Neurology · 2020
Muscle ultrasound is a responsive biomarker in facioscapulohumeral dystrophy.
СтатьяНаблюдательное исследованиеПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод пока не подготовлен
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Goselink Rianne J M, Schreuder Tim H A, Mul Karlien, Voermans Nicol C, Erasmus Corrie E, van Engelen Baziel G M, van Alfen Nens
Аннотация
OBJECTIVE: With drug trials starting soon, responsive, relevant, and patient-friendly biomarkers are highly needed in facioscapulohumeral dystrophy (FSHD). Our objective was to assess muscle ultrasound (MUS) as an imaging biomarker in patients with FSHD.
METHODS: One-year observational, longitudinal study of both quantitative and qualitative MUS changes in FSHD.
RESULTS: Twenty-two patients with symptomatic FSHD1 underwent a clinical examination and MUS at baseline and after 1-year follow-up. The qualitative MUS sum score increased from 18.59 to 20.32 (p = 0.005) and the quantitative MUS sum z scores increased from 19.96 to 24.72 (p = 0.003). The clinical scores did not change over 1 year. Muscle echogenicity correlated with the FSHD clinical score at baseline (r = 0.61, p = 0.002).
CONCLUSIONS: MUS shows a significant increase in echogenicity in FSHD over 1 year. Both quantitative and qualitative MUS correlate cross-sectionally with clinical severity in FSHD and identify structural muscle changes in a clinically stable group of patients. MUS thus seems a potentially responsive biomarker that could be standardized between centers. We recommend its use in therapeutic trials.
CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that in patents with FSHD1, MUS findings correlate with baseline FSHD clinical scores.
METHODS: One-year observational, longitudinal study of both quantitative and qualitative MUS changes in FSHD.
RESULTS: Twenty-two patients with symptomatic FSHD1 underwent a clinical examination and MUS at baseline and after 1-year follow-up. The qualitative MUS sum score increased from 18.59 to 20.32 (p = 0.005) and the quantitative MUS sum z scores increased from 19.96 to 24.72 (p = 0.003). The clinical scores did not change over 1 year. Muscle echogenicity correlated with the FSHD clinical score at baseline (r = 0.61, p = 0.002).
CONCLUSIONS: MUS shows a significant increase in echogenicity in FSHD over 1 year. Both quantitative and qualitative MUS correlate cross-sectionally with clinical severity in FSHD and identify structural muscle changes in a clinically stable group of patients. MUS thus seems a potentially responsive biomarker that could be standardized between centers. We recommend its use in therapeutic trials.
CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that in patents with FSHD1, MUS findings correlate with baseline FSHD clinical scores.
Предметные рубрики MeSH
AdolescentAdultAgedAnatomy, Cross-SectionalBiomarkersChildCohort StudiesDisease ProgressionFemaleFollow-Up StudiesHumansLongitudinal StudiesMaleMiddle AgedMuscle, SkeletalMuscular Dystrophy, FacioscapulohumeralPredictive Value of TestsProspective StudiesReproducibility of ResultsTreatment OutcomeUltrasonographyYoung Adult