Evidence map›Paper›PMID 34961728›Full record

ArticleNeuromuscular disorders : NMD2022

Patient reported quality of life in limb girdle muscular dystrophy.

Laurel V Kovalchick, Kameron Bates, Jeffrey Statland, Conrad Weihl, Peter B Kang, Linda P Lowes, Tahseen Mozaffar, Volker Straub, Matthew Wicklund, Chad Heatwole and 1 more

Open access · greenAbstract read
In one paragraph

Article in Neuromuscular disorders : NMD, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
0.6field-weighted citation impact, top 32% 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

10 citing papers in PubMed, 13 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Observational
  5. Article
  6. Observational
  7. Assessment of the quality of life in patients with LGMD. The case of transportinopathy.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2024
    Review
  8. Article
  9. Article
  10. Review
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

11 authors at 9 institutions in 2 countries.

Laurel V KovalchickDepartment of Neurology, Virginia Commonwealth University, 1101 E Marshall St Box 980599, Richmond, VA 23298 USA.
Kameron BatesDepartment of Neurology, Virginia Commonwealth University, 1101 E Marshall St Box 980599, Richmond, VA 23298 USA.
Jeffrey StatlandDepartment of Neurology, Kansas University Medical Center, 2100 W. 36th Avenue Suite 122, Kansas City, KS 66103 USA.
Conrad WeihlDepartment of Neurology Washington University, Campus Box 8111 660 S. Euclid Ave., St Louis, MO 63110 USA.
Peter B KangPaul & Sheila Wellstone Muscular Dystrophy Center and Department of Neurology, University of Minnesota, 516 Delaware ST SE, Minneapolis, MN 55455 USA.
Linda P LowesCenter for Gene Therapy, Nationwide Children's Hospital, 575 Children's Crossroad Columbus, OH 43205 USA.
Tahseen MozaffarDepartment of Neurology, University of California, Irvine, 200 S. Manchester Ave. Ste. 206, Orange, CA 92868 USA.
Volker StraubJohn Walton Muscular Dystrophy Research Centre, Newcastle University, Central Pkwy, Newcastle upon Tyne NE1 3BZ, UK.
Matthew WicklundDepartment of Neurology, University of Colorado, 12700 East 19th Avenue 5018, Aurora, CO 80045 USA.
Chad HeatwoleDepartment of Neurology, University of Rochester, Box 673 601 Elmwood Ave. Rochester, NY 14642 USA; Center for Health and Technology, University of Rochester, 265 Crittenden Blvd, CU 420694 Rochester, NY 14642 USA.
Nicholas E JohnsonDepartment of Neurology, Virginia Commonwealth University, 1101 E Marshall St Box 980599, Richmond, VA 23298 USA. Electronic address: Nicholas.johnson@vcuhealth.org.
Virginia Commonwealth University · USMuscular Dystrophy UK · GBNationwide Children's Hospital · USUniversity Medical Center · USUniversity of California, Irvine · USUniversity of Colorado Denver · USUniversity of Minnesota · USUniversity of Rochester · USWashington University in St. Louis · US

Funding

Chaperone Dysfunction in Myopathy: Connecting Yeast Genetics with Mouse ModelsR01AR068797 · NIAMS · WASHINGTON UNIVERSITY · PI TRUE-KROB, HEATHER L, WEIHL, CONRAD C · 2015 to 2025
$6.1M
Identifying the RNA Splicing and Gene Expression Changes that Cause Congenital Myotonic Dystrophy (Renewal)R01NS104010 · NINDS · VIRGINIA COMMONWEALTH UNIVERSITY · PI Nicholas Elwood Johnson · 2018 to 2026
$4.4M
Clinical and Translational Studies in Muscle DiseaseK24AR073317 · NIAMS · WASHINGTON UNIVERSITY · PI CONRAD C WEIHL · 2018 to 2026
$1.6M
Health Endpoints and Longitudinal Progression in Congenital Myotonic Dystrophy (HELP-CDM)K23NS091511 · NINDS · VIRGINIA COMMONWEALTH UNIVERSITY · PI JOHNSON, NICHOLAS ELWOOD · 2015 to 2019
$997k
FDA HHS R01 FD006071NIAMS NIH HHS K24 AR073317NIAMS NIH HHS R01 AR068797NINDS NIH HHS K23 NS091511NINDS NIH HHS R01 NS104010
6 · The paper itself

Abstract

This study determined the frequency and impact of symptoms on quality of life in patients diagnosed with limb girdle muscular dystrophy (LGMD). Participants with a diagnosis of LGMD in registries based at the Coalition to Cure Calpain-3, the Jain foundation, and the Global FKRP Registry competed a survey to report the frequency and relative impact of themes and symptoms of LGMD. Frequency, mean impact, and population impact scores were calculated, and responses were categorized by age, symptom duration, gender, employment status, use of assistive devices, and LGMD subtypes. 134 participants completed the survey. The most prevalent themes included an inability to do activities (100%), limitation with mobility (99.3%), and lower extremity weakness (97.0%). Themes with the greatest impact were: limitations with mobility, lower extremity weakness, and an inability to do activities. Symptom duration and the use of assistive devices were associated with the presence of multiple themes. Employment was associated with the impact of several themes with no differences in frequency. The prevalence and impact of these themes vary in the LMGD population. The most prevalent and impactful themes were related to weakness, but additional concerns related to emotional challenges should also be considered in clinical and research settings.

Indexed as

Quality of LifeAdolescentAdultAgedAged, 80 and overFemaleHumansMaleMiddle AgedMuscle WeaknessMuscular Dystrophies, Limb-GirdlePatient Reported Outcome MeasuresPhenotypeRegistriesSurveys and QuestionnairesYoung AdultDysferlin, Calpain-3FKRPLimb girdle muscular dystrophyPatient reportQuality of life

Identifiers

PMID34961728
PMCPMC10311477
OpenAlexW3213013440

What OpenQuestion holds

Textmetadata
LicenceTDM
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.