Table 4.
Change in CMTES for patients with mild disease at baseline including only pathogenic/likely pathogenic variants by year of follow-up
| Year of follow-up | n | Female, n (%) | Mean baseline score ± SD | Mean change ± SD | Q1–Q3 | P | SRM |
|---|---|---|---|---|---|---|---|
| 1 | 25 | 17 (68) | 4.5 ± 2.0 | 1.5 ± 2.5 | 0.00–3.00 | 0.004 | 0.63 |
| 2 | 28 | 16 (57) | 4.5 ± 1.8 | 1.0 ± 2.3 | 0.00–2.00 | 0.025 | 0.45 |
| 3 | 21 | 11 (52) | 5.1 ± 1.9 | 2.3 ± 2.5 | 0.50–4.00 | 0.001 | 0.90 |
| 4 | 16 | 6 (38) | 4.9 ± 2.3 | 2.0 ± 2.4 | 0.00–3.75 | 0.004 | 0.85 |
| 5 | 14 | 8 (57) | 4.6 ± 2.3 | 2.2 ± 2.5 | 0.00–4.00 | 0.006 | 0.88 |
Significant P-values and SRMs highlighted in bold. Q1–Q3 = interquartile range for change in CMTES or CMTES-R; CMTES(-R) = Charcot-Marie-Tooth examination score (Rasch-modified); SD = standard deviation; SRM = standard response mean.