Longitudinal Assessment of Creatine Kinase, Creatine/Creatinine

Humans Muscular Dystrophy, Duchenne / diagnosis Dystrophin Creatine Creatinine Myostatin Prospective Studies Biomarkers Creatine Kinase Disease Progression

Journal

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

Informations de publication

Date de publication:
28 02 2023
Historique:
received: 26 01 2022
accepted: 11 10 2022
entrez: 27 2 2023
pubmed: 28 2 2023
medline: 3 3 2023
Statut: ppublish

Résumé

The slow and variable disease progression of Becker muscular dystrophy (BMD) urges the development of biomarkers to facilitate clinical trials. We explored changes in 3 muscle-enriched biomarkers in serum of patients with BMD over 4-year time and studied associations with disease severity, disease progression, and dystrophin levels in BMD. We quantitatively measured creatine kinase (CK) using the International Federation of Clinical Chemistry reference method, creatine/creatinine Thirty-four patients with 106 visits were included. Eight patients were nonambulant at baseline. Cr/Crn and myostatin were highly patient specific (intraclass correlation coefficient for both = 0.960). Cr/Crn was strongly negatively correlated, whereas myostatin was strongly positively correlated with the NSAA, TMRv, and 6MWT (Cr/Crn rho = -0.869 to -0.801 and myostatin rho = 0.792 to 0.842, all Both Cr/Crn and myostatin could potentially serve as monitoring biomarkers in BMD, as higher Cr/Crn and lower myostatin were associated with lower motor performance and predictive of concurrent functional performance when combined with age. Future studies are needed to more precisely determine the context of use of these biomarkers.

Sections du résumé

BACKGROUND AND OBJECTIVES
The slow and variable disease progression of Becker muscular dystrophy (BMD) urges the development of biomarkers to facilitate clinical trials. We explored changes in 3 muscle-enriched biomarkers in serum of patients with BMD over 4-year time and studied associations with disease severity, disease progression, and dystrophin levels in BMD.
METHODS
We quantitatively measured creatine kinase (CK) using the International Federation of Clinical Chemistry reference method, creatine/creatinine
RESULTS
Thirty-four patients with 106 visits were included. Eight patients were nonambulant at baseline. Cr/Crn and myostatin were highly patient specific (intraclass correlation coefficient for both = 0.960). Cr/Crn was strongly negatively correlated, whereas myostatin was strongly positively correlated with the NSAA, TMRv, and 6MWT (Cr/Crn rho = -0.869 to -0.801 and myostatin rho = 0.792 to 0.842, all
DISCUSSION
Both Cr/Crn and myostatin could potentially serve as monitoring biomarkers in BMD, as higher Cr/Crn and lower myostatin were associated with lower motor performance and predictive of concurrent functional performance when combined with age. Future studies are needed to more precisely determine the context of use of these biomarkers.

Identifiants

DOI: 10.1212/WNL.0000000000201609 PMID: 36849458 PMC: PMC9990441
pubmed: 36849458
pii: WNL.0000000000201609
doi: 10.1212/WNL.0000000000201609
pmc: PMC9990441
doi:

Substances chimiques

Dystrophin 0
Creatine MU72812GK0
Creatinine AYI8EX34EU
Myostatin 0
Biomarkers 0
Creatine Kinase EC 2.7.3.2

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

e975-e984

Informations de copyright

Copyright © 2022 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.

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Auteurs

Nienke M van de Velde (NM)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.
ORCID: 0000-0002-1541-130X

Zaïda Koeks (Z)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.

Mirko Signorelli (M)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.
ORCID: 0000-0002-8102-3356

Nisha Verwey (N)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.

Maurice Overzier (M)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.
ORCID: 0000-0002-8143-4884

Jaap A Bakker (JA)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.
ORCID: 0000-0002-2918-7361

Gautam Sajeev (G)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.
ORCID: 0000-0002-1827-8938

James Signorovitch (J)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.

Valeria Ricotti (V)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.
ORCID: 0000-0003-1800-1987

Jan Verschuuren (J)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.
ORCID: 0000-0002-4572-1501

Kristy Brown (K)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.
ORCID: 0000-0002-1047-4328

Pietro Spitali (P)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom.
ORCID: 0000-0003-2783-688X

Erik H Niks (EH)

From the Departments of Neurology (N.M.V., Z.K., J.V., E.H.N.), Biomedical Data Sciences (M.S.), Human Genetics (N.V., M.O., P.S.), and Clinical Chemistry and Laboratory Medicine (J.A.B.), Leiden University Medical Center, the Netherlands; Duchenne Center Netherlands (N.M.V., J.V., P.S., E.H.N.); European Reference Network for Rare Neuromuscular Diseases [ERN EURO-NMD] (N.M.V., Z.K., N.V., M.O., J.V., P.S., E.H.N.); Mathematical Institute (M.S.), Leiden University, the Netherlands; Analysis Group Inc (G.S., J.S.), Boston, MA; Solid Biosciences Inc (V.R., K.B.), Cambridge, MA; and NIHR Great Ormond Street Hospital Biomedical Research Centre (V.R.), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, United Kingdom. e.h.niks@lumc.nl.
ORCID: 0000-0001-5892-5143

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Longitudinal Assessment of Creatine Kinase,...
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Dystrophies musculaires › Myopathie de Duchenne › Longitudinal Assessment of Creatine Kinase,...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines membranaires › Dystrophine › Longitudinal Assessment of Creatine Kinase,...
questionsmedicales.fr › Acides aminés, peptides et protéines › Acides aminés › Créatine › Longitudinal Assessment of Creatine Kinase,...
questionsmedicales.fr › Composés hétérocycliques › Composés hétéromonocycliques › Azoles › Imidazoles › Créatinine › Longitudinal Assessment of Creatine Kinase,...
questionsmedicales.fr › Facteurs biologiques › Protéines et peptides de signalisation intercellulaire › Protéines de la superfamille du TGF bêta › Facteurs de croissance et de différenciation › Myostatine › Longitudinal Assessment of Creatine Kinase,...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études épidémiologiques › Études de cohortes › Études prospectives › Longitudinal Assessment of Creatine Kinase,...
questionsmedicales.fr › Facteurs biologiques › Marqueurs biologiques › Longitudinal Assessment of Creatine Kinase,...
questionsmedicales.fr › Enzymes et coenzymes › Enzymes › Transferases › Phosphotransferases › Phosphotransferases (Nitrogenous Group Acceptor) › Creatine kinase › Longitudinal Assessment of Creatine Kinase,...
questionsmedicales.fr › États, signes et symptômes pathologiques › Processus pathologiques › Caractéristiques de la maladie › Évolution de la maladie › Longitudinal Assessment of Creatine Kinase,...