Table 1.
Patient demographics and baseline characteristics from the beginning of migalastat treatment
| Overall n=97 | ERT-naïve patients n=48 | ERT-experienced patients n=49 | |
| Sex, n (%) | |||
| Male | 37 (38.1) | 18 (37.5) | 19 (38.8) |
| Female | 60 (61.9) | 30 (62.5) | 30 (61.2) |
| Age, years | |||
| Mean (SD) | 46.4 (13.15) | 43.1 (11.27) | 49.5 (14.17) |
| Median (Q1, Q3) | 47.0 (36.0, 57.0) | 45.0 (35.0, 49.5) | 53.0 (43.0, 59.0) |
| Time since Fabry diagnosis, years | |||
| Mean (SD) | 9.84 (10.38) | 7.13 (7.69) | 12.45 (11.93) |
| Median (Q1, Q3) | 5.5 (3.0, 11.0) | 5.0 (2.0, 9.0) | 6.0 (5.0, 18.0) |
| eGFRCKD-EPI, mean (SD), mL/min/1.73 m2 | 90.83 (22.35) | 92.75 (24.26) | 88.96 (20.39) |
| eGFRCKD-EPI category, n (%) | |||
| >90 mL/min/1.73 m2 | 50 (51.5) | 26 (54.2) | 24 (49.0) |
| 60–90 mL/min/1.73 m2 | 39 (40.2) | 17 (35.4) | 22 (44.9) |
| >30–<60 mL/min/1.73 m2 | 8 (8.2) | 5 (10.4) | 3 (6.1) |
| Urinary protein, median (Q1, Q3), mg/24 hours | 198.0 (82.0, 353.0) | 245.0 (121.5, 399.5) | 116.0 (0.0, 265.0) |
| LVMI, mean (SD), g/m2 | 93.88 (29.59) | 96.50 (32.90) | 91.48 (26.32) |
| Patients taking ACEI/ARB/RI, n (%) | 40 (41.2) | 14 (29.2) | 26 (53.1) |
ACEI, angiotensin-converting enzyme inhibitor; ARB, angiotensin receptor blocker; eGFRCKD-EPI, estimated glomerular filtration rate using the chronic kidney disease epidemiology collaboration equation; ERT, enzyme replacement therapy; LVMI, left ventricular mass index; RI, renin inhibitor.