Longitudinal data of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy
Rattachement africain : jp. Niveau de preuve : code pays fourni par la source.
Le résumé fourni par la source
INTRODUCTION/AIMS: Duchenne muscular dystrophy (DMD) presents with skeletal muscle weakness, followed by cardiorespiratory involvement. The need for longitudinal data regarding DMD that could serve as a control for determining treatment efficacy in clinical trials has increased notably. The present study examined the longitudinal data of Japanese DMD patients collectively and assessed individual patients with pathogenic variants eligible for exon-skipping therapy. METHODS: Patients with DMD who visited Kobe University Hospital between March 1991 and March 2019 were enrolled. Data between the patients' first visit until age 20 years were examined. RESULTS: Three hundred thirty-seven patients were included. Serum creatine kinase levels showed extremely high values until the age of 6 years and a rapid decline from ages 7-12 years. Both the median 10-m run/walk velocity and rise-from-floor velocity peaked at the age of 4 years and declined with age. The values for respiratory function declined from the age of 11 years. The median left ventricular ejection fraction was >60% until the age of 12 years and rapidly declined from ages 13-15 years. Examination of the relationship between pathogenic variants eligible for exon-skipping therapy and longitudinal data revealed no characteristic findings. DISCUSSION: We found that creatine kinase levels and motor, respiratory, and cardiac functions each exhibited various changes over time. These findings provide useful information about the longitudinal data of several outcome measures for patients with DMD not receiving corticosteroids. These data may serve as historical controls in comparing the natural history of DMD patients not on regular steroid use in appropriate clinical trials.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Longitudinal data of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy
- Date Crossref
- 21/03/2024
- Éditeur
- Wiley
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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Tottori University Research Initiative Center pays non établi dans la noticeUniversité ou école supérieure
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Kobe University Department of Pediatrics pays non établi dans la noticeUniversité ou école supérieure
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Kobe University Hospital pays non établi dans la noticeÉtablissement de santé
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Nagahama Institute of Bio-Science and Technology pays non établi dans la noticeUniversité ou école supérieure
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Hyogo Medical University Department of Pediatrics pays non établi dans la noticeUniversité ou école supérieure
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Hyogo University pays non établi dans la noticeUniversité ou école supérieure
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Kobe Tokiwa University pays non établi dans la noticeUniversité ou école supérieure
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Nagahama Institute of Bio‐Science and Technology Nagahama Japan pays non établi dans la noticeStructure de recherche
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Faculty of Health Sciences pays non établi dans la noticeUniversité ou école supérieure
Research Initiative Center — Tottori University, Department of Pediatrics — Kobe University et Kobe University Hospital, avec 6 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.