Table 1.
Baseline demographics and clinical characteristics
| Variable | 0.2 mg/kg EOW | 0.2 mg/kg weekly | 0.4 mg/kg weekly | P-value (0.2 mg/kg, EOW vs weekly) |
|---|---|---|---|---|
| Number of patients | 20 | 19 | 5 | |
| Age, years | ||||
| Mean (SD) | 50.3 (7.2) | 51.8 (11.4) | 49.4 (9.8) | 0.6418 |
| Median (range) | 50.5 (37.0–63.0) | 49.6 (31.0–70.0) | 52.7 (33.0–57.0) | |
| Age group, n (%) | ||||
| >45 years old | 15 (75.0) | 13 (68.4) | 4 (80.0) | 0.7311 |
| Sex | ||||
| Male, n (%) | 14 (70.0) | 10 (52.6) | 2 (40.0) | 0.3332 |
| Time since FD diagnosis, mean years (SD) | 2.1 (2.38) | 1.57 (2.41) | 8.13 (10.66) | |
| NYHA classification, n (%) | 0.0717 | |||
| Class I (asymptomatic) | 6 (30.0) | 10 (52.6) | 2 (40.0) | |
| Class II (mild) | 11 (55.0) | 9 (47.4) | 2 (40.0) | |
| Class III (moderate) | 3 (15.0) | 0 | 1 (20.0) | |
| Class IV (severe) | 0 | 0 | 0 | |
| Blood pressure, mmHg | ||||
| Systolic, mean (SD) | 121.2 (16.7) | 122.2 (14.2) | 119.8 (1.0) | |
| Diastolic, mean (SD) | 75.7 (9.6) | 72.7 (11.1) | 68.4 (4.4) | |
| Concomitant medication, n (%) | ||||
| ACEIs | 10 (50.0) | 8 (42.1) | – | |
| ARBs | 2 (10.0) | 7 (36.8) | – | |
Note: Concomitant medication data is not provided for the exploratory 0.4 mg/kg weekly group as shown by dashes.
Abbreviations: ACEIs, angiotensin-converting enzyme inhibitors; ARBs, angiotensin receptor blockers; EOW, every other week; FD, Fabry disease; NYHA, New York Heart Association; SD, standard deviation.