Pediatric experience with mipomersen as adjunctive therapy for homozygous familial hypercholesterolemia Frederick J. Raal, PhD, Marjet J. Braamskamp,

Slides:

Advertisements

Similar presentations

Corn oil improves the plasma lipoprotein lipid profile compared with extra-virgin olive oil consumption in men and women with elevated cholesterol: Results.

Advertisements

Interrupting bile-acid handling and lipid and glucose control: Effects of colesevelam on glucose levels Michael H. Davidson, MD Journal of Clinical.

Lipid abnormalities in foreign-born and US-born patients in a medical group Jawali Jaranilla, MD, MPH, Terese DeFor, MS, Gabriela Vazquez Benitez, PhD,

Lipoprotein(a), Cardiovascular Disease, and Contemporary Management

Patient and Physician Perspectives on Administration of the PCSK9 Monoclonal Antibody Alirocumab, an Injectable Medication to Lower LDL-C Levels† Eli.

Charles R. Harper, MD, Terry A. Jacobson, MD Mayo Clinic Proceedings

2013 Cholesterol Guidelines Revisited: Percent LDL Cholesterol Reduction or Attained LDL Cholesterol Level or Both for Prognosis? Sripal Bangalore, MD,

Corn oil improves the plasma lipoprotein lipid profile compared with extra-virgin olive oil consumption in men and women with elevated cholesterol: Results.

Efficacy and safety of the proprotein convertase subtilisin/kexin type 9 monoclonal antibody alirocumab vs placebo in patients with heterozygous familial.

Efficacy of alirocumab in high cardiovascular risk populations with or without heterozygous familial hypercholesterolemia: Pooled analysis of eight ODYSSEY.

2016 Canadian Cardiovascular Society Guidelines for the Management of Dyslipidemia for the Prevention of Cardiovascular Disease in the Adult Todd J.

Purified palmitoleic acid for the reduction of high-sensitivity C-reactive protein and serum lipids: A double-blinded, randomized, placebo controlled.

Charles R. Harper, MD, Terry A. Jacobson, MD Mayo Clinic Proceedings

Rounding numbers The Journal of Thoracic and Cardiovascular Surgery

Efficacy and safety of gemcabene as add-on to stable statin therapy in hypercholesterolemic patients Evan Stein, MD, Harold Bays, MD, Michael Koren,

KDOQI Clinical Practice Guideline for Diabetes and CKD: 2012 Update

Emerging LDL therapies: Mipomersen—antisense oligonucleotide therapy in the management of hypercholesterolemia Peter P. Toth, MD, PhD, FAAFP, FICA, FNLA,

Omega-3 carboxylic acids in patients with severe hypertriglyceridemia: EVOLVE II, a randomized, placebo-controlled trial Erik S.G. Stroes, MD, PhD, Andrey.

Glucagon-like peptide-1 receptor agonists reduced the low-density lipoprotein cholesterol in Japanese patients with type 2 diabetes mellitus treated with.

Efficacy and safety of pemafibrate (K-877), a selective peroxisome proliferator-activated receptor α modulator, in patients with dyslipidemia: Results.

My Approach to the Patient With Familial Hypercholesterolemia

Icosapent ethyl (eicosapentaenoic acid ethyl ester): Effects on plasma apolipoprotein C- III levels in patients from the MARINE and ANCHOR studies Christie.

Predictors of first-year statin medication discontinuation: A cohort study Heli Halava, MD, Risto Huupponen, MD, PhD, Jaana Pentti, BSc, Mika Kivimäki,

Efficacy of alirocumab in 1191 patients with a wide spectrum of mutations in genes causative for familial hypercholesterolemia Joep C. Defesche, PhD,

Effects of icosapent ethyl on lipoprotein particle concentration and size in statin-treated patients with persistent high triglycerides (the ANCHOR Study)

A 3-year study of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia Gisle Langslet, MD, Andrei Breazna, PhD, Euridiki.

Effect of tofacitinib on lipid levels and lipid-related parameters in patients with moderate to severe psoriasis Robert Wolk, MD, PhD, DSc, Ehrin J.

Jonathan C. Cohen, PhD Journal of Clinical Lipidology

J. W. Balder, MD, PhD, P. J. Lansberg, MD, PhD, M. H. Hof, MSc, A

Efficacy and safety of alirocumab, a monoclonal antibody to PCSK9, in statin-intolerant patients: Design and rationale of ODYSSEY ALTERNATIVE, a randomized.

Meredith Kilgore, PhD, Paul Muntner, PhD, J

Non–high-density lipoprotein cholesterol target achievement in patients on lipid-lowering drugs and stratified by triglyceride levels in the Arabian Gulf

Lysosomal acid lipase deficiency: A hidden disease among cohorts of familial hypercholesterolemia? Joana Rita Chora, MSc, Ana Catarina Alves, PhD, Ana.

Charles J. Glueck, MD, Alan Brown, MD, Anne C. Goldberg, MD, James M

National Lipid Association recommendations for patient-centered management of dyslipidemia: Part 1 – executive summary Terry A. Jacobson, MD, Matthew.

Paul D. Thompson, MD, John Rubino, MD, Matthew J. Janik, MD, Diane E

Increased prevalence of clinical and subclinical atherosclerosis in patients with damaging mutations in ABCA1 or APOA1 Omar Abdel-Razek, MD, Singh N.

Ali A. Hasnie, MD, Ashok Kumbamu, PhD, Maya S

Barriers to PCSK9 inhibitor prescriptions for patients with high cardiovascular risk: Results of a healthcare provider survey conducted by the National.

An assessment by the Statin Intolerance Panel: 2014 update

Task-sharing interventions for cardiovascular risk reduction and lipid outcomes in low- and middle-income countries: A systematic review and meta-analysis

Assessment of statin therapy, LDL-C levels, and cardiovascular events among high-risk patients in the United States Sudhir K. Unni, PhD, MBA, Ruben G.W.

Effect of atorvastatin, cholesterol ester transfer protein inhibition, and diabetes mellitus on circulating proprotein subtilisin kexin type 9 and lipoprotein(a)

Long-term treatment adherence to the proprotein convertase subtilisin/kexin type 9 inhibitor alirocumab in 6 ODYSSEY Phase III clinical studies with treatment.

Corn oil improves the plasma lipoprotein lipid profile compared with extra-virgin olive oil consumption in men and women with elevated cholesterol: Results.

To B or Not to B: Is Non–High-Density Lipoprotein Cholesterol an Adequate Surrogate for Apolipoprotein B? Carl J. Lavie, MD, Richard V. Milani, MD, James.

Alirocumab dosing patterns during 40 months of open-label treatment in patients with heterozygous familial hypercholesterolemia G. Kees Hovingh, MD,

Risk-corrected impact of mechanical versus bioprosthetic valves on long-term mortality after aortic valve replacement Ole Lund, MD, PhD, Martin Bland,

Optimal high-density lipoprotein cholesterol cutoff for predicting cardiovascular disease: Comparison of the Korean and US National Health and Nutrition.

Alirocumab in patients with heterozygous familial hypercholesterolemia undergoing lipoprotein apheresis: Rationale and design of the ODYSSEY ESCAPE trial

Alirocumab efficacy in patients with double heterozygous, compound heterozygous, or homozygous familial hypercholesterolemia Merel L. Hartgers, MD, Joep.

Mipomersen preferentially reduces small low-density lipoprotein particle number in patients with hypercholesterolemia Raul D. Santos, Frederick J. Raal,

Sokunthea Peou, PharmD, Brittany Milliard-Hasting, MD, Sachin A

Familial hypercholesterolemia in a European Mediterranean population—Prevalence and clinical data from 2.5 million primary care patients Alberto Zamora,

Patterns of statin use and cholesterol goal attainment in a high-risk cardiovascular population: A retrospective study of primary care electronic medical.

Influence of low-glucose peritoneal dialysis on serum lipids and apolipoproteins in the IMPENDIA/EDEN trials Allan D. Sniderman, MD, James A. Sloand,

Efficacy, safety, and tolerability of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia: Rationale and design of the HAUSER-RCT.

Cyclophosphamide-Induced Hepatitis in Wegener's Granulomatosis

Diabetes is associated with an increased risk of cardiovascular disease in patients with familial hypercholesterolemia Martine Paquette, MSc, Sophie.

Efficacy and safety of lipoprotein apheresis in children with homozygous familial hypercholesterolemia: A systematic review Ilse K. Luirink, MD, Jim.

Alirocumab efficacy in patients with double heterozygous, compound heterozygous, or homozygous familial hypercholesterolemia Merel L. Hartgers, MD, Joep.

Individualized low-density lipoprotein cholesterol reduction with alirocumab titration strategy in heterozygous familial hypercholesterolemia: Results.

Can Low-Radiation Coronary Computed Tomography Angiography Improve Upon Clinical Risk Assessment in Patients with Familial Hypercholesterolemia?* Anna.

Prediction of Cardiovascular Disease Events by Lipoprotein(a) According to LDL-C in Older High Risk Adults Nathan D. Wong, PhD, Shravanthi R. Gandra,

PCSK9 inhibition in patients with and without prior myocardial infarction or ischemic stroke: A pooled analysis of nine randomized-controlled studies.

Emerging low-density lipoprotein (LDL) therapies: Management of severely elevated LDL cholesterol—The role of LDL-apheresis Mary P. McGowan, MD, FNLA

LINDA S. SNYDER, M.D., RUSSELL I. HEIGH, M.D., MONTE L. ANDERSON, M.D.

LOWER, a registry of lomitapide-treated patients with homozygous familial hypercholesterolemia: Rationale and design Dirk J. Blom, MD, PhD, Zahi A. Fayad,

Long-term clinical results of microsomal triglyceride transfer protein inhibitor use in a patient with homozygous familial hypercholesterolemia Anna.

National Lipid Association recommendations for patient-centered management of dyslipidemia: Part 1 – executive summary Terry A. Jacobson, MD, Matthew.

Presentation transcript:

Pediatric experience with mipomersen as adjunctive therapy for homozygous familial hypercholesterolemia Frederick J. Raal, PhD, Marjet J. Braamskamp, MD, PhD, Sheryl L. Selvey, PharmD, Charlotte H. Sensinger, MA, John J. Kastelein, MD, PhD Journal of Clinical Lipidology Volume 10, Issue 4, Pages 860-869 (July 2016) DOI: 10.1016/j.jacl.2016.02.018 Copyright © 2016 Sanofi-Genzyme Terms and Conditions

Figure 1 Mipomersen mechanism of action. apo B–100, apolipoprotein B–100; LDL-C, low-density lipoprotein cholesterol; mRNA, messenger ribonucleic acid; RNase H, ribonuclease H; VLDL, very low density lipoprotein. Reprinted from the Journal of the American College of Cardiology, Vol. 62/No.23, Thomas GS, Cromwell WC, Ali S, Chin W, Flaim JD, Davidson M, Mipomersen, an apolipoprotein B synthesis inhibitor, reduces atherogenic lipoproteins in patients with severe hypercholesterolemia at high cardiovascular risk: A Randomized, Double-Blind, Placebo-Controlled Trial, pp 2178–2184, Copyright 2013, with permission from the American College of Cardiology Foundation. Journal of Clinical Lipidology 2016 10, 860-869DOI: (10.1016/j.jacl.2016.02.018) Copyright © 2016 Sanofi-Genzyme Terms and Conditions

Figure 2 Pediatric patient flow in randomized controlled trial and open-label extension. ALT, alanine aminotransferase; AST, aspartate aminotransferase; OLE, open-label extension study; RCT, randomized controlled trial. Journal of Clinical Lipidology 2016 10, 860-869DOI: (10.1016/j.jacl.2016.02.018) Copyright © 2016 Sanofi-Genzyme Terms and Conditions

Figure 3 Low-density lipoprotein cholesterol concentration at measured time points in the randomized controlled trial and the open-label extension study. LDL-C, low-density lipoprotein cholesterol; OLE, open-label extension study; RCT, randomized controlled trial. Journal of Clinical Lipidology 2016 10, 860-869DOI: (10.1016/j.jacl.2016.02.018) Copyright © 2016 Sanofi-Genzyme Terms and Conditions

Figure 4 Apo B concentration at measured time points in the randomized controlled trial and the open-label extension study. apo B, apolipoprotein B; OLE, open-label extension study; RCT, randomized controlled trial. Journal of Clinical Lipidology 2016 10, 860-869DOI: (10.1016/j.jacl.2016.02.018) Copyright © 2016 Sanofi-Genzyme Terms and Conditions

Figure 5 Lipoprotein(a) concentration at measured time points in the randomized controlled trial and the open-label extension study. Lp(a), lipoprotein(a); OLE, open-label extension study; RCT, randomized controlled trial. Journal of Clinical Lipidology 2016 10, 860-869DOI: (10.1016/j.jacl.2016.02.018) Copyright © 2016 Sanofi-Genzyme Terms and Conditions