From: Longitudinal functional and imaging outcome measures in FKRP limb-girdle muscular dystrophy
Baseline | 16- week follow-up (Day 1) | 16-week follow-up (Day 2 – repeatability testing) | |||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Summary statistics | Summary statistics | Difference from baseline | Summary statistics | Difference from Day 1 | |||||||||
Outcome | Median | IQR | Range | Median | IQR | Range | p-value | 95% CI | Median | IQR | Range | p-value | 95% CI |
Two minute walk distance (2MWD), meters | 135 | 130–163 | 17–233 | 141 | 127–163 | 14–239 | 0.984 | −3.31-5 | 145 | 129–168 | 18–236 | 0.082 | −2.47-4.82 |
Ten meter walk/run (10MWR), seconds | 7.1 | 6.3–7.6 | 2.8–40.4 | 6.8 | 6.3–8 | 2.9–74 | 0.629 | −0.2-0.29 | 7.3 | 6.5–8 | 2.8–58 | 0.269 | −0.1–0 |
Four-stair climb (4SC), seconds | 4.9 | 3.9–6.6 | 1.7–49.8 | 5.1 | 4.2–7.2 | 1.9–54.2 | 0.016 | 0.1–0.7 | 5.8 | 4.5–8.2 | 1.9–61 | 0.475 | −0.35-0.58 |
Timed up-and-go (TUG), seconds | 10 | 9–12 | 6–80 | 11 | 9–12 | 7–81 | 0.103 | 0–1 | 11 | 9–13 | 6–90 | 0.716 | −0.82-0.82 |
Performance of upper limb (PUL) | 42 | 37–42 | 30–42 | 42 | 37–42 | 29–42 | 0.553 | −1-0 | 41.5 | 35–42 | 29–42 | 0.567 | 0–0 |
Manual muscle sum score (MMT) | 182 | 167–198 | 145–262 | 186 | 175–200 | 142–258 | 0.158 | −3-13 | |||||
Forced vital capacity (FVC), liters | 2.905 | 2.45–3.71 | 1.98–5.94 | 2.97 | 2.53–3.57 | 1.96–5.76 | 0.647 | −0.13-0.12 | |||||
Forced expiratory volume (FEV1), liters | 2.535 | 2.26–2.97 | 1.53–4.72 | 2.56 | 1.88–2.94 | 1.55–4.69 | 0.053 | −0.17-.004 | |||||
Mean inspiratory pressure (MIP), cmH2O | 48.5 | 39–62 | 26–124 | 55 | 43–72 | 23–117 | 0.037 | 0.02–7.98 | |||||
Mean expiratory pressure (MEP), cmH2O | 59.5 | 49–80 | 31–154 | 70 | 52–90 | 29–128 | 0.129 | −3.96-13.97 | |||||
DEXA - lean body mass, kg | 35.3 | 29.2–38.9 | 22.7–50.5 | 34.7 | 29.8–39 | 22.1–51.4 | 0.856 | −0.46-0.73 | |||||
DEXA - body fat, kg | 29.7 | 27.9–38.2 | 15.7–45.8 | 29.2 | 28.2–37 | 16.3–49.2 | 0.809 | −1.03-0.83 | |||||
MRI - intramuscular fat fraction, % | 67.9 | 57.8–72.9 | 36.2–90.3 | 69.3 | 62.4–72.8 | 38.1–88.5 | 0.266 | −.072–1.86 |