Table 1. Baseline Demographics and Clinical Characteristics of the Patients.
Data are means ± SD.; NIS data are medians (range).
| Characteristic | Overall (N=130) |
Placebo (N=66) |
Diflunisal (N=64) |
|---|---|---|---|
| Demographics | |||
| Age | 59.7±11.9 | 59.2±12.2 | 60.3±11.7 |
| Gender – no. of patients (%) | |||
| Male | 87 (66.9%) | 44 (66.7%) | 43 (67.2%) |
| Female | 43 (33.1%) | 22 (33.3%) | 21 (32.8%) |
| Race – no. of patients (%) | |||
| Asian | 14 (10.8%) | 6 (9.1%) | 8 (12.5%) |
| Black | 6 (4.6%) | 5 (7.6%) | 1 (1.6%) |
| White | 102 (78.5%) | 50 (75.8%) | 52 (81.3%) |
| Other | 1 (0.8%) | 1 (1.5%) | 0 (0.0%) |
| Multiracial | 7 (5.4%) | 4 (6.1%) | 3 (4.7%) |
| ATTR Genotypes – no. of patients (%) | |||
| Met30 | 71 (54.6%) | 35 (53%) | 36 (56.3%) |
| Non-Met30 | 59 (45.4%) | 31 (47%) | 28 (43.8%) |
| Lab Data | |||
| Serum Albumin (g/dL) | 4.1±0.4 | 4.1±0.4 | 4.1±0.4 |
| Postural Systolic Blood Pressure Change (mmHg) | −11±18.9 | −13.2±20.2 | −8.8±17.4 |
| Outcomes | |||
| Disease Stage based on PND– no. of patients (%) ⩨ | |||
| I | 49 (37.7%) | 21 (31.8%) | 28 (43.8%) |
| II | 41 (31.5%) | 23 (34.8%) | 18 (28.1%) |
| IIIA | 19 (14.6%) | 8 (12.1%) | 11 (17.2%) |
| IIIB | 13 (10%) | 10 (15.2%) | 3 (4.7%) |
| IV | 8 (6.2%) | 4 (6.1%) | 4 (6.3%) |
| NIS+7 ¶ | 55.3±46.5 | 59±50 | 51.6±42.8 |
| Median (Range) | 41.4 (0–181.6) | 42.3 (0–176.1) | 39.3 (3.6–181.6) |
| NIS § | 42.5±43.2 | 45.4±46.3 | 39.4±39.9 |
| Median (Range) | 27.9 (0–164.8) | 30.8 (0–160.3) | 23.5 (0–164.8) |
| NIS-LL ǁ | 26.1±23.2 | 27.2±24.5 | 24.9±22 |
| Median (Range) | 20 (0–79.9) | 21.5 (0–79.8) | 17.8 (0–79.9) |
| Kumamoto Score ◊ | 16±12.2 | 16.7±13.5 | 15.3±10.8 |
| mBMI ** | 1021.7±240.4 | 1019±255 | 1024.4±226.3 |
| SF36 Physical † | 35.4±11.3 | 34.8±11 | 35.9±11.6 |
| SF36 Mental ǂ | 46.6±12.9 | 46.5±11.8 | 46.6±14.1 |
Plus-minus values are means ±SD. NIS scores are also expressed as medians with ranges. There were no significant differences between the groups. Percentages may not sum to 100 because of rounding.
A Polyneuropathy Disability (PND) score of I indicates sensory disturbances but preserved walking capability; a grade of II indicates impaired walking, ability without need for a stick; a grade of IIIA indicates walking only with the help of one stick; a grade of IIIB indicates walking with the help of two sticks; a grade of IV indicates confined to a wheelchair or bedridden.
Neuropathy Impairment Score plus 7 nerve tests (NIS+7) ranges from 0–270 with higher scores indicating greater neurologic deficits.
Neuropathy Impairment Score (NIS) ranges from 0–244 with higher scores indicating greater neurologic deficits.
Neuropathy Impairment Score of the Lower Limbs (NIS-LL) ranges from 0–88 with higher scores indicating greater neurologic deficits.
Kumamoto Score ranges from 0 to 102 with higher scores indicating more severe polyneuropathy.
Modified body-mass index (mBMI) is the product of BMI (weight in kilograms divided by the square of the height in meters) and serum albumin (g/L).
Physical component scores of the 36-Item Short-Form Health Survey (SF-36) range from 0 to 100, with higher scores indicating greater physical quality of life.
Mental component scores of the 36-Item Short-Form Health Survey (SF-36) range from 0 to 100, with higher scores indicating greater mental quality of life.