Evidence map›Paper›PMID 33106369›Full record

ArticleJournal of neurology, neurosurgery, and psychiatry2020

Motor unit changes in children with symptomatic spinal muscular atrophy treated with nusinersen.

Didu Kariyawasam, Arlene D'Silva, James Howells, Karen Herbert, Peter Geelan-Small, Cindy Shin-Yi Lin, Michelle Anne Farrar

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of neurology, neurosurgery, and psychiatry, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
36citing papers in PubMed, 2 pooled it
4.4field-weighted citation impact, top 5% 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

36 citing papers in PubMed, 2 syntheses or guidelines pooled it, 64 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors at 3 institutions in 1 country.

Didu KariyawasamNeurology, Sydney Children's Hospital Randwick, Randwick, New South Wales, Australia Didu.Kariyawasam@health.nsw.gov.au.ORCID http://orcid.org/0000-0002-4176-4010
Arlene D'SilvaSchool of Women's and Children's Health, UNSW Medicine, University of New South Wales, Sydney, New South Wales, Australia.
James HowellsCentral Clinical School, Faculty of Medicine and Health, Brain and Mind Centre, The University of Sydney, Sydney, New South Wales, Australia.
Karen HerbertPhysiotherapy, Syndey Children's Hospital, Randwick, Sydney, New South Wales, Australia.
Peter Geelan-SmallMark Wainwright Analytical Centre, University of New South Wales, Sydney, New South Wales, Australia.
Cindy Shin-Yi LinCentral Clinical School, Faculty of Medicine and Health, Brain and Mind Centre, The University of Sydney, Sydney, New South Wales, Australia.
Michelle Anne FarrarNeurology, Sydney Children's Hospital Randwick, Randwick, New South Wales, Australia.ORCID http://orcid.org/0000-0002-4472-0902
UNSW Sydney · AUThe University of Sydney · AUSydney Children's Hospital · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesTo elucidate the motor unit response to intrathecal nusinersen in children with symptomatic spinal muscular atrophy (SMA) using a novel motor unit number estimation technique.

methodsMScanFit MUNE studies were sequentially undertaken from the abductor pollicis brevis muscle after stimulation of the median nerve in a prospective cohort of symptomatic children with SMA, undergoing intrathecal treatment with nusinersen at a single neuromuscular centre from June 2017 to August 2019. Electrophysiological measures included compound muscle action potential (CMAP), motor unit number estimation (MUNE), motor unit number contributing to 50%-100% of CMAP (N50) and measures of collateral reinnervation including largest single motor unit potential (LSMUP) and amplitude of the smallest unit contributing to N50 (A50).

resultsTwenty children (median age 99 months, range 4-193) were followed for a median of 13.8 (4-33.5) months. Therapeutic intervention was an independent and significant contributor to an increase in CMAP (p = 0.005), MUNE (p = 0.001) and N50 (p = 0.04). The magnitude of this electrophysiological response was increased in children with shorter disease durations (p<0.05). Electrophysiological changes delineated children who were functionally stable from those who attained clinically significant gains in motor function.

interpretationNusinersen therapy facilitated functional innervation in SMA through recovery of smaller motor units. Delineation of biomechanisms of therapeutic response may be the first step in identifying potential novel targets for disease modification in this and other motor neuropathies. MScanFit MUNE techniques may have a broader role in establishing biomarkers of therapeutic response in similar adult-onset diseases.

Identifiers

PMID33106369
PMCPMC7803907
OpenAlexW3094366038

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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Registered trials

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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.