Evidence map›Paper›PMID 35577579›Full record

ArticleNeurology2022

Prospective Natural History Study in 24 Adult Patients With LGMDR12 Over 2 Years of Follow-up: Quantitative MRI and Clinical Outcome Measures.

Bram De Wel, Lotte Huysmans, Ronald Peeters, Veerle Goosens, Stefan Ghysels, Kris Byloos, Guido Putzeys, Ann D'Hondt, Jan L De Bleecker, Patrick Dupont and 2 more

Abstract read
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Article in Neurology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed, 1 pooled it
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.

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

16 citing papers in PubMed, 1 synthesis or guideline pooled it, 19 citations in OpenAlex.

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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

12 authors at 1 institution in 1 country.

Bram De WelFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Lotte HuysmansFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Ronald PeetersFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Veerle GoosensFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Stefan GhyselsFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Kris ByloosFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Guido PutzeysFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Ann D'HondtFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Jan L De BleeckerFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Patrick DupontFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Frederik MaesFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium.
Kristl G ClaeysFrom the Departments of Neurology (B.D.W., A.D.H., K.G.C.) and Radiology (R.P., V.G., S.G., K.B., G.P.), and Medical Imaging Research Centre (L.H., F.M.), University Hospitals Leuven; Laboratories for Muscle Diseases and Neuropathies (B.D.W., K.G.C.) and Cognitive Neurology (P.D.), Department of Neurosciences, and Department ESAT-PSI (L.H., F.M.), KU Leuven; Leuven Brain Institute (LBI) (B.D.W., K.G.C., P.D.); and Department of Neurology (J.L.D.B.), University Hospital Gent, Belgium. kristl.claeys@uzleuven.be.
Allen Institute for Brain Science · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BACKGROUND AND

objectivesLimb-girdle muscular dystrophy autosomal recessive type 12 (LGMDR12) is a rare hereditary muscular dystrophy for which outcome measures are currently lacking. We evaluated quantitative MRI and clinical outcome measures to track disease progression to determine which tests could be useful in future clinical trials to evaluate potential therapies.

methodsWe prospectively measured the following outcome measures in all participants at baseline and after 1 and 2 years: 6-minute walk distance (6MWD), 10-meter walk test (10MWT), the Medical Research Council (MRC) sum scores, Biodex isometric dynamometry, serum creatine kinase, and 6-point Dixon MRI of the thighs.

resultsWe included 24 genetically confirmed, adult patients with LGMDR12 and 24 age-matched and sex-matched healthy controls. Patients with intermediate-stage thigh muscle fat replacement at baseline (proton density fat fraction [PDFF] 20%-70%) already showed an increase in PDFF in 8 of the 14 evaluated thigh muscles after 1 year. The standardized response mean demonstrated a high responsiveness to change in PDFF for 6 individual muscles over 2 years in this group. However, in patients with early-stage (<20%) or end-stage (>70%) muscle fat replacement, PDFF did not increase significantly over 2 years of follow-up. Biodex isometric dynamometry showed a significant decrease in muscle strength in all patients in the right and left hamstrings (-6.2 Nm, DISCUSSION: Thigh muscle PDFF imaging is a sensitive outcome measure to track progressive muscle fat replacement in selected patients with LGMDR12 even after 1 year of follow-up and correlates with clinical outcome measures. Biodex isometric dynamometry can reliably capture the loss of muscle strength over the course of 1 year in patients with LGMDR12 and should be included as an outcome measure in future clinical trials as well.

Indexed as

Magnetic Resonance ImagingProtonsAdultCreatine KinaseHumansMuscle, SkeletalMuscular Dystrophies, Limb-GirdleOutcome Assessment, Health CareProspective StudiesCreatine KinaseProtons

Identifiers

PMID35577579
OpenAlexW4280587081

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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.