Table 1.
Parameter | Tafamidis–tafamidis (n = 38) | Placebo–tafamidis (n = 33) |
---|---|---|
Median age, years (range) | 36.5 (25 to 74) | 34.0 (23 to 71) |
Female, n (%) | 21 (55) | 18 (54) |
Caucasian, n (%) | 37 (97) | 33 (100) |
Median duration of TTR-FAP symptoms, months (range) | 17.5 (3 to 268) | 18.0 (2 to 133) |
Mean height, m (SD) | 1.66 (0.1) | 1.67 (0.1) |
Mean weight, kg (SD) | 65.1 (12.4) | 64.4 (14.4) |
66.2 (11.9)a | 63.3 (14.1)a | |
Mean serum albumin, g/l (SD) | 43.9 (3.5) | 44.2 (4.2) |
44.7 (3.4)a | 43.1 (4.0)a | |
Mean BMI, kg/m2 (SD) | 23.6 (3.6) | 23.1 (5.2) |
23.9 (3.0)a | 22.7 (5.2)a | |
Mean mBMI, kg/m2 g/l (SD) | 1,031.6 (166.5) | 1,020.2 (244.3) |
1,068.4 (142.4)a | 990.1 (265.0)a | |
NIS-LL (range 0–88) | ||
Mean (SD) | 6.8 (10.0) | 11.6 (14.5) |
Median (range) | 4.0 (0 to 49) | 6.0 (2 to 57) |
TQOL, median (range) | 14 (−1 to 110) | 17 (0 to 107) |
11 (−1 to 97)a | 28 (−1 to 96)a |
BMI body mass index, ITT intent-to-treat, NIS-LL Neuropathy Impairment Score-Lower Limbs, SD standard deviation, TQOL Norfolk Quality of Life-Diabetic Neuropathy total quality of life, TTR-FAP transthyretin familial amyloid polyneuropathy
aAt baseline of the open-label extension study