Skip to main content

Table 2 Participant distribution, median age at LOA, and Cox regression

From: Genotype characterization and delayed loss of ambulation by glucocorticoids in a large cohort of patients with Duchenne muscular dystrophy

Cox regression factor

Level of factor

Total No. of participants (No. with LOA)

Median age, years,

at LOA (95% CI)

HR (95% CI)

p Value

DMD mutation

Other deletions

315 (92)

11.03 (10.29, 11.77)

1b

 

Nonsense mutations

174 (31)

13.29 (11.24, 15.34)

0.66 (0.44, 0.99)

0.045a

 

Exon 44 amenable skipping

81 (18)

13.34 (10.53, 16.15)

0.56 (0.33, 0.94)

0.029a

 

Exon 45 amenable skipping

101 (31)

10.18 (9.79, 10.57)

1.31 (0.87, 1.97)

0.193

 

Exon 51 amenable skipping

146 (31)

10.72 (9.90, 11.54)

1.08 (0.72, 1.63)

0.709

 

Exon 53 amenable skipping

150 (40)

11.03 (10.06, 12.00)

1.13 (0.78, 1.65)

0.508

GC drug

Untreated (or treated < 1 month)

326 (111)

10.23 (9.97, 10.49)

1b

 

Deflazacort

65 (4)

13.95 (11.80, 16.10)

0.06 (0.02, 0.19)

 < 0.001

 

Prednisone or prednisolone

465 (146)

12.02 (11.40, 12.64)

0.40 (0.31, 0.52)

 < 0.001

  1. CI, confidence interval; DMD, dystrophin gene; HR, hazard ratio; LOA, loss of ambulation
  2. aSignificant
  3. bAn HR of 1 is assigned to factor levels that are taken as reference in the Cox regression model