PMID- 26687694 OWN - NLM STAT- MEDLINE DCOM- 20160921 LR - 20170729 IS - 1933-2874 (Print) IS - 1876-4789 (Linking) VI - 9 IP - 6 DP - 2015 Nov-Dec TI - Efficacy and safety of rosuvastatin therapy in children and adolescents with familial hypercholesterolemia: Results from the CHARON study. PG - 741-750 LID - S1933-2874(15)00320-7 [pii] LID - 10.1016/j.jacl.2015.07.011 [doi] AB - OBJECTIVE: Heterozygous familial hypercholesterolemia (HeFH) is an autosomal dominant disorder leading to premature atherosclerosis. Guidelines recommend initiating statins early to reduce low-density lipoprotein cholesterol (LDL-C). Studies have evaluated rosuvastatin in children aged >/=10 years, but its efficacy and safety in younger children is unknown. METHODS: Children with HeFH and fasting LDL-C >4.92 mmol/L (190 mg/dL) or >4.10 mmol/L (>158 mg/dL) with other cardiovascular risk factors received rosuvastatin 5 mg daily. Based on LDL-C targets (<2.85 mmol/L [<110 mg/dL]), rosuvastatin could be uptitrated to 10 mg (aged 6-9 years) or 20 mg (aged 10-17 years). Treatment lasted 2 years. Changes in lipid values, growth, sexual maturation, and adverse events (AEs) were assessed. RESULTS: The intention-to-treat analysis included 197 patients. At 24 months, LDL-C was reduced by 43, 45, and 35% vs baseline in patients aged 6-9, 10-13, and 14-17 years, respectively (P < .001 for all groups). Most AEs were mild. Intermittent myalgia was reported in 11 (6%) patients and did not lead to discontinuation of rosuvastatin treatment. Serious AEs were reported by 9 (5%) patients, all considered unrelated to treatment by the investigators. No clinically important changes in hepatic biochemistry were reported. Rosuvastatin treatment did not appear to adversely affect height, weight, or sexual maturation. CONCLUSIONS: In HeFH patients aged 6-17 years, rosuvastatin 5-20 mg over 2 years significantly reduced LDL-C compared with baseline. Treatment was well tolerated, with no adverse effects on growth or sexual maturation. CI - Copyright (c) 2015 National Lipid Association. Published by Elsevier Inc. All rights reserved. FAU - Braamskamp, Marjet J A M AU - Braamskamp MJAM AD - Department of Vascular Medicine, Academic Medical Center, Amsterdam, The Netherlands; Department of Pediatrics, Academic Medical Center, Amsterdam, The Netherlands. Electronic address: J.A.Braamskamp@amc.uva.nl. FAU - Langslet, Gisle AU - Langslet G AD - Lipid Clinic, Oslo University Hospital, Oslo, Norway. Electronic address: glangsle@ous-hf.no. FAU - McCrindle, Brian W AU - McCrindle BW AD - Department of Pediatrics, Labatt Family Health Center, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada. FAU - Cassiman, David AU - Cassiman D AD - Department of Hepatology and Metabolic Center, University Hospitals Leuven, Leuven, Belgium. FAU - Francis, Gordon A AU - Francis GA AD - Department of Medicine, Healthy Heart Program Prevention Clinic, University of British Columbia, Vancouver, BC, Canada. FAU - Gagne, Claude AU - Gagne C AD - Clinique des Maladies Lipidiques de Quebec, Quebec, QC, Canada. FAU - Gaudet, Daniel AU - Gaudet D AD - Department of Medicine, Universite de Montreal, Chicoutimi, QC, Canada. FAU - Morrison, Katherine M AU - Morrison KM AD - Department of Pediatrics, McMaster University, Hamilton, ON, Canada. FAU - Wiegman, Albert AU - Wiegman A AD - Department of Pediatrics, Academic Medical Center, Amsterdam, The Netherlands. FAU - Turner, Traci AU - Turner T AD - Metabolic & Atherosclerosis Research Center, Cincinnati, OH, USA. FAU - Kusters, D Meeike AU - Kusters DM AD - Department of Vascular Medicine, Academic Medical Center, Amsterdam, The Netherlands; Department of Pediatrics, Academic Medical Center, Amsterdam, The Netherlands. FAU - Miller, Elinor AU - Miller E AD - Department of Research and Development, AstraZeneca LP, Wilmington, DE, USA. FAU - Raichlen, Joel S AU - Raichlen JS AD - Department of Research and Development, AstraZeneca LP, Wilmington, DE, USA. FAU - Wissmar, Jenny AU - Wissmar J AD - Department of Biometrics and Information Sciences, AstraZeneca, Molndal, Sweden. FAU - Martin, Paul D AU - Martin PD AD - Department of Pharmacology, AstraZeneca, Macclesfield, Cheshire, UK. FAU - Stein, Evan A AU - Stein EA AD - Metabolic & Atherosclerosis Research Center, Cincinnati, OH, USA. FAU - Kastelein, John J P AU - Kastelein JJP AD - Department of Vascular Medicine, Academic Medical Center, Amsterdam, The Netherlands. LA - eng PT - Clinical Trial PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20150801 PL - United States TA - J Clin Lipidol JT - Journal of clinical lipidology JID - 101300157 RN - 0 (Cholesterol, LDL) RN - 83MVU38M7Q (Rosuvastatin Calcium) SB - IM MH - Adolescent MH - Child MH - Cholesterol, LDL/blood MH - Female MH - Humans MH - Hyperlipoproteinemia Type II/blood/*drug therapy MH - Male MH - Rosuvastatin Calcium/*adverse effects/pharmacokinetics/*therapeutic use MH - *Safety MH - Treatment Outcome OTO - NOTNLM OT - Children OT - Heterozygous familial hypercholesterolemia OT - Low-density lipoprotein cholesterol OT - Rosuvastatin OT - Safety OT - Tolerability EDAT- 2015/12/22 06:00 MHDA- 2016/09/23 06:00 CRDT- 2015/12/22 06:00 PHST- 2014/11/11 00:00 [received] PHST- 2015/02/27 00:00 [revised] PHST- 2015/07/26 00:00 [accepted] PHST- 2015/12/22 06:00 [entrez] PHST- 2015/12/22 06:00 [pubmed] PHST- 2016/09/23 06:00 [medline] AID - S1933-2874(15)00320-7 [pii] AID - 10.1016/j.jacl.2015.07.011 [doi] PST - ppublish SO - J Clin Lipidol. 2015 Nov-Dec;9(6):741-750. doi: 10.1016/j.jacl.2015.07.011. Epub 2015 Aug 1.