Evidence map›Paper›PMID 35103306›Full record

ArticleDevelopmental medicine and child neurology2022

Evolution of bulbar function in spinal muscular atrophy type 1 treated with nusinersen.

Harriet Weststrate, Georgia Stimpson, Lily Thomas, Mariacristina Scoto, Emily Johnson, Alexandra Stewart, Francesco Muntoni, Giovanni Baranello, Eleanor Conway, SMA p-FOIS Working Group*

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

Article in Developmental medicine and child neurology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers, 6 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
29citing papers in PubMed, 6 pooled it
6.1field-weighted citation impact, top 3% 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

29 citing papers in PubMed, 6 syntheses or guidelines pooled it, 43 citations in OpenAlex.

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  14. Swallowing function in patients with spinal muscular atrophy before and after the introduction of new gene-based therapies: what has changed?Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025
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  18. Fiberoptic endoscopic evaluation of swallowing (FEES) in children with spinal muscular atrophy type 1: feasibility, swallowing safety and efficacy, and dysphagia phenotype.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2024
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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

10 authors at 3 institutions in 2 countries.

Harriet WeststrateThe Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute of Child Health, London, UK.ORCID 0000-0001-7575-4323
Georgia StimpsonThe Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute of Child Health, London, UK.
Lily ThomasDepartment of Language and Communication, University College London, London, UK.
Mariacristina ScotoThe Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute of Child Health, London, UK.
Emily JohnsonThe Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute of Child Health, London, UK.
Alexandra StewartDepartment of Language and Communication, University College London, London, UK.
Francesco MuntoniThe Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute of Child Health, London, UK.
Giovanni BaranelloThe Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute of Child Health, London, UK.
Eleanor ConwayThe Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, UCL Great Ormond Street Institute of Child Health, London, UK.
SMA p-FOIS Working Group*
Great Ormond Street Hospital · GBGreat Ormond Street Hospital for Children NHS Foundation Trust · GBLanguage Science (South Korea) · KR

Funding

Department of Health
6 · The paper itself

Abstract

aimTo assess the evolution of bulbar function in nusinersen-treated spinal muscular atrophy type 1 (SMA1).

methodThis single-centre retrospective study identified 24 patients (14 females and 10 males) with SMA1, treated with nusinersen between 2017 and 2020. We adapted and validated the Paediatric Functional Oral Intake Scale (p-FOIS), which is an outcome measure to assess bulbar function. Analysis considered SMA1 subtype, nutritional support, and Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) and p-FOIS scores at initiation of nusinersen treatment (baseline) and at 6, 12, and 24 months after initiation.

resultsThe median age at baseline was 11 months (range 1 month-7 years 6 months). Median age at initiation of tube feeding was 8 months (range 0-2 years 2 months). Fourteen patients were tube fed at baseline. The median p-FOIS score was 3 at baseline and 2 at 12 and 24 months. Four patients, all with type 1c SMA, remained orally fed at 24 months. Median CHOP INTEND scores increased from 32 at baseline to 42 at 12 and 24 months.

interpretationImpaired bulbar function persisted as a significant complication in most nusinersen-treated patients with SMA1, in contrast to the improvement in motor abilities demonstrated in the majority. p-FOIS allows for tracking of bulbar function progression and treatment response. Larger, prospective studies investigating the longer-term impacts of nusinersen on bulbar function are warranted.

Indexed as

Muscular Atrophy, SpinalSpinal Muscular Atrophies of ChildhoodChildFemaleHumansInfantInfant, NewbornMaleOligonucleotidesProspective StudiesRetrospective StudiesnusinersenOligonucleotides

Identifiers

PMID35103306
PMCPMC9306995
OpenAlexW4210671837

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.