Evidence map›Paper›PMID 36776075›Full record

ArticleJournal of neuromuscular diseases2023

Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes.

Sally Dunaway Young, Amy Pasternak, Tina Duong, Katlyn E McGrattan, Sarah Stranberg, Elizabeth Maczek, Courtney Dias, Whitney Tang, Dana Parker, Alexis Levine and 6 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 13 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed, 2 pooled it
4.6field-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

13 citing papers in PubMed, 2 syntheses or guidelines pooled it, 16 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
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  7. Observational
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  12. 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
    Review
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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

16 authors at 5 institutions in 1 country.

Sally Dunaway YoungDepartment of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA, USA.
Amy PasternakDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Tina DuongDepartment of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA, USA.
Katlyn E McGrattanDepartment of Speech-Language-Hearing Sciences, University of Minnesota, Minneapolis, MN, USA.
Sarah StranbergOutpatient Neurologic Rehabilitation Program, Stanford Health Care, Palo Alto, CA, USA.
Elizabeth MaczekDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Courtney DiasDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Whitney TangDepartment of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA, USA.
Dana ParkerDepartment of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA, USA.
Alexis LevineDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Alyssa RohanDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Connie WolfordDepartment of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA, USA.
William MartensDepartment of Neurology, University of Rochester, Rochester, NY, USA.
Michael P McDermottDepartment of Neurology, University of Rochester, Rochester, NY, USA.
Basil T DarrasDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
John W DayDepartment of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, CA, USA.
Boston Children's Hospital · USStanford University · USUniversity of Rochester · USStanford Health Care · USUniversity of Minnesota · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundNovel Spinal Muscular Atrophy (SMA) treatments have demonstrated improvements on motor measures that are clearly distinct from the natural history of progressive decline. Comparable measures are needed to monitor bulbar function, which is affected in severe SMA.

objectiveTo assess bulbar function with patient-reported outcome measures (PROs) and determine their relationships with clinical characteristics.

methodsWe recruited 47 non-ambulatory participants (mean (SD) age = 29.8 (13.7) years, range = 10.3-73.2) with SMA. PROs including Voice Handicap Index (VHI) and Eating Assessment Tool-10 (EAT-10) were collected alongside clinical characteristics and standardized motor assessments. Associations were assessed using Spearman correlation coefficients and group comparisons were performed using Wilcoxon rank sum tests.

resultsA majority of the 47 participants were SMA type 2 (70.2%), non-sitters (78.7%), 3 copies of SMN2 (77.5%), and using respiratory support (66.0%). A majority (94%) reported voice issues primarily in 8/30 VHI questions. Problems included: difficulty understanding me in a noisy room (87.2%); difficult for people to hear me (74.5%); and people ask me to repeat when speaking face-to-face (72.3%). A majority (85.1%) reported swallowing issues primarily in 3/10 EAT-10 questions: swallowing pills (68.1%); food sticks to my throat (66.0%); and swallowing solids (61.7%). The two PROs were moderately associated (rs = 0.66).

conclusionsWeaker individuals with SMA experience bulbar problems including difficulties with voice and swallowing. Further refinement and assessment of functional bulbar scales will help determine their relevance and responsiveness to changes in SMA. Additional study is needed to quantify bulbar changes caused by SMA and their response to disease-modifying treatments.

Indexed as

Deglutition DisordersMuscular Atrophy, SpinalSpinal Muscular Atrophies of ChildhoodAdolescentAdultAgedChildDeglutitionHumansMiddle AgedPatient Reported Outcome MeasuresYoung Adultcommunicationdeglutitiondysphagiapatient-reported outcome measuresspeechSpinal muscular atrophyswallowvoice

Identifiers

PMID36776075
PMCPMC10258884
OpenAlexW4319789683

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.