SIBLING CONCORDANCE FOR CLINICAL FEATURES OF DUCHENNE AND BECKER MUSCULAR DYSTROPHIES

Details

• Alternative Title:
  Muscle Nerve
• Personal Author:
  PETTYGROVE, SYDNEY ; LU, ZHENQIANG ; ANDREWS, JENNIFER G. ; MEANEY, F. JOHN ; SHEEHAN, DANIEL W. ; PRICE, ELINORA T. ; FOX, DEBORAH J. ; PANDYA, SHREE ; OUYANG, LIJING ; APKON, SUSAN D. ; POWIS, ZOE ; CUNNIFF, CHRISTOPHER
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• Description:
  Introduction ; The correlation of markers of disease severity among brothers with Duchenne or Becker muscular dystrophy has implications for clinical guidance and clinical trials. ; Methods ; Sibling pairs with Duchenne or Becker muscular dystrophy (n = 60) were compared for ages when they reached clinical milestones of disease progression, including ceased ambulation, scoliosis of ≥ 20°, and development of cardiomyopathy. ; Results ; The median age at which younger brothers reached each milestone, compared with their older brothers ranged from 25 months younger for development of cardiomyopathy to 2 months older for ceased ambulation. For each additional month of ambulation by the older brother, the hazard of ceased ambulation by the younger brother decreased by 4%. ; Conclusions ; The ages when siblings reach clinical milestones of disease vary widely between siblings. However, the time to ceased ambulation for older brothers predicts the time to ceased ambulation for their younger brothers.
• Subjects:
  • Age Factors
  • Cardiomyopathies
  • Child
  • Child, Preschool
  • Disease Progression
  • Humans
  • Incidence
  • Male
  • Muscular Dystrophy, Duchenne
  • Prognosis
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• Keywords:
  • ambulation
  • Becker muscular dystrophy
  • cardiomyopathy
  • Duchenne muscular dystrophy
  • scoliosis
  • sibling features
• Source:
  Muscle Nerve. 2013; 49(6):814-821
• Pubmed ID:
  24030636
• Pubmed Central ID:
  PMC4481732
• Document Type:
  Journal Article
• Funding:
  CC999999/Intramural CDC HHSUnited States/ ; U01 DD000187/DD/NCBDD CDC HHSUnited States/ ; DD000187/DD/NCBDD CDC HHSUnited States/
• Volume:
  49
• Issue:
  6
• Download URL:
  https://stacks.cdc.gov/view/cdc/31762/cdc_31762_DS1.pdf
• File Type:
  [PDF - 395.73 KB]
• Collection(s):
  CDC Public Access
• Main Document Checksum:
  urn:sha256:1d56553fc1cb107e228e44c192c74ebf5a875093d5c98d42ad10282d02e051dc