Abstract

5q-associated spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS) are two distinct neurological disorders leading to degeneration of lower motor neurons. The antisense oligonucleotides (ASOs) nusinersen and tofersen are novel disease-modifying agents for these diseases, respectively. In the context of ASO treatment, the cytological characteristics and composition of cerebrospinal fluid (CSF) have recently garnered particular interest. This report presents a case series of CSF cytology findings in two patients with SMA and ALS revealing comparable unspecified macrophage inclusions following treatment initiation with nusinersen and tofersen. Yet, the presence of these “asophages” in the treatment course of two different ASOs is of unclear significance. While both treatments have been well tolerated, this phenomenon warrants attention, given the long-term nature of these treatments.

Details

Title
Macrophage inclusions in cerebrospinal fluid following treatment initiation with antisense oligonucleotide therapies in motor neuron diseases
Author
Vidovic, Maximilian 1 ; Menschikowski, Mario 2 ; Freigang, Maren 1 ; Lapp, Hanna Sophie 1 ; Günther, René 3 

 University Hospital Carl Gustav Carus, Technische Universität Dresden, Department of Neurology, Dresden, Germany (GRID:grid.4488.0) (ISNI:0000 0001 2111 7257) 
 University Hospital Carl Gustav Carus, Technische Universität Dresden, Institute of Clinical Chemistry and Laboratory Medicine, Dresden, Germany (GRID:grid.4488.0) (ISNI:0000 0001 2111 7257) 
 University Hospital Carl Gustav Carus, Technische Universität Dresden, Department of Neurology, Dresden, Germany (GRID:grid.4488.0) (ISNI:0000 0001 2111 7257); German Center for Neurodegenerative Diseases (DZNE) Dresden, Dresden, Germany (GRID:grid.424247.3) (ISNI:0000 0004 0438 0426) 
Pages
11
Publication year
2024
Publication date
2024
Publisher
Springer Nature B.V.
e-ISSN
25243489
Source type
Scholarly Journal
Language of publication
English
ProQuest document ID
2929321126
Copyright
© The Author(s) 2024. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.