Table 1

Patient demographics and baseline characteristics from the beginning of migalastat treatment

Overall n=97ERT-naïve patients n=48ERT-experienced patients n=49
Sex, n (%)
 Male37 (38.1)18 (37.5)19 (38.8)
 Female60 (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 hours198.0 (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.