Evidence map›Paper›PMID 34162720›Full record

ArticleNeurology2021

Selection Approach to Identify the Optimal Biomarker Using Quantitative Muscle MRI and Functional Assessments in Becker Muscular Dystrophy.

Nienke M van de Velde, Melissa T Hooijmans, Aashley S D Sardjoe Mishre, Kevin R Keene, Zaïda Koeks, Thom T J Veeger, Iris Alleman, Erik W van Zwet, Jan-Willem M Beenakker, Jan J G M Verschuuren and 2 more

2 registry-linked trialsOpen access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Neurology, 2021. 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 20 papers.

0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed
1.6field-weighted citation impact, top 16% 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.

NCT05618080 active not recruitingnot on this mapstarted 2024, after this paper: background citation

GRASP-01-003: Trial Readiness and Endpoint Assessment in LGMD R1

TypeobservationalSponsorVirginia Commonwealth UniversityRan2024 to 2028Enrolled100ConditionsCalpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A, Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type R1, LGMD2A
NCT05989620 recruitingnot on this mapstarted 2023, after this paper: background citation

Long-Term Development of Muscular Dystrophy Outcome Assessments (GRASP-01-005)

TypeobservationalSponsorVirginia Commonwealth UniversityRan2023 to 2029Enrolled1,000ConditionsLGMD1B, LGMD1C, LGMD1D, LGMD1E
3 · Its place in the literature

Who cites it

20 citing papers in PubMed, 26 citations in OpenAlex.

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  15. Water T2 could predict functional decline in patients with dysferlinopathy.Journal of cachexia, sarcopenia and muscle · 2022
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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 1 institution in 2 countries.

Nienke M van de VeldeFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.ORCID 0000-0002-1541-130X
Melissa T HooijmansFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.ORCID 0000-0002-2233-1383
Aashley S D Sardjoe MishreFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.ORCID 0000-0002-8813-4876
Kevin R KeeneFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.ORCID 0000-0001-9300-9888
Zaïda KoeksFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.
Thom T J VeegerFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.ORCID 0000-0001-8153-8862
Iris AllemanFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.
Erik W van ZwetFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.ORCID 0000-0001-5537-3179
Jan-Willem M BeenakkerFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.ORCID 0000-0003-0479-5587
Jan J G M VerschuurenFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.ORCID 0000-0002-4572-1501
Hermien E KanFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands.ORCID 0000-0002-5772-7177
Erik H NiksFrom the Department of Neurology (N.M.v.d.V., K.R.K., Z.K., J.J.G.M.V., E.H.N.), C.J. Gorter Center for High-Field MRI, Department of Radiology (M.T.H., A.S.D.S.M., K.R.K., T.T.J.V., J.-W.M.B., H.E.K.), Department of Orthopaedics, Rehabilitation and Physical Therapy (I.A.), Department of Biomedical Data Sciences (E.W.v.Z.), and Department of Ophthalmology (J.-W.M.B.), Leiden University Medical Center, the Netherlands; and Duchenne Center Netherlands (N.M.v.d.V., J.J.G.M.V., H.E.K., E.H.N.), the Netherlands. e.h.niks@lumc.nl.ORCID 0000-0001-5892-5143
Duchenne Parent Project · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveTo identify the best quantitative fat-water MRI biomarker for disease progression of leg muscles in Becker muscular dystrophy (BMD) by applying a stepwise approach based on standardized response mean (SRM) over 24 months, correlations with baseline ambulatory tests, and reproducibility.

methodsDixon fat-water imaging was performed at baseline (n = 24) and 24 months (n = 20). Fat fractions (FF) were calculated for 3 center slices and the whole muscles for 19 muscles and 6 muscle groups. Contractile cross-sectional area (cCSA) was obtained from the center slice. Functional assessments included knee extension and flexion force and 3 ambulatory tests (North Star Ambulatory Assessment [NSAA], 10-meter run, 6-minute walking test). MRI measures were selected using SRM (≥0.8) and correlation with all ambulatory tests (ρ ≤ -0.8). Measures were evaluated based on intraclass correlation coefficient (ICC) and SD of the difference. Sample sizes were calculated assuming 50% reduction in disease progression over 24 months in a clinical trial with 1:1 randomization.

resultsMedian whole muscle FF increased between 0.2% and 2.6% without consistent cCSA changes. High SRMs and strong functional correlations were found for 8 FF but no cCSA measures. All measures showed excellent ICC (≥0.999) and similar SD of the interrater difference. Whole thigh 3 center slices FF was the best biomarker (SRM 1.04, correlations ρ ≤ -0.81, ICC 1.00, SD 0.23%, sample size 59) based on low SD and acquisition and analysis time.

conclusionIn BMD, median FF of all muscles increased over 24 months. Whole thigh 3 center slices FF reduced the sample size by approximately 40% compared to NSAA.

Indexed as

Adipose TissueAdultBiomarkersDisease ProgressionFemaleFollow-Up StudiesHumansLegMagnetic Resonance ImagingMaleMiddle AgedMuscle ContractionMuscle, SkeletalMuscular Dystrophy, DuchenneObserver VariationReproducibility of ResultsBiomarkers

Identifiers

PMID34162720
PMCPMC8356376
OpenAlexW3176887072

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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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.