose of the Study:

  • To assess the long-term tolerability and adherence to statin therapy in young adult patients with FH.
  • To evaluate outcomes 10 years after initiating statin treatment during childhood.

Main Methods:

  • A questionnaire was administered to 214 young adult FH patients who started statin therapy at least 10 years prior.
  • Adherence was defined as taking 80% or more of prescribed medication.
  • Tolerability was assessed by the percentage of patients discontinuing therapy due to side effects.

Main Results:

  • Follow-up was achieved in 205 patients (age 18-30 years).
  • 19.5% reported mild side effects (muscle/GI complaints); only 1.5% discontinued therapy.
  • 82.4% remained on statins, with 78.7% demonstrating good adherence; no serious adverse events occurred.

Conclusions:

  • Childhood-initiated statin therapy in FH patients is well-tolerated and demonstrates good long-term adherence.
  • Mild side effects were reported by a minority, with most patients continuing treatment successfully for a decade.
Abstract

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