Table 4 Changes in clinical assessments at 24 months with patisiran treatment
Assessment | Change from baseline to Month 24 | |
|---|---|---|
n | Mean (SEM) | |
EQ-5D (max. impairment: 0) | 26 | −0.01 (0.02) |
R-ODS (no limitations: 48) | 25 | −1.8 (0.8) |
10-m walk (m/s) | 21 | 0.03 (0.04) |
Hand grip strength (kg) | 26 | 1.5 (1.2) |
mBMI (kg/m2 × g/L) | 22 | −60.8 (34.9) |
COMPASS-31 (max. impairment: 100) | 26 | 1.3 (1.8) |
Cardiac subgroup (n = 11) | ||
NT-proBNP (pg/mL) | 8 | −49.6 (170.8) |
Troponin I (ng/mL) | 8 | −0.1 (0.1) |
LV mass (g) | 10 | −16.7 (11.7) |
LV wall thickness (cm) | 10 | −0.08 (0.1) |
Ejection fraction (%) | 10 | −0.6 (1.5) |
Peak longitudinal strain (%) | 10 | 0.9 (0.9) |
10-m walk (m/s) | 7 | 0.03 (0.05) |