Neurofilament Light Chain as a Biomarker of Hereditary Transthyretin-Mediated Amyloidosis.

Aged Amyloid Neuropathies, Familial / blood Biomarkers / blood Case-Control Studies Female Humans Male Middle Aged Neurofilament Proteins / blood Prognosis Proteome RNA, Small Interfering / therapeutic use

Journal

Neurology
ISSN: 1526-632X
Titre abrégé: Neurology
Pays: United States
ID NLM: 0401060

Informations de publication

Date de publication:
19 01 2021
Historique:
received: 09 04 2020
accepted: 01 09 2020
pubmed: 23 10 2020
medline: 2 2 2021
entrez: 22 10 2020
Statut: ppublish

Résumé

To identify changes in the proteome associated with onset and progression of hereditary transthyretin-mediated (hATTR) amyloidosis, also known as ATTRv amyloidosis, we performed an observational, case-controlled study that compared proteomes of patients with ATTRv amyloidosis and healthy controls. Plasma levels of >1,000 proteins were measured in patients with ATTRv amyloidosis with polyneuropathy who received either placebo or patisiran in a Phase 3 study of patisiran (APOLLO), and in healthy controls. The effect of patisiran on the time profile of each protein was determined by linear mixed model at 0, 9, and 18 months. Neurofilament light chain (NfL) was further assessed with an orthogonal quantitative approach. Levels of 66 proteins were significantly changed with patisiran vs placebo, with NfL change most significant ( Findings suggest that NfL may serve as a biomarker of nerve damage and polyneuropathy in ATTRv amyloidosis, enable earlier diagnosis of patients with ATTRv amyloidosis, and facilitate monitoring of disease progression. This study provides Class III evidence that NfL levels may enable earlier diagnosis of polyneuropathy in patients with ATTRv amyloidosis and facilitate monitoring of disease progression.

Identifiants

DOI: 10.1212/WNL.0000000000011090 PMID: 33087494 PMC: PMC7884985
pubmed: 33087494
pii: WNL.0000000000011090
doi: 10.1212/WNL.0000000000011090
pmc: PMC7884985
doi:

Substances chimiques

Biomarkers 0
Neurofilament Proteins 0
Proteome 0
RNA, Small Interfering 0
neurofilament protein L 0
patisiran 50FKX8CB2Y

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

e412-e422

Informations de copyright

Copyright © 2020 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.

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Auteurs

Simina Ticau (S)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

Gautham V Sridharan (GV)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

Shira Tsour (S)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

William L Cantley (WL)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

Amy Chan (A)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

Jason A Gilbert (JA)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

David Erbe (D)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

Emre Aldinc (E)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

Mary M Reilly (MM)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

David Adams (D)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

Michael Polydefkis (M)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

Kevin Fitzgerald (K)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

Akshay Vaishnaw (A)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD.

Paul Nioi (P)

From Alnylam Pharmaceuticals (S.T., G.V.S., S.T., W.L.C., A.C., J.A.G., D.E., E.A., K.F., A.V., P.N.) , Cambridge, MA; MRC Centre for Neuromuscular Diseases (M.M.R.), UCL Queen Square Institute of Neurology, London, UK; AP-HP (D.A.), Université Paris-Saclay, CHU Bicêtre, INSERM U1195, Le Kremlin Bicêtre, France; and Department of Neurology (M.P.), Johns Hopkins University School of Medicine, Baltimore, MD. pnioi@alnylam.com.

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Sujet âgé › Neurofilament Light Chain as a Biomarker of...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Troubles de l'homéostasie des protéines › Amyloïdose › Amyloïdose familiale › Neuropathies amyloïdes familiales › Neurofilament Light Chain as a Biomarker of...
questionsmedicales.fr › Facteurs biologiques › Marqueurs biologiques › Neurofilament Light Chain as a Biomarker of...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études épidémiologiques › Études cas-témoins › Neurofilament Light Chain as a Biomarker of...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Neurofilament Light Chain as a Biomarker of...
questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Adulte d'âge moyen › Neurofilament Light Chain as a Biomarker of...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines de tissu nerveux › Protéines neurofilamenteuses › Neurofilament Light Chain as a Biomarker of...
questionsmedicales.fr › Diagnostic › Pronostic › Neurofilament Light Chain as a Biomarker of...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéome › Neurofilament Light Chain as a Biomarker of...
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Acides nucléiques › ARN › ARN non traduit › Petit ARN non traduit › Petit ARN interférent › Neurofilament Light Chain as a Biomarker of...