| Title | Colesevelam Hydrochloride: Efficacy and Safety in Pediatric Subjects with Heterozygous Familial Hypercholesterolemia. | | Author(s) | Stein EA, Marais AD, Szamosi T, Raal FJ, Schurr D, Urbina EM, Hopkins PN, Karki S, Xu J, Misir S, Melino M | | Institution | From Metabolic and Atherosclerosis Research Center (E.A.S.), and Cincinnati Children's Hospital (E.M.U.), Cincinnati, OH, Cardiovascular Genetics, Research Clinic, University of Utah, Salt Lake City, UT (P.N.H.), Daiichi Sankyo Pharma Development, Edison (S.K., S.M., M.M.), and Daiichi Sankyo, Inc., Parsippany (J.X.), NJ, University of Cape Town Health Science Faculty, Observatory, Cape Town (A.D.M.), and Carbohydrate and Lipid Metabolism Research Unit, Johannesburg Hospital, Parktown (F.J.R.), South Africa, the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); and the Center for Research, Prevention and Treatment of Atherosclerosis, Hadassah University Hospital, Kiryat Hadassah, Jerusalem, Israel (D.S.). | | Source | J Pediatr 2009 Oct 29. | | Abstract | OBJECTIVE: Evaluate the efficacy and safety of colesevelam hydrochloride in children with heterozygous familial hypercholesterolemia (heFH).
STUDY DESIGN: This was a randomized, double-blind, 41-site study in 194 children aged 10 to 17 years (inclusive) with heFH (statin-naïve or on a stable statin regimen). After a 4-week stabilization period (period I), subjects were randomized 1:1:1 to placebo, colesevelam 1.875 g/d, or colesevelam 3.75 g/d for 8 weeks (period II). All then received open-label colesevelam 3.75 g/d for 18 weeks (period III), with follow-up 2 weeks later. The primary endpoint was percent change in low-density lipoprotein (LDL)-cholesterol from baseline to week 8. Secondary endpoints included percent change in other lipoprotein variables, including non-high-density lipoprotein (non-HDL)-cholesterol. Adverse events were also evaluated.
RESULTS: At week 8, a significant difference from baseline in LDL-cholesterol was reported with colesevelam 1.875 g/d (-6.3%; P = .031) and colesevelam 3.75 g/d (-12.5%; P < .001) compared with placebo. Significant treatment effects were also reported for total cholesterol (-7.4%), non-HDL-cholesterol (-10.9%), HDL-cholesterol (+6.1%), apolipoprotein A-I (+6.9%), and apolipoprotein B (-8.3%) and a nonsignificant effect for triglycerides (+5.1%) with colesevelam 3.75 g/d compared with placebo at week 8. These treatment effects were maintained during period III.
CONCLUSIONS: Colesevelam significantly lowered LDL-cholesterol levels in children with heFH. | | Language | ENG | | Pub Type(s) | JOURNAL ARTICLE
| | PubMed ID | 19879596 |
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