Table 3. . Baseline demographics and disease characteristics for Study 019 patients with nonsense mutation Duchenne muscular dystrophy who received ataluren (40 mg/kg/day) plus standard of care in at least Study 019 (n = 60) and for propensity-score matched patients with Duchenne muscular dystrophy receiving standard of care alone in the CINRG DNHS (n = 60), for the evaluation of loss of ambulation.
| Study 019 (n = 60) | CINRG DNHS (n = 60) | p-value | |
|---|---|---|---|
| Age at first symptoms, years† | |||
| Mean (SD) | NA | 3.9 (1.7) | 0.3859‡ |
| Age at diagnosis, years | |||
| Mean (SD) | 3.6 (2.0) | 4.9 (2.3) | |
| Age at corticosteroid initiation, years§ | |||
| Mean (SD) | 10.9 (8.1) | 10.1 (8.1) | 0.6182 |
| Deflazacort duration, n (%)¶ | |||
| <1 month | 24 (40.0) | 27 (45.0) | 0.6865 |
| ≥1 to <12 months | 1 (1.7) | 2 (3.3) | |
| ≥12 months | 35 (58.3) | 31 (51.7) | |
| Other corticosteroid duration, n (%)¶ | |||
| <1 month | 37 (61.7) | 37 (61.7) | 0.6816 |
| ≥1 to <12 months | 4 (6.7) | 2 (3.3) | |
| ≥12 months | 19 (31.7) | 21 (35.0) | |
| Time to climb four stairs at first assessment, s# | |||
| n | 60 | 31 | |
| Mean (SD) | 5.3 (5.9) | 6.9 (6.5) | 0.2247 |
| Time to walk/run 10 m at first assessment, s# | |||
| n | 60 | 33 | |
| Mean (SD) | 6.6 (4.2) | 8.2 (4.5) | 0.0851 |
| Time to stand from supine at first assessment, s# | |||
| n | 60 | 26 | |
| Mean (SD) | 7.8 (8.5) | 7.2 (5.9) | 0.7296 |
P-values were calculated based on a two-sample t-test for continuous variables or a χ2 test for categorical variables.
The patients' age at first symptoms was not captured in Study 019.
P-value is for the comparison between the age at diagnosis for Study 019 patients and age at first symptoms for CINRG DNHS patients.
Age at initiation of corticosteroid use for steroid-naive patients (patients who had never used steroids or used steroids after loss of ambulation) in Study 019 was set to 30 years.
Corticosteroid duration is calculated from starting use of corticosteroid to loss of ambulation/censored date.
Time to climb four stairs, walk/run 10 m, and stand from supine at first assessment were determined using baseline values from the prior ataluren studies that the patients were enrolled in, i.e., Study 007/007e or Study 004/004e.
NA: Not available; SD: Standard deviation.