Baseline characteristics of the safety population
| Migalastat n=36 | ERT n=21 | All N=57 | |
|---|---|---|---|
| Age (years)±SEM (min, max) | 50.5±2.3 (18, 70) | 46.3±3.3 (18, 72) | 48.9±1.9 (18, 72) |
| Sex, n (%) | |||
| Male | 16 (44) | 9 (43) | 25 (44) |
| Female | 20 (56) | 12 (57) | 32 (56) |
| Years since diagnosis (mean±SEM) | 10.2±2.0 | 13.4±2.6 | 11.4±1.6 |
| 24-hour protein (mg/24 hours, mean±SEM) | 267±69 | 360±150 | 301±70 |
| Median | 129 | 108 | 128 |
| IQR | 393 | 483 | 408 |
| Min, max | 0, 2282 | 0, 3154 | 0, 3154 |
| mGFRiohexol (mL/min/1.73 m2, mean±SEM) | 82.4±3.0 | 83.6±5.2 | 82.8±2.6 |
| eGFRCKD-EPI (mL/min/1.73 m2, mean±SEM) | 89.6±3.7 | 95.8±4.1 | 91.9±2.8 |
| eGFRMDRD (mL/min/1.73 m2, mean±SEM) | 83.6±3.7 | 87.8±19.0 | 85.1±28.0 |
| Left ventricular mass index (g/m2) | 97.5±4.7 | 94.6±5.6 | 96.5±3.6 |
| ERT, n (%) | |||
| Agalsidase beta | 11 (31) | 8 (38) | 19 (33) |
| Agalsidase alfa | 24 (67) | 13 (62) | 37 (65) |
| Use of ACEI/ARB/RI, n (%) | 16 (44) | 11 (52) | 27 (47) |
| Amenable GLA mutation, n (%) | 34 (94) | 19 (90) | 53 (93) |
Based on randomised and treated patients. There were no statistically significant differences between treatment groups for these parameters (t-test; Fisher's exact).
ACEI, ACE inhibitor; ARB, angiotensin II receptor blocker; eGFRCKD-EPI, estimated GFR using the Chronic Kidney Disease Epidemiology Collaboration formula; eGFRMDRD, annualised change in estimated GFR using the Modification of Diet in Renal Disease; ERT, enzyme replacement therapy; GFR, glomerular filtration rate; mGFRiohexol, measured GFR using iohexol clearance; RI, renin inhibitor.