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Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients

Approval of nusinersen, an intrathecally administered antisense oligonucleotide, for the treatment of 5q-spinal muscular atrophy (SMA) marked the beginning of a new therapeutic era in neurological diseases. Changes in routine cerebrospinal fluid (CSF) parameters under nusinersen have only recently b...

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Autores principales: Müschen, Lars Hendrik, Osmanovic, Alma, Binz, Camilla, Jendretzky, Konstantin F., Ranxha, Gresa, Bronzlik, Paul, Abu-Fares, Omar, Wiehler, Flavia, Möhn, Nora, Hümmert, Martin W., Gingele, Stefan, Götz, Friedrich, Stangel, Martin, Skripuletz, Thomas, Schreiber-Katz, Olivia, Petri, Susanne
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7996901/
https://www.ncbi.nlm.nih.gov/pubmed/33652830
http://dx.doi.org/10.3390/brainsci11030296
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author Müschen, Lars Hendrik
Osmanovic, Alma
Binz, Camilla
Jendretzky, Konstantin F.
Ranxha, Gresa
Bronzlik, Paul
Abu-Fares, Omar
Wiehler, Flavia
Möhn, Nora
Hümmert, Martin W.
Gingele, Stefan
Götz, Friedrich
Stangel, Martin
Skripuletz, Thomas
Schreiber-Katz, Olivia
Petri, Susanne
author_facet Müschen, Lars Hendrik
Osmanovic, Alma
Binz, Camilla
Jendretzky, Konstantin F.
Ranxha, Gresa
Bronzlik, Paul
Abu-Fares, Omar
Wiehler, Flavia
Möhn, Nora
Hümmert, Martin W.
Gingele, Stefan
Götz, Friedrich
Stangel, Martin
Skripuletz, Thomas
Schreiber-Katz, Olivia
Petri, Susanne
author_sort Müschen, Lars Hendrik
collection PubMed
description Approval of nusinersen, an intrathecally administered antisense oligonucleotide, for the treatment of 5q-spinal muscular atrophy (SMA) marked the beginning of a new therapeutic era in neurological diseases. Changes in routine cerebrospinal fluid (CSF) parameters under nusinersen have only recently been described in adult SMA patients. We aimed to explore these findings in a real-world setting and to identify clinical and procedure-associated features that might impact CSF parameters. Routinely collected CSF parameters (leukocyte count, lactate, total protein, CSF/serum albumin quotient (QAlbumin), oligoclonal bands) of 28 adult SMA patients were examined for up to 22 months of nusinersen treatment. Total protein and QAlbumin values significantly increased in the first 10 months, independent of the administration procedure. By month 14, no further increases were detected. Two patients developed transient pleocytosis. In two cases, positive oligoclonal bands were found in the beginning and in four patients throughout the whole observation period. No clinical signs of inflammatory central nervous system disease were apparent. Our data confirm elevated CSF total protein and QAlbumin during nusinersen treatment. These alterations may be caused by both repeated lumbar punctures and the interval between procedures rather than by the medication itself. Generally, there were no severe alterations of CSF routine parameters. These results further underline the safety of nusinersen therapy.
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spelling pubmed-79969012021-03-27 Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients Müschen, Lars Hendrik Osmanovic, Alma Binz, Camilla Jendretzky, Konstantin F. Ranxha, Gresa Bronzlik, Paul Abu-Fares, Omar Wiehler, Flavia Möhn, Nora Hümmert, Martin W. Gingele, Stefan Götz, Friedrich Stangel, Martin Skripuletz, Thomas Schreiber-Katz, Olivia Petri, Susanne Brain Sci Article Approval of nusinersen, an intrathecally administered antisense oligonucleotide, for the treatment of 5q-spinal muscular atrophy (SMA) marked the beginning of a new therapeutic era in neurological diseases. Changes in routine cerebrospinal fluid (CSF) parameters under nusinersen have only recently been described in adult SMA patients. We aimed to explore these findings in a real-world setting and to identify clinical and procedure-associated features that might impact CSF parameters. Routinely collected CSF parameters (leukocyte count, lactate, total protein, CSF/serum albumin quotient (QAlbumin), oligoclonal bands) of 28 adult SMA patients were examined for up to 22 months of nusinersen treatment. Total protein and QAlbumin values significantly increased in the first 10 months, independent of the administration procedure. By month 14, no further increases were detected. Two patients developed transient pleocytosis. In two cases, positive oligoclonal bands were found in the beginning and in four patients throughout the whole observation period. No clinical signs of inflammatory central nervous system disease were apparent. Our data confirm elevated CSF total protein and QAlbumin during nusinersen treatment. These alterations may be caused by both repeated lumbar punctures and the interval between procedures rather than by the medication itself. Generally, there were no severe alterations of CSF routine parameters. These results further underline the safety of nusinersen therapy. MDPI 2021-02-26 /pmc/articles/PMC7996901/ /pubmed/33652830 http://dx.doi.org/10.3390/brainsci11030296 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) ).
spellingShingle Article
Müschen, Lars Hendrik
Osmanovic, Alma
Binz, Camilla
Jendretzky, Konstantin F.
Ranxha, Gresa
Bronzlik, Paul
Abu-Fares, Omar
Wiehler, Flavia
Möhn, Nora
Hümmert, Martin W.
Gingele, Stefan
Götz, Friedrich
Stangel, Martin
Skripuletz, Thomas
Schreiber-Katz, Olivia
Petri, Susanne
Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients
title Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients
title_full Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients
title_fullStr Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients
title_full_unstemmed Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients
title_short Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients
title_sort cerebrospinal fluid parameters in antisense oligonucleotide-treated adult 5q-spinal muscular atrophy patients
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7996901/
https://www.ncbi.nlm.nih.gov/pubmed/33652830
http://dx.doi.org/10.3390/brainsci11030296
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