Pediatric experience with mipomersen as adjunctive therapy for homozygous familial hypercholesterolemia

Journal of Clinical Lipidology

Volumn 10, Issue 4, 2016, Pages 860-869

  Raal, Frederick J ^a   Braamskamp, Marjet J ^b,c   Selvey, Sheryl L ^d   Sensinger, Charlotte H ^d   Kastelein, John J ^b  

^a UNIVERSITY OF THE WITWATERSRAND   (South Africa)

^b ACADEMIC MEDICAL CENTER   (Netherlands)

^c EMMA CHILDREN S HOSPITAL   (Netherlands)

^d GENZYME CORPORATION   (United States)

Author keywords

Cholesterol; Hypercholesterolemia; Lipoproteins; Mipomersen; Pediatrics

Indexed keywords

ALANINE AMINOTRANSFERASE; APOLIPOPROTEIN A1; APOLIPOPROTEIN B; BILIRUBIN; C REACTIVE PROTEIN; HIGH DENSITY LIPOPROTEIN CHOLESTEROL; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE INHIBITOR; LIPOPROTEIN A; LOW DENSITY LIPOPROTEIN; MIPOMERSEN; NICOTINIC ACID; PLACEBO; SEQUESTERING AGENT; TRIACYLGLYCEROL; VERY LOW DENSITY LIPOPROTEIN CHOLESTEROL; HYPOCHOLESTEROLEMIC AGENT; OLIGONUCLEOTIDE;

ADOLESCENT; ADVERSE OUTCOME; ARTICLE; CHILD; CHOLESTEROL BLOOD LEVEL; CLINICAL ARTICLE; CLINICAL ASSESSMENT; COHORT ANALYSIS; CONCENTRATION RESPONSE; CONTROLLED STUDY; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; ERYTHEMA; FAMILIAL HYPERCHOLESTEROLEMIA; FEMALE; FLU LIKE SYNDROME; HOMOZYGOSITY; HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA; HUMAN; HYPERTRANSAMINASEMIA; INJECTION SITE DISCOLORATION; INJECTION SITE ERYTHEMA; INJECTION SITE PAIN; INJECTION SITE REACTION; KIDNEY DYSFUNCTION; LIPID ANALYSIS; MALE; MEDICATION COMPLIANCE; PAIN; PATIENT COMPLIANCE; PERSONAL EXPERIENCE; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SCHOOL CHILD; SIDE EFFECT; SYSTEMIC INFLAMMATORY RESPONSE SYNDROME; THROMBOCYTOPENIA; DRUG INTERACTION; GENETICS; HOMOZYGOTE; HYPERLIPOPROTEINEMIA TYPE II; SAFETY;

ADOLESCENT; ANTICHOLESTEREMIC AGENTS; CHILD; DRUG INTERACTIONS; FEMALE; HOMOZYGOTE; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; MALE; OLIGONUCLEOTIDES; SAFETY;

EID: 84963959871     PISSN: 19332874     EISSN: 18764789     Source Type: Journal    
DOI: 10.1016/j.jacl.2016.02.018     Document Type: Article

References (14)

1. 1 Raal, F.J., Pilcher, G.J., Panz, V.R., et al. Reduction in mortality in subjects with homozygous familial hypercholesterolemia associated with advances in lipid-lowering therapy. Circulation 124:20 (2011), 2202–2207.
2. 2 Sjouke, B., Kusters, D.M., Kindt, I., et al. Homozygous autosomal dominant hypercholesterolaemia in the Netherlands: prevalence, genotype-phenotype relationship, and clinical outcome. Eur Heart J 36:9 (2015), 560–565.
3. 3 Thompson, G.R., HEART-UK LDL Apheresis Working Group. Recommendations for the use of LDL apheresis. Atherosclerosis 198:2 (2008), 247–255.
4. 4 Braamskamp, M.J., Hutten, B.A., Wiegman, A., Kastelein, J.J., Management of hypercholesterolemia in children. Paediatr Drugs 16:2 (2014), 105–114.
5. 5 Braamskamp, M.J., Roelants, R.J., Hutten, B.A., Kastelein, J.J., The role of rosuvastatin in the treatment of pediatric homozygous familial hypercholesterolemia. Expert Opin Orphan Drugs 12 (2014), 1325–1330.
6. 6 Vogt, A., The genetics of familial hypercholesterolemia and emerging therapies. Appl Clin Genet 8 (2015), 27–36.
7. 7 Goldberg, A.C., Hopkins, P.N., Toth, P.P., et al. Familial hypercholesterolemia: screening, diagnosis and management of pediatric and adult patients. Clinical guidance from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Clin Lipidol 5:3 (2011), 133–140.
8. 8 Bell, D.A., Hooper, A.J., Burnett, J.R., Mipomersen, an antisense apolipoprotein B synthesis inhibitor. Expert Opin Investig Drugs 20:2 (2011), 265–272.
9. 9 Kynamro [package insert], 2013, Genzyme Corporation, Cambridge, MA.
10. 10 Raal, F.J., Santos, R.D., Blom, D.J., et al. Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Lancet 375:9719 (2010), 998–1006.
11. 11 Santos, R.D., Duell, P.B., East, C., et al. Long-term efficacy and safety of mipomersen in patients with familial hypercholesterolaemia: 2-year interim results of an open-label extension. Eur Heart J 36:9 (2013), 566–575.
12. 12 Santos, R.D., Raal, F.J., Catapano, A.L., Witztum, J.L., Steinhagen-Thiessen, E., Tsimikas, S., Mipomersen, an antisense oligonucleotide to apolipoprotein B-100, reduces lipoprotein(a) in various populations with hypercholesterolemia: results of 4 phase iii trials. Arterioscler Thromb Vasc Biol 35:3 (2015), 689–699.
13. 13 Wiegman, A., Gidding, S.S., Watts, G.F., et al., For the European Atherosclerosis Society Consensus Panel. Familial hypercholesterolaemia in children and adolescents: gaining decades of life by optimizing detection and treatment. Eur Heart J 36:36 (2015), 2425–2437.
14. 14 Schwartzman, S., Morgan, G.J. Jr., Does route of administration affect the outcome of TNF antagonist therapy?. Arthritis Res Ther 6:Suppl 2 (2004), S19–S23.