Evidence map›Paper›PMID 37280513›Full record

ArticleBMC neurology2023

Cognitive performance of adult patients with SMA before and after treatment initiation with nusinersen.

Maximilian Vidovic, Maren Freigang, Elisa Aust, Katharina Linse, Daniel Petzold, René Günther

Erratum issuedOpen access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in BMC neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 7 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed, 1 pooled it
2.3field-weighted citation impact, top 12% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 1 synthesis or guideline pooled it, 8 citations in OpenAlex.

  1. Pooled it
  2. Review
  3. Article
  4. Exploring variability in cognitive functioning in patients with spinal muscular atrophy: a scoping review.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024
    Article
  5. Article
  6. Review
  7. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

6 authors at 3 institutions in 1 country.

Maximilian VidovicDepartment of Neurology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Maren FreigangDepartment of Neurology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Elisa AustDepartment of Neurology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Katharina LinseDepartment of Neurology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Daniel PetzoldDepartment of Neurology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
René GüntherDepartment of Neurology, University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany. rene.guenther@uniklinikum-dresden.de.
University Hospital Carl Gustav Carus · DEGerman Center for Neurodegenerative Diseases · DETU Dresden · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundSpinal muscular atrophy (SMA) is a genetic neuromuscular disease caused by mutations of the SMN1 gene. Deficient SMN protein causes irreversible degeneration of alpha motor neurons characterized by progressive muscle weakness and atrophy. Considering that SMA is a multi-systemic disorder and SMN protein was found to be expressed in cortical structures, the cognitive profile of adult patients with SMA has recently been of particular interest. With nusinersen, a novel, disease-modifying drug has been established, but its effects on neuropsychological functions have not been validated yet. Aim of this study was to investigate the cognitive profile of adult patients with SMA during treatment initiation with nusinersen and to reveal improvement or deterioration in cognitive performance.

methodsThis monocentric longitudinal study included 23 patients with SMA type 2 and 3. All patients were assessed with the Edinburgh Cognitive and Behavioral ALS Screen (ECAS) before and after 14 months of treatment initiation with nusinersen. Additionally, motor function was evaluated by Hammersmith Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module (RULM) and Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R).

resultsOf the treatment-naive patients, only three were below the age- and education-matched cut-off for cognitive impairment in the ECAS total score. Significant differences between SMA type 2 and 3 were only detected in the domain of Language. After 14 months of treatment, patients showed significant improvement of absolute scores in all three ALS-specific domains, in the non-ALS-specific domain of Memory, in both subscores and in the ECAS total score. No associations were detected between cognitive and functional outcome measures.

conclusionsIn some adult patients with SMA abnormal cognitive performance in ALS-specific functions of the ECAS was evident. However, the presented results suggest no clinically significant cognitive changes during the observed treatment period with nusinersen.

Indexed as

Muscular Atrophy, SpinalSpinal Muscular Atrophies of ChildhoodAdultCognitionHumansLongitudinal StudiesOligonucleotidesnusinersenOligonucleotidesCognitionCognitive performanceEdinburgh cognitive and behavioral ALS screen (ECAS)NusinersenSpinal muscular atrophy (SMA)

Identifiers

PMID37280513
PMCPMC10243018
OpenAlexW4379536004

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.