Table 2.
CMTPedS responsiveness by baseline age, disability level, and CMT subtype.
| Criterion | All CMT | CMT1A | ||||
|---|---|---|---|---|---|---|
| Mean ± SD (n) | SRM | Sample size per arm 1 | Mean ± SD | SRM | Sample size per arm 1 | |
| Whole sample | 2.4 ± 4.92 (n = 187) | 0.5 | 66 | 1.8 ± 4.22 (n = 111) | 0.4 | 86 |
| Mild disability | 3.3 ± 5.0 (n = 79) | 0.7 | 37 | 2.8 ± 4.3 (n = 58) | 0.7 | 38 |
| Moderate disability | 2.0 ± 4.9 (n = 89) | 0.4 | 95 | 0.7 ± 3.8 (n = 52) | 0.2 | 463 |
| Severe disability | 0.5 ± 2.9 (n = 19) | 0.2 | 529 | −3.0 (n = 1) | N/A | N/A |
| Aged 11–20 years | 1.7 ± 4.0 (n = 69) | 0.4 | 87 | 1.2 ± 4.1 (n = 49) | 0.3 | 184 |
| Aged 3–10 years | 2.9 ± 4.9 (n = 95) | 0.6 | 45 | 2.2 ± 4.2 (n = 62) | 0.5 | 58 |
| Aged 3–9 years | 3.3 ± 4.9 (n = 84) | 0.7 | 35 | 2.5 ± 4.2 (n = 54) | 0.6 | 45 |
| Aged 3–8 years | 3.5 ± 5.0 (n = 69) | 0.7 | 33 | 2.7 ± 4.2 (n = 43) | 0.6 | 38 |
| Aged 3–10 years and mild disability | 3.1 ± 4.6 (n = 53) | 0.7 | 35 | 2.8 ± 4.2 (n = 44) | 0.7 | 36 |
| Aged 3–9 years and mild disability | 3.3 ± 4.6 (n = 49) | 0.7 | 31 | 3.0 ± 4.2 (n = 40) | 0.7 | 31 |
| Aged 3–8 years and mild disability | 3.7 ± 4.5 (n = 42) | 0.8 | 24 | 3.5 ± 3.9 (n = 33) | 0.9 | 20 |
1
Sample size calculated for a 2‐year randomized (1:1) double‐blind, parallel‐group, placebo‐controlled trial of an intervention aiming to halt the rate of progression per treatment arm. SRM, standardized response mean.