PMID- 21259004
OWN - NLM
STAT- MEDLINE
DCOM- 20110811
LR  - 20240330
IS  - 1432-1971 (Electronic)
IS  - 0172-0643 (Print)
IS  - 0172-0643 (Linking)
VI  - 32
IP  - 4
DP  - 2011 Apr
TI  - An eight-week trial investigating the efficacy and tolerability of atorvastatin 
      for children and adolescents with heterozygous familial hypercholesterolemia.
PG  - 433-41
LID - 10.1007/s00246-011-9885-z [doi]
AB  - This study aimed to assess the efficacy and tolerability of atorvastatin in 
      Tanner stage (TS) 1 patients ages 6 to 10 years and TS >/= 2 patients ages 10 to 
      <18 years with genetically confirmed heterozygous familial hypercholesterolemia 
      (HeFH) and a low density lipoprotein cholesterol (LDL-C) level of 4 mmol/l (155 
      mg/dl) or higher. In this open-label, 8-week study, 15 TS 1 children were treated 
      initially with atorvastatin 5 mg/day and 24 TS >/= 2 children with 10 mg/day. Doses 
      were doubled at week 4 if the LDL-C target (<3.35 mmol/l [130 mg/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 8 weeks, 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 10 mg (TS 1) 
      or atorvastatin 10 to 20 mg (TS >/= 2) doses for 8 weeks, 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.
FAU - Gandelman, Kuan
AU  - Gandelman K
AD  - Pfizer Inc, 235 East 42nd Street, New York, NY 10017, USA. 
      kuan.gandelman@pfizer.com
FAU - Glue, Paul
AU  - Glue P
FAU - Laskey, Rachel
AU  - Laskey R
FAU - Jones, Jonathan
AU  - Jones J
FAU - LaBadie, Robert
AU  - LaBadie R
FAU - Ose, Leiv
AU  - Ose L
LA  - eng
PT  - Comparative Study
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20110123
PL  - United States
TA  - Pediatr Cardiol
JT  - Pediatric cardiology
JID - 8003849
RN  - 0 (Cholesterol, HDL)
RN  - 0 (Cholesterol, LDL)
RN  - 0 (Heptanoic Acids)
RN  - 0 (Hydroxymethylglutaryl-CoA Reductase Inhibitors)
RN  - 0 (Pyrroles)
RN  - A0JWA85V8F (Atorvastatin)
SB  - IM
MH  - Adolescent
MH  - Atorvastatin
MH  - Child
MH  - Cholesterol, HDL/*blood/drug effects
MH  - Cholesterol, LDL/*blood/drug effects
MH  - Dose-Response Relationship, Drug
MH  - *Drug Tolerance
MH  - Female
MH  - Follow-Up Studies
MH  - Heptanoic Acids/administration & dosage/*therapeutic use
MH  - Heterozygote
MH  - Humans
MH  - Hydroxymethylglutaryl-CoA Reductase Inhibitors/administration & 
      dosage/*therapeutic use
MH  - Hyperlipoproteinemia Type II/blood/*drug therapy/genetics
MH  - Male
MH  - Pyrroles/administration & dosage/*therapeutic use
MH  - Retrospective Studies
MH  - Treatment Outcome
PMC - PMC3061213
EDAT- 2011/01/25 06:00
MHDA- 2011/08/13 06:00
PMCR- 2011/01/23
CRDT- 2011/01/25 06:00
PHST- 2010/08/20 00:00 [received]
PHST- 2011/01/03 00:00 [accepted]
PHST- 2011/01/25 06:00 [entrez]
PHST- 2011/01/25 06:00 [pubmed]
PHST- 2011/08/13 06:00 [medline]
PHST- 2011/01/23 00:00 [pmc-release]
AID - 9885 [pii]
AID - 10.1007/s00246-011-9885-z [doi]
PST - ppublish
SO  - Pediatr Cardiol. 2011 Apr;32(4):433-41. doi: 10.1007/s00246-011-9885-z. Epub 2011 
      Jan 23.