Table 2.
Demographic and baseline characteristics in the full safety population
| Characteristics | Val30Met | Non-Val30Met | |
|---|---|---|---|
| Tafamidis-to-tafamidisa (n = 65) |
Placebo-to-tafamidisb (n = 63) |
Tafamidis (n = 21) |
|
| Gender, n (%) | |||
| Female | 33 (50.8) | 37 (58.7) | 8 (38.1) |
| Male | 32 (49.2) | 26 (41.3) | 13 (61.9) |
| Race, n (%) | |||
| Caucasian | 57 (87.7) | 55 (87.3) | 19 (90.5) |
| Latin American | 6 (9.2) | 7 (11.1) | |
| Asian | 1 (4.8) | ||
| African–Caribbean | 1 (4.8) | ||
| Not available | 2 (3.1) | 1 (1.6) | |
| Age, years | |||
| Mean (SD) | 40.0 (12.7) | 38.1 (12.8) | 63.1 (9.9) |
| Median (range) | 36.0 (25–74) | 34.0 (22–71) | 64.3 (44–77) |
| mBMI, mean (SD), [g/L] × [kg/m2] | 1006.4 (164.5) | 1010.7 (211.1) | 1052.5 (206.7) |
| Symptom duration, mean (SD), years | 3.9 (4.0) | 2.9 (2.7) | 5.4 (5.1) |
| NIS-LL (range, 0–88), mean (SD) | 8.4 (11.3) | 11.1 (13.4) | 27.6 (24.7) |
| Norfolk QOL-DN (TQOL) (range, −2 to 138), mean (SD) | 27.4 (24.0) | 30.5 (26.7) | 47.8 (35.1) |
aTafamidis-to-tafamidis refers to patients randomized to tafamidis in the registration trial and continued with tafamidis treatment in the open-label extension studies
bPlacebo-to-tafamidis refers to patients randomized to placebo in the registration trial and switched to tafamidis in the open-label extension studies
Baseline refers to the baseline of the parent studies [17, 18]
mBMI modified body mass index, NIS-LL Neuropathy Impairment Score for the Lower Limbs, Norfolk QOL-DN Norfolk Quality of Life-Diabetic Neuropathy questionnaire, TQOL total quality-of-life score