Table 2.

Comparison of estimated rate of change per year of clinical characteristics by treatment status

Clinical outcome	Tafamidis (Est ± SE)	Matched controls (Est ± SE)	Tafamidis—matched controls (Est ± SE)	p value
Mapped NIS-LL	0.32 ± 0.49	2.94 ± 0.37	− 2.61 ± 0.46	< 0.001
NCS	0.75 ± 1.03	5.82 ± 0.75	− 5.07 ± 1.10	< 0.001
Reflex score	0.17 ± 0.06	0.33 ± 0.04	− 0.16 ± 0.07	0.023
Motor score	0.32 ± 0.42	2.65 ± 0.30	− 2.33 ± 0.41	< 0.001
Sensory score	0.53 ± 0.56	2.37 ± 0.38	− 1.84 ± 0.64	0.004
Norfolk TQoL score	− 1.56 ± 0.78	1.67 ± 0.46	− 3.23 ± 0.91	< 0.001
Karnofsky index	− 0.43 ± 0.31	0.09 ± 0.17	− 0.53 ± 0.35	0.13
mBMI, kg/m2 × g/L	18.21 ± 4.81	18.57 ± 3.02	− 0.35 ± 5.68	0.95
BUN, mg/dL	0.94 ± 0.91	4.03 ± 0.54	− 3.10 ± 1.06	0.004

Estimates were calculated from models of tafamidis treatment effect on outcome, adjusted for the following covariates: baseline value, follow-up time, baseline treatment propensity score, gender, age at baseline, duration of ATTR-related symptoms at baseline, and a treatment-by-time interaction. The number of treated subjects (numbers were matched with controls) at baseline, 12 months, and 24 months, respectively, for each measure were as follows: Mapped NIS-LL 203, 173, 51; NCS 175, 146, 43; Reflex score 238, 200, 73; Motor score 227, 192, 68; Sensory score 178, 150, 44; Norfolk TQoL score 189, 176, 58; Karnofsky index 243, 206, 74; mBMI 170, 130, 47; BUN 210, 171, 56

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