Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia.

Adolescent Antibodies, Monoclonal, Humanized / adverse effects Anticholesteremic Agents / adverse effects Child Cholesterol, LDL / blood Double-Blind Method Drug Therapy, Combination Female Heterozygote Humans Hydroxymethylglutaryl-CoA Reductase Inhibitors / therapeutic use Hyperlipoproteinemia Type II / drug therapy Lipids / blood Male PCSK9 Inhibitors Treatment Outcome

Journal

The New England journal of medicine
ISSN: 1533-4406
Titre abrégé: N Engl J Med
Pays: United States
ID NLM: 0255562

Informations de publication

Date de publication:
01 10 2020
Historique:
pubmed: 1 9 2020
medline: 21 10 2020
entrez: 1 9 2020
Statut: ppublish

Résumé

Evolocumab, a fully human monoclonal antibody directed against proprotein convertase subtilisin-kexin type 9, is widely used in adult patients to lower low-density lipoprotein (LDL) cholesterol levels. Its effects in pediatric patients with heterozygous familial hypercholesterolemia are not known. We conducted a 24-week, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia. Patients 10 to 17 years of age who had received stable lipid-lowering treatment for at least 4 weeks before screening and who had an LDL cholesterol level of 130 mg per deciliter (3.4 mmol per liter) or more and a triglyceride level of 400 mg per deciliter (4.5 mmol per liter) or less were randomly assigned in a 2:1 ratio to receive monthly subcutaneous injections of evolocumab (420 mg) or placebo. The primary end point was the percent change in LDL cholesterol level from baseline to week 24; key secondary end points were the mean percent change in LDL cholesterol level from baseline to weeks 22 and 24 and the absolute change in LDL cholesterol level from baseline to week 24. A total of 157 patients underwent randomization and received evolocumab (104 patients) or placebo (53 patients). At week 24, the mean percent change from baseline in LDL cholesterol level was -44.5% in the evolocumab group and -6.2% in the placebo group, for a difference of -38.3 percentage points (P<0.001). The absolute change in the LDL cholesterol level was -77.5 mg per deciliter (-2.0 mmol per liter) in the evolocumab group and -9.0 mg per deciliter (-0.2 mmol per liter) in the placebo group, for a difference of -68.6 mg per deciliter (-1.8 mmol per liter) (P<0.001). Results for all secondary lipid variables were significantly better with evolocumab than with placebo. The incidence of adverse events that occurred during the treatment period was similar in the evolocumab and placebo groups. In this trial involving pediatric patients with familial hypercholesterolemia, evolocumab reduced the LDL cholesterol level and other lipid variables. (Funded by Amgen; HAUSER-RCT ClinicalTrials.gov number, NCT02392559.).

Sections du résumé

BACKGROUND
Evolocumab, a fully human monoclonal antibody directed against proprotein convertase subtilisin-kexin type 9, is widely used in adult patients to lower low-density lipoprotein (LDL) cholesterol levels. Its effects in pediatric patients with heterozygous familial hypercholesterolemia are not known.
METHODS
We conducted a 24-week, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia. Patients 10 to 17 years of age who had received stable lipid-lowering treatment for at least 4 weeks before screening and who had an LDL cholesterol level of 130 mg per deciliter (3.4 mmol per liter) or more and a triglyceride level of 400 mg per deciliter (4.5 mmol per liter) or less were randomly assigned in a 2:1 ratio to receive monthly subcutaneous injections of evolocumab (420 mg) or placebo. The primary end point was the percent change in LDL cholesterol level from baseline to week 24; key secondary end points were the mean percent change in LDL cholesterol level from baseline to weeks 22 and 24 and the absolute change in LDL cholesterol level from baseline to week 24.
RESULTS
A total of 157 patients underwent randomization and received evolocumab (104 patients) or placebo (53 patients). At week 24, the mean percent change from baseline in LDL cholesterol level was -44.5% in the evolocumab group and -6.2% in the placebo group, for a difference of -38.3 percentage points (P<0.001). The absolute change in the LDL cholesterol level was -77.5 mg per deciliter (-2.0 mmol per liter) in the evolocumab group and -9.0 mg per deciliter (-0.2 mmol per liter) in the placebo group, for a difference of -68.6 mg per deciliter (-1.8 mmol per liter) (P<0.001). Results for all secondary lipid variables were significantly better with evolocumab than with placebo. The incidence of adverse events that occurred during the treatment period was similar in the evolocumab and placebo groups.
CONCLUSIONS
In this trial involving pediatric patients with familial hypercholesterolemia, evolocumab reduced the LDL cholesterol level and other lipid variables. (Funded by Amgen; HAUSER-RCT ClinicalTrials.gov number, NCT02392559.).

Identifiants

DOI: 10.1056/NEJMoa2019910 PMID: 32865373
pubmed: 32865373
doi: 10.1056/NEJMoa2019910
doi:

Substances chimiques

Antibodies, Monoclonal, Humanized 0
Anticholesteremic Agents 0
Cholesterol, LDL 0
Hydroxymethylglutaryl-CoA Reductase Inhibitors 0
Lipids 0
PCSK9 Inhibitors 0
PCSK9 protein, human EC 3.4.21.-
evolocumab LKC0U3A8NJ

Banques de données

ClinicalTrials.gov
['NCT02392559']

Types de publication

Journal Article Multicenter Study Randomized Controlled Trial Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1317-1327

Investigateurs

D Sullivan (D)
S Greber-Platzer (S)
B Simma (B)
D Weghuber (D)
O Descamps (O)
E Sokal (E)
P Witters (P)
R D Santos (RD)
M H Costa Gurgel Castelo (MH)
M C Izar (MC)
J L Cunha Borges (JL)
J Bergeron (J)
D Gaudet (D)
J St Pierre (J)
R Campo Torrenegra (R)
A E Quintero Baiz (AE)
J Maly (J)
G Kolovou (G)
T Szamosi (T)
C Stefanutti (C)
A Bartuli (A)
L De Sanctis (L)
M Arca (M)
T Sampietro (T)
M Averna (M)
M Bin Mohamed (M)
V Gerdes (V)
H Risstad (H)
C Tondel (C)
E Asprusten (E)
M Hennig (M)
M Salgado (M)
M Kostik (M)
U Groselj (U)
L Burgess (L)
J L Diaz (JL)
F Fuentes Jimenez (F)
J I Vidal Pardo (JI)
F Mach (F)
A Miserez (A)
M Coker (M)
I Okur (I)
S Santra (S)
J Kelley (J)
J Mahgerefteh (J)
J Steinberger (J)
T Turner (T)
F Zappalla (F)

Commentaires et corrections

Type : CommentIn
Type : CommentIn
Type : CommentIn

Informations de copyright

Copyright © 2020 Massachusetts Medical Society.

Auteurs

Raul D Santos (RD)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).
ORCID: 0000-0002-9860-6582

Andrea Ruzza (A)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

G Kees Hovingh (GK)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).
ORCID: 0000-0002-8043-951X

Albert Wiegman (A)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

François Mach (F)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Christopher E Kurtz (CE)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Andrew Hamer (A)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Ian Bridges (I)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Andrea Bartuli (A)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Jean Bergeron (J)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Tamás Szamosi (T)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Saikat Santra (S)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Claudia Stefanutti (C)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Olivier S Descamps (OS)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Susanne Greber-Platzer (S)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Ilse Luirink (I)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

John J P Kastelein (JJP)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

Daniel Gaudet (D)

From the Lipid Clinic Heart Institute, University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo (R.D.S.); Amgen, Thousand Oaks, CA (A.R., C.E.K, A.H.); the Departments of Vascular Medicine (G.K.H., J.J.P.K.) and Pediatrics (A.W., I.L.), Amsterdam UMC, Amsterdam; the Cardiology Department, Geneva University Hospital, Geneva (F.M.); the Biostatistics Department, Amgen, Cambridge (I.B.), and the Department of Clinical Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham (S.S.) - both in the United Kingdom; the Rare Diseases and Clinical Genetics Unit, Academic Pediatric Department, Bambino Gesù Children's Hospital (A.B.), and the Department of Molecular Medicine, Umberto I Hospital, Sapienza University of Rome (C.S.), Rome; the Lipid Clinic, Department of Medicine, Centre Hospitalier Universitaire de Québec-Université Laval, Quebec (J.B.), and the Clinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montréal, Chicoutimi, QC (D.G.) - both in Canada; the 2nd Department of Pediatrics, Semmelweis University, Budapest, Hungary (T.S.); the Department of Internal Medicine, Centres Hospitaliers Jolimont, La Louvière, Belgium (O.S.D.); and the Division of Pediatric Pulmonology, Allergology, and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna (S.G.-P.).

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Adolescent › Evolocumab in Pediatric Heterozygous Familial...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Globulines › Sérum-globulines › Immunoglobulines › Anticorps › Anticorps monoclonaux › Anticorps monoclonaux humanisés › Evolocumab in Pediatric Heterozygous Familial...
questionsmedicales.fr › Actions chimiques et utilisations › Actions pharmacologiques › Utilisations thérapeutiques › Agents régulateurs du métabolisme des lipides › Hypolipémiants › Anticholestérolémiants › Evolocumab in Pediatric Heterozygous Familial...
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Evolocumab in Pediatric Heterozygous Familial...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Lipoprotéines › Lipoprotéines LDL › Cholestérol LDL › Evolocumab in Pediatric Heterozygous Familial...
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questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Association de médicaments › Evolocumab in Pediatric Heterozygous Familial...
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questionsmedicales.fr › Actions chimiques et utilisations › Actions pharmacologiques › Utilisations thérapeutiques › Agents régulateurs du métabolisme des lipides › Hypolipémiants › Anticholestérolémiants › Inhibiteurs de l'hydroxyméthylglutaryl-CoA réductase › Evolocumab in Pediatric Heterozygous Familial...
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