Table 3.
Comparison of estimated rate of change per year in clinical characteristics by treatment status, excluding subjects who initiated tafamidis treatment prior to enrollment in THAOS
Clinical outcome | Tafamidis (Est ± SE) | Matched controls (Est ± SE) | Tafamidis—matched controls (Est ± SE) | p value |
---|---|---|---|---|
Mapped NIS-LL | 0.25 ± 0.56 | 2.80 ± 0.44 | − 2.55 ± 0.50 | < 0.001 |
NCS | 0.51 ± 1.14 | 4.88 ± 0.84 | − 4.37 ± 1.16 | < 0.001 |
Reflex score | 0.21 ± 0.06 | 0.19 ± 0.03 | 0.03 ± 0.07 | 0.71 |
Motor score | 0.40 ± 0.46 | 2.47 ± 0.33 | − 2.07 ± 0.44 | < 0.001 |
Sensory score | 0.20 ± 0.69 | 1.88 ± 0.46 | − 1.67 ± 0.77 | 0.029 |
Norfolk TQoL score | − 2.75 ± 0.92 | 1.60 ± 0.54 | − 4.35 ± 1.05 | < 0.001 |
Karnofsky index | − 0.47 ± 0.34 | 0.18 ± 0.19 | − 0.65 ± 0.38 | 0.085 |
mBMI, kg/m2 × g/L | 20.54 ± 5.18 | 20.57 ± 3.13 | − 0.03 ± 6.05 | > 0.99 |
BUN, mg/dL | 0.44 ± 0.85 | 3.27 ± 0.49 | − 2.83 ± 0.98 | 0.004 |
Estimates were calculated from models of tafamidis treatment effect on outcome, adjusted for the baseline covariate
BUN blood urea nitrogen, Est estimate, mBMI modified body mass index, NCS Neurologic Composite Score, NIS-LL Neuropathy Impairment Score in the Lower Limbs, SE standard error, TQoL Total Quality of Life