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Comparative Study
. 2014 Jun;49(6):814-21.
doi: 10.1002/mus.24078.

Sibling concordance for clinical features of Duchenne and Becker muscular dystrophies

Sydney Pettygrove  1 , Zhenqiang Lu, Jennifer G Andrews, F John Meaney, Daniel W Sheehan, Elinora T Price, Deborah J Fox, Shree Pandya, Lijing Ouyang, Susan D Apkon, Zoe Powis, Christopher Cunniff
Affiliations
Comparative Study

Sibling concordance for clinical features of Duchenne and Becker muscular dystrophies

Sydney Pettygrove et al. Muscle Nerve. 2014 Jun.

Abstract

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.

Keywords: Becker muscular dystrophy; Duchenne muscular dystrophy; ambulation; cardiomyopathy; scoliosis; sibling features.

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Figures

FIGURE 1
FIGURE 1
Study population flow chart.
FIGURE 2
FIGURE 2
Kaplan-Meier survival analysis demonstrating the relationship between the proportion of younger brothers still ambulatory and whether the older brother ceased ambulation early or late. Probability of younger siblings still being ambulatory by age (years) and when the older brother has ceased ambulation is P < 0.001. Solid line, older brother ceased ambulation after the median age of cessation; broken line, older brother ceased ambulation before the median age of cessation; vertical lines, censoring.
See this image and copyright information in PMC

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