Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment
Abstract Objective To determine whether serum creatine kinase activity (CK) and serum creatinine concentration (Crn) are prognostic and predictive biomarkers for disease severity, disease progression, and nusinersen treatment effects in adult patients with 5q‐associated spinal muscular atrophy (SMA)...
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2021-05-01
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Series: | Annals of Clinical and Translational Neurology |
Online Access: | https://doi.org/10.1002/acn3.51340 |
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doaj-b45a627a87f940ddbcd346930af78a592021-05-10T11:31:14ZengWileyAnnals of Clinical and Translational Neurology2328-95032021-05-01851049106310.1002/acn3.51340Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatmentMaren Freigang0Claudia D. Wurster1Tim Hagenacker2Benjamin Stolte3Markus Weiler4Christoph Kamm5Olivia Schreiber‐Katz6Alma Osmanovic7Susanne Petri8Alexander Kowski9Thomas Meyer10Jan C. Koch11Isabell Cordts12Marcus Deschauer13Paul Lingor14Elisa Aust15Daniel Petzold16Albert C. Ludolph17Björn Falkenburger18Andreas Hermann19René Günther20Department of Neurology Universitätsklinikum Carl Gustav CarusTechnische Universität Dresden Dresden GermanyDepartment of Neurology Ulm University Ulm GermanyDepartment of Neurology University Hospital Essen Essen GermanyDepartment of Neurology University Hospital Essen Essen GermanyDepartment of Neurology Heidelberg University Hospital Heidelberg GermanyDepartment of Neurology University of Rostock Rostock GermanyDepartment of Neurology Hannover Medical School Hannover GermanyDepartment of Neurology Hannover Medical School Hannover GermanyDepartment of Neurology Hannover Medical School Hannover GermanyDepartment of Neurology Charité – Universitätsmedizin Berlin Berlin GermanyDepartment of Neurology Charité – Universitätsmedizin Berlin Berlin GermanyDepartment of Neurology University Medicine Göttingen Göttingen GermanyDepartment of Neurology Klinikum Rechts der IsarTechnical University of Munich Munich GermanyDepartment of Neurology Klinikum Rechts der IsarTechnical University of Munich Munich GermanyDepartment of Neurology Klinikum Rechts der IsarTechnical University of Munich Munich GermanyDepartment of Neurology Universitätsklinikum Carl Gustav CarusTechnische Universität Dresden Dresden GermanyDepartment of Neurology Universitätsklinikum Carl Gustav CarusTechnische Universität Dresden Dresden GermanyDepartment of Neurology Ulm University Ulm GermanyDepartment of Neurology Universitätsklinikum Carl Gustav CarusTechnische Universität Dresden Dresden GermanyDepartment of Neurology Translational Neurodegeneration Section “Albrecht‐Kossel”University Medical Center RostockUniversity of Rostock Rostock GermanyDepartment of Neurology Universitätsklinikum Carl Gustav CarusTechnische Universität Dresden Dresden GermanyAbstract Objective To determine whether serum creatine kinase activity (CK) and serum creatinine concentration (Crn) are prognostic and predictive biomarkers for disease severity, disease progression, and nusinersen treatment effects in adult patients with 5q‐associated spinal muscular atrophy (SMA). Methods Within this retrospective, multicenter observational study in 206 adult patients with SMA, we determined clinical subtypes (SMA types, ambulatory ability) and repeatedly measured CK and Crn and examined disease severity scores (Hammersmith Functional Motor Scale Expanded, Revised Upper Limb Module, and revised Amyotrophic Lateral Sclerosis Functional Rating Scale). Patients were followed under nusinersen treatment for 18 months. Results CK and Crn differed between clinical subtypes and correlated strongly with disease severity scores (e.g., for Hammersmith Functional Motor Scale Expanded: (CK) ρ = 0.786/ (Crn) ρ = 0.558). During the 18 months of nusinersen treatment, CK decreased (∆CK = −17.56%, p < 0.0001), whereas Crn slightly increased (∆Crn = +4.75%, p < 0.05). Interpretation Serum creatine kinase activity and serum creatinine concentration reflect disease severity of spinal muscular atrophy and are promising biomarkers to assess patients with spinal muscular atrophy during disease course and to predict treatment response. The decrease of creatine kinase activity, combined with the tendency of creatinine concentration to increase during nusinersen treatment, suggests reduced muscle mass wasting with improved muscle energy metabolism.https://doi.org/10.1002/acn3.51340 |
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DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Maren Freigang Claudia D. Wurster Tim Hagenacker Benjamin Stolte Markus Weiler Christoph Kamm Olivia Schreiber‐Katz Alma Osmanovic Susanne Petri Alexander Kowski Thomas Meyer Jan C. Koch Isabell Cordts Marcus Deschauer Paul Lingor Elisa Aust Daniel Petzold Albert C. Ludolph Björn Falkenburger Andreas Hermann René Günther |
spellingShingle |
Maren Freigang Claudia D. Wurster Tim Hagenacker Benjamin Stolte Markus Weiler Christoph Kamm Olivia Schreiber‐Katz Alma Osmanovic Susanne Petri Alexander Kowski Thomas Meyer Jan C. Koch Isabell Cordts Marcus Deschauer Paul Lingor Elisa Aust Daniel Petzold Albert C. Ludolph Björn Falkenburger Andreas Hermann René Günther Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment Annals of Clinical and Translational Neurology |
author_facet |
Maren Freigang Claudia D. Wurster Tim Hagenacker Benjamin Stolte Markus Weiler Christoph Kamm Olivia Schreiber‐Katz Alma Osmanovic Susanne Petri Alexander Kowski Thomas Meyer Jan C. Koch Isabell Cordts Marcus Deschauer Paul Lingor Elisa Aust Daniel Petzold Albert C. Ludolph Björn Falkenburger Andreas Hermann René Günther |
author_sort |
Maren Freigang |
title |
Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment |
title_short |
Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment |
title_full |
Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment |
title_fullStr |
Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment |
title_full_unstemmed |
Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment |
title_sort |
serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment |
publisher |
Wiley |
series |
Annals of Clinical and Translational Neurology |
issn |
2328-9503 |
publishDate |
2021-05-01 |
description |
Abstract Objective To determine whether serum creatine kinase activity (CK) and serum creatinine concentration (Crn) are prognostic and predictive biomarkers for disease severity, disease progression, and nusinersen treatment effects in adult patients with 5q‐associated spinal muscular atrophy (SMA). Methods Within this retrospective, multicenter observational study in 206 adult patients with SMA, we determined clinical subtypes (SMA types, ambulatory ability) and repeatedly measured CK and Crn and examined disease severity scores (Hammersmith Functional Motor Scale Expanded, Revised Upper Limb Module, and revised Amyotrophic Lateral Sclerosis Functional Rating Scale). Patients were followed under nusinersen treatment for 18 months. Results CK and Crn differed between clinical subtypes and correlated strongly with disease severity scores (e.g., for Hammersmith Functional Motor Scale Expanded: (CK) ρ = 0.786/ (Crn) ρ = 0.558). During the 18 months of nusinersen treatment, CK decreased (∆CK = −17.56%, p < 0.0001), whereas Crn slightly increased (∆Crn = +4.75%, p < 0.05). Interpretation Serum creatine kinase activity and serum creatinine concentration reflect disease severity of spinal muscular atrophy and are promising biomarkers to assess patients with spinal muscular atrophy during disease course and to predict treatment response. The decrease of creatine kinase activity, combined with the tendency of creatinine concentration to increase during nusinersen treatment, suggests reduced muscle mass wasting with improved muscle energy metabolism. |
url |
https://doi.org/10.1002/acn3.51340 |
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