Evidence map›Paper›PMID 31876124›Full record

ArticleAnnals of clinical and translational neurology2020

Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials.

Melanie L Leitner, Kush Kapur, Basil T Darras, Michele Yang, Brenda Wong, Laura Dalle Pazze, Julaine Florence, Martin Buck, Laura Freedman, Jose Bohorquez and 2 more

2 registry-linked trialsOpen access · goldAbstract readMulticenter Study
In one paragraph

Article in Annals of clinical and translational neurology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 16 papers.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed
0.8field-weighted citation impact, top 29% 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.

NCT02340923 completednot on this map

A Device for Rapid, Painless, Bedside Muscle Evaluation of Children

Typeobservational_patient_registrySponsorSkulpt, Inc.Ran2015 to 2016Enrolled327ConditionsDuchenne Muscular Dystrophy
NCT07502989 recruitingnot on this mapstarted 2025, after this paper: background citation

Convenient Quantification of Myopathic Change in Muscle Via Electrical Impedance Myography

Typeobservational_patient_registrySponsorBeth Israel Deaconess Medical CenterRan2025 to 2027Enrolled150ConditionsMyopathy, Muscular Dystrophies, Myositis, Myofibrillar MyopathyArmsElectrical Impedance Myography
3 · Its place in the literature

Who cites it

16 citing papers in PubMed, 32 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

12 authors at 6 institutions in 1 country.

Melanie L LeitnerAccelerating NeuroVentures, LLC, Needham, Massachusetts.ORCID 0000-0002-0748-6125
Kush KapurDepartment of Neurology, Boston Children's Hospital, Boston, Massachusetts.
Basil T DarrasDepartment of Neurology, Boston Children's Hospital, Boston, Massachusetts.
Michele YangDepartment of Neurology, Children's Hospital Colorado, Denver, Colorado.
Brenda WongDepartment of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts.
Laura Dalle PazzeCharley's Fund, New York, New York.
Julaine FlorenceDepartment of Neurology, Washington University in St. Louis, St. Louis, Missouri.
Martin BuckMyolex Inc, Brookline, Massachusetts.
Laura FreedmanMyolex Inc, Brookline, Massachusetts.
Jose BohorquezMyolex Inc, Brookline, Massachusetts.
Seward RutkoveMyolex Inc, Brookline, Massachusetts.
Craig ZaidmanDepartment of Neurology, Washington University in St. Louis, St. Louis, Missouri.
Myolex (United States) · USBoston Children's Hospital · USWashington University in St. Louis · USAdenoid Cystic Carcinoma Research Foundation · USChildren's Hospital Colorado · USUniversity of Massachusetts Chan Medical School · US

Funding

A device for rapid, painless, bedside muscle evaluation of children with neuromusR44NS073188 · NINDS · MYOLEX, INC. · PI BOHORQUEZ, JOSE LUIS, ZAIDMAN, CRAIG MITCHELL · 2014 to 2015
$1.5M
Charley's FundNINDS NIH HHS R44 NS073188
6 · The paper itself

Abstract

objectiveTo evaluate the sensitivity of electrical impedance myography (EIM) to disease progression in both ambulatory and non-ambulatory boys with DMD. METHODS AND

participantsA non-blinded, longitudinal cohort study of 29 ambulatory and 15 non-ambulatory boys with DMD and age-similar healthy boys. Subjects were followed for up to 1 year and assessed using the Myolex

resultsIn the ambulatory group, EIM 100 kHz resistance values showed significant change compared to the healthy boys. For example, in lower extremity muscles, the average change in EIM 100 kHz resistance values over 12 months led to an estimated effect size of 1.58. Based on these results, 26 DMD patients/arm would be needed for a 12-month clinical trial assuming a 50% treatment effect. In non-ambulatory boys, EIM changes were greater in upper limb muscles. For example, biceps at 100kHz resistance gave an estimated effect size of 1.92 at 12 months. Based on these results, 18 non-ambulatory DMD patients/arm would be needed for a 12-month clinical trial assuming a 50% treatment effect. Longitudinal changes in the 100 kHz resistance values for the ambulatory boys correlated with the longitudinal changes in the timed supine-to-stand test. EIM was well-tolerated throughout the study.

interpretationThis study supports that EIM 100 kHz resistance is sensitive to DMD progression in both ambulatory and non-ambulatory boys. Given the technology's ease of use and broad age range of utility it should be employed as an exploratory endpoint in future clinical therapeutic trials in DMD.

trial registrationClincialtrials.gov registration #NCT02340923.

Indexed as

Disease ProgressionAdolescentChildChild, PreschoolClinical Trials as TopicElectric ImpedanceHumansLongitudinal StudiesMaleMobility LimitationMuscle, SkeletalMuscular Dystrophy, DuchenneMyographySample SizeSensitivity and Specificity

Identifiers

PMID31876124
PMCPMC6952321
OpenAlexW2998311009

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

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.