Evidence map›Paper›PMID 37212069›Full record

ArticleJournal of neuromuscular diseases2023

Development of an International SMA Bulbar Assessment for Inter-professional Administration.

Sally Dunaway Young, Katlyn McGrattan, Emily Johnson, Marise van der Heul, Tina Duong, Merete Bakke, Ulla Werlauff, Amy Pasternak, Camilla Cattaneo, Katie Hoffman and 7 more

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

Article in Journal of neuromuscular diseases, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 2 pooled it
3.2field-weighted citation impact, top 8% 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

9 citing papers in PubMed, 2 syntheses or guidelines pooled it, 11 citations in OpenAlex.

  1. Pooled it
  2. Guideline
  3. Article
  4. Article
  5. Observational
  6. Article
  7. Article
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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

17 authors at 14 institutions in 5 countries.

Sally Dunaway YoungDepartment of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA, USA.
Katlyn McGrattanDepartment of Speech-Language-Hearing Sciences, University of Minnesota, Minneapolis, MN, USA.
Emily JohnsonDepartment of Speech and Language Therapy, Great Ormond Street Hospital, London, UK.
Marise van der HeulDepartment of Neurology, University Medical Center Utrecht, Brain Center UMC Utrecht, Department of Neurology & Neurosurgery, Spieren voor Spieren Kindercentrum, Utrecht, the Netherlands.
Tina DuongDepartment of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA, USA.
Merete BakkeDepartment of Odontology, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark.
Ulla WerlauffNational Rehabilitation Centre for Neuromuscular Diseases, Aarhus, Denmark.
Amy PasternakDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Camilla CattaneoCentro Clinico Nemo, Fondazione Serena Onlus, Milan, Italy.
Katie HoffmanAnn and Robert H. Lurie Children's Hospital, Chicago, IL, USA.
Lavinia FanelliCentro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Anne BreaksAcute Speech and Language Therapy Service, Evelina London Children's Hospital, London, UK.
Kristen AllisonDepartment of Communication Sciences & Disorders, Northeastern University, Boston, MA, USA.
Giovanni BaranelloDubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Foundation Trust, London.
Richard FinkelCenter for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.
Giorgia CorattiPediatric Neurology, Catholic University of Sacred Heart, Rome, Italy.
Robert Muni LofraThe John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Great Ormond Street Hospital · GBStanford University · USAgostino Gemelli University Polyclinic · ITBoston Children's Hospital · USCentro Clinico Nemo · ITEvelina London Children's Healthcare · GBLurie Children's Hospital · USNewcastle upon Tyne Hospitals NHS Foundation Trust · GBNortheastern University · USSt. Jude Children's Research Hospital · USUniversità Cattolica del Sacro Cuore · ITUniversity Medical Center Utrecht · NLUniversity of Copenhagen · DKUniversity of Minnesota · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundProgressive weakness can affect bulbar muscles in individuals with moderate to severe forms of spinal muscular atrophy (SMA). The paucity of standardized, valid bulbar assessments capturing clinically significant deficits in SMA impedes the ability to monitor function, facilitate intervention, or detect treatment response.

objectiveTo fill this void, an international multidisciplinary team gathered to develop an agreed upon consensus-derived assessment of bulbar function in SMA for inter-professional administration to enhance our ability to monitor disease progression, support clinical management, and evaluate treatment effects.

methodsFifty-six international clinicians experienced in SMA were invited and engaged using the Delphi method over multiple rounds of web-based surveys to establish consensus.

resultsSerial virtual meetings occurred with 42 clinicians (21 speech and language therapists, 11 physical therapists, 5 neurologists, 4 occupational therapists, and 1 dentist). Seventy-two validated assessments of bulbar function were identified for potential relevance to individuals with SMA (32 accessible objective, 11 inaccessible objective, 29 patient-reported outcomes). Delphi survey rounds (n = 11, 15, 15) achieved consensus on individual items with relevance and wording discussed. Key aspects of bulbar function identified included: oral intake status, oral facial structure and motor strength, swallowing physiology, voice & speech, and fatigability.

conclusionsMultidisciplinary clinicians with expertise in bulbar function and SMA used Delphi methodology to reach consensus on assessments/items considered relevant for SMA across all age groups. Future steps include piloting the new scale moving towards validation/reliability. This work supports the advancement of assessing bulbar function in children and adults with SMA by a variety of professionals.

Indexed as

Muscular Atrophy, SpinalAdultChildDeglutitionFatigueHumansReproducibility of ResultsSurveys and Questionnairesbulbar functiondeglutitionoutcome measuresspeechSpinal muscular atrophyswallowingvoice

Identifiers

PMID37212069
PMCPMC10357133
OpenAlexW4377091776

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.