caa a22 4500 445824859 CHVBK 20180317145256.0 cr unu---uuuuu 170323e20110401xx s 000 0 eng 10.1007/s00246-011-9885-z doi (NATIONALLICENCE)springer-10.1007/s00246-011-9885-z An Eight-Week Trial Investigating the Efficacy and Tolerability of Atorvastatin for Children and Adolescents With Heterozygous Familial Hypercholesterolemia [Elektronische Daten] [Kuan Gandelman, Paul Glue, Rachel Laskey, Jonathan Jones, Robert LaBadie, Leiv Ose] This study aimed to assess the efficacy and tolerability of atorvastatin in Tanner stage (TS) 1 patients ages 6 to 10years and TS ≥2 patients ages 10 to <18years with genetically confirmed heterozygous familial hypercholesterolemia (HeFH) and a low density lipoprotein cholesterol (LDL-C) level of 4mmol/l (155mg/dl) or higher. In this open-label, 8-week study, 15 TS 1 children were treated initially with atorvastatin 5mg/day and 24 TS ≥2 children with 10mg/day. Doses were doubled at week 4 if the LDL-C target (<3.35mmol/l [130mg/dl]) was not achieved. The efficacy variables were the percentage change from baseline in LDL-C, total cholesterol (TC), triglycerides (TG), high density lipoprotein cholesterol (HDL-C), very low density lipoprotein cholesterol (VLDL-C), and apolipoprotein (Apo) A-I and Apo B. Safety evaluations included clinical monitoring, subject-reported adverse events (AEs), vital signs, and clinical laboratory tests. The mean values for LDL-C, TC, VLDL-C, and Apo B decreased by week 2 among all TS 1 and TS ≥2 patients, whereas TG, HDL-C, and Apo A-I varied considerably from week to week. After 8weeks, the mean reduction in LDL-C was −40.7%±8.4 for the TS 1 children and −39.7%±10.3 for the TS ≥2 children. For the TS 1 patients, the mean reductions were −34.1%±6.9 for TC and −6.0%±32.1 for TG. The corresponding changes for the TS ≥2 patients were −35.6%±9.5 for TC and −21.1%±29.7 for TG. Four patients experienced mild to moderate treatment-related AEs. No serious AEs or discontinuations were reported. Overall, no difference in safety or tolerability was observed between the younger and older cohorts. Across the range of exposures after atorvastatin 5 to 10mg (TS 1) or atorvastatin 10 to 20mg (TS ≥2) doses for 8weeks, clinically meaningful reductions in LDL-C, TC, VLDL-C, and Apo were observed with atorvastatin in pediatric patients who had HeFH. Atorvastatin also was well tolerated in this population. The Author(s), 2011 Heterozygous familial hypocholesterolemia nationallicence Low density lipoprotein cholesterol nationallicence Statin nationallicence Gandelman Kuan Pfizer Inc, 235 East 42nd Street, 10017, New York, NY, USA aut Glue Paul Dunedin School of Medicine, P.O. Box 913, Dunedin, New Zealand aut Laskey Rachel Pfizer Inc, 235 East 42nd Street, 10017, New York, NY, USA aut Jones Jonathan Pfizer Ltd, Walton Oaks, Surrey, UK aut LaBadie Robert Pfizer Inc, 235 East 42nd Street, 10017, New York, NY, USA aut Ose Leiv Department of Medicine, Oslo University Hospital, Rikshospitalet, Oslo, Norway aut Pediatric Cardiology Springer-Verlag 32/4(2011-04-01), 433-441 0172-0643 32:4<433 2011 32 246 https://doi.org/10.1007/s00246-011-9885-z text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1007/s00246-011-9885-z text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Gandelman Kuan Pfizer Inc, 235 East 42nd Street, 10017, New York, NY, USA aut NATIONALLICENCE 700 1- Glue Paul Dunedin School of Medicine, P.O. Box 913, Dunedin, New Zealand aut NATIONALLICENCE 700 1- Laskey Rachel Pfizer Inc, 235 East 42nd Street, 10017, New York, NY, USA aut NATIONALLICENCE 700 1- Jones Jonathan Pfizer Ltd, Walton Oaks, Surrey, UK aut NATIONALLICENCE 700 1- LaBadie Robert Pfizer Inc, 235 East 42nd Street, 10017, New York, NY, USA aut NATIONALLICENCE 700 1- Ose Leiv Department of Medicine, Oslo University Hospital, Rikshospitalet, Oslo, Norway aut NATIONALLICENCE 773 0- Pediatric Cardiology Springer-Verlag 32/4(2011-04-01), 433-441 0172-0643 32:4<433 2011 32 246 Metadata rights reserved Springer special CC-BY-NC licence nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-springer