Table 4.
Change between baseline and year 1 evaluation in muscle function tests, spirometry, quantitative muscle MRI and patient reported outcome measures in symptomatic and asymptomatic LOPD patients.
| Symptomatic treated patients (baseline vs. 12-month follow-up) (n = 22) | P value | Asymptomatic patients (baseline vs. 12-month follow-up) (n = 10) | P value | |
|---|---|---|---|---|
| Muscle function tests | ||||
| MRC total, score | 0 (−4; +6) | 0.79 | 0 (0; +1) | 0.31 |
| MRC Lower limbs, score | 0 (−5; +4) | 0.54 | 0 (0) | 0.31 |
| Knee extension (Nm) | 10.74 (0; +20.44) | 0.19 | −0.96 (−18.11; +3.15) | 0.40 |
| Knee flexion (Nm) | 1.84 (−1.26; +5.97) | 0.20 | −0.51 (−10.13; +6.43) | 0.67 |
| Hip flexion (Nm) | 3.97 (−1.7; +9.84) | 0.14 | −0.75 (−15.1; +3.9) | 0.40 |
| Hip extension (Nm) | 2 (−0.13; +5.47) | 0.08 | −4.15 (−16.04; +1.82) | 0.40 |
| 6-MWT, m | 0 (−9.5; +11) | 0.96 | 10 (−24.7; +7.5) | 0.48 |
| Time to walk 10 m, seconds | 0(−1.44; +0.4) | 0.43 | −0.1(−2.52; +0.32) | 0.91 |
| Time to climb 4 steps, seconds | 0 (−0.42; +0.5) | 0.77 | −0.1 (−0.32; 0) | 0.39 |
| Time to descend 4 steps, seconds | 0 (−0.35; +1.32) | 0.43 | −0.05 (−0.12; +0.1) | 0.55 |
| Timed up & go test, sec | 0.6 (−0.4; +5.2) | 0.47 | −0.15 (−0.72; +0.97) | 0.94 |
| MFM-20, score | 0 (−2; +2) | 0.63 | 0 (0; +1) | 0.33 |
| Respiratory studies | ||||
| CVF seated (%) | −0.3 (−4; +3) | 0.24 | 0 (−6; +5.77) | 0.73 |
| CVF lying (%) | 0 (−6.2; +7.62) | 0.73 | 9 (−2; +12) | 0.13 |
| Muscle MRI | ||||
| qMRI Thighs | 1.79 (+0.2; +2.4) | 0.001 | −0.11 (−0.82; +0.4) | 0.57 |
| qMRI Paraspinal | −0.03 (−2.23; +0.73) | 0.34 | 0 (−1.25; +1.89) | 0.77 |
| Patient-reported outcomes | ||||
| Activlim | 33 (+1; +59) | 0.81 | 67.5 (+37.5; +70.2) | 0.31 |
| SF36 | 1.8 (+0.67; +10.73) | 0.22 | 3.28 (+24.84; +10.12) | 0.89 |
| INQoL | −2.9 (−5.5; +0.73) | 0.17 | −4.3 (−8.2; +1.1) | 0.31 |
Median value and 25th–75th percentiles are shown. Paired Wilcoxon signed rank test was used to find out whether the differences observed between baseline and year 1 evaluation were statistically significant. Adjustment for multiple comparisons using Bonferroni correction was applied; p was considered significant if lower than 0.002.