Table 1.
Demographic And Clinical Characteristics At Treatment Start For The Evaluable Treated Renal And Cardiac Cohorts
| Characteristic | Evaluable Treated Renal Cohort | Evaluable Treated Cardiac Cohort | ||||
|---|---|---|---|---|---|---|
| Female | Male | Total | Female | Male | Total | |
| Patients, n | 62 | 90 | 152 | 34 | 35 | 69 |
| White race, n (%) | 60 (96.8) | 85 (94.4) | 145 (95.4) | 30 (88.2) | 33 (94.3) | 63 (91.3) |
| Median (range) age at treatment start, years | 48.75 (17.9–67.3) | 34.35 (18.0–66.8) | 39.95 (17.9–67.3) | 46.70 (3.7–67.3) | 28.20 (4.0–54.2) | 38.80 (3.7–67.3) |
| Median (range) time from start of ERT to FOS data extraction, years | 12.50 (10.1–17.1) | 14.40 (10.1–18.4) | 13.30 (10.1–18.4) | 12.75 (10.1–16.5) | 14.30 (10.2–17.1) | 13.60 (10.1–17.1) |
| Mean (SD) blood pressure at baseline in adult patients, mm Hg | ||||||
| Systolic | 125.0 (17.9) n=55 | 125.1 (15.4) n=81 | 125.1 (16.4) n=136 | 123.7 (17.4) n=30 | 122.5 (11.2) n=33 | 123.1 (14.4) n=63 |
| Diastolic | 73.3 (12.0) n=55 | 75.2 (11.8) n=81 | 74.4 (11.9) n=136 | 72.6 (10.7) n=30 | 72.9 (11.8) n=33 | 72.8 (11.2) n=63 |
| Mean (SD) total FOS-MSSI score | 20.48 (8.43) n=62 | 21.48 (10.16) n=90 | 21.08 (9.48) n=152 | 21.04 (9.99) n=34 | 21.57 (10.09) n=35 | 21.31 (9.97) n=69 |
| Patients who received ACE/ARB medications, n (%) | 41 (66.1) n=62 | 73 (83.9) n=87 | 114 (76.5) n=149 | 23 (67.6) n=34 | 26 (74.3) n=35 | 49 (71.0) n=69 |
| Patients with urinary protein ≥1.0 g/24 hrs at baseline, n (%) | 3 (6.7) n=45 | 9 (12.9) n=70 | 12 (10.4) n=115 | 3 (11.5) n=26 | 5 (17.9) n=28 | 8 (14.8) n=54 |
| Mean (SD) baseline eGFR, mL/min/1.73 m2 | 80.40 (17.99) n=62 | 95.00 (29.68) n=90 | 89.04 (26.49) n=152 | NA | NA | NA |
| Patients in eGFR category <60 mL/min/1.73 m2 at baseline, n (%) | 10 (16.1) n=62 | 11 (12.2) n=90 | 21 (13.8) n=152 | 6 (20.0) n=30 | 4 (14.8) n=27 | 10 (17.5) n=57 |
| Mean (SD) baseline LVMI, g/m2.7 | NA | NA | NA | 52.21 (16.99) n=34 | 49.13 (16.92) n=35 | 50.65 (16.90) n=69 |
| Patients with LVH at baseline, n (%) | 25 (58.1) n=43 | 23 (48.9) n=47 | 48 (53.3) n=90 | 18 (52.9) n=34 | 14 (40.0) n=35 | 32 (46.4) n=69 |
| Mean (SD) baseline PWTD, mm | NA | NA | NA | 11.6 (2.5) n=34 | 11.1 (3.2) n=35 | 11.3 (2.9) n=69 |
Abbreviations: ACE, angiotensin-converting enzyme inhibitor; ARB, angiotensin receptor blocker; eGFR, estimated glomerular filtration rate; ERT, enzyme replacement therapy; FOS, Fabry Outcome Survey; LVH, left ventricular hypertrophy; LVMI, left ventricular mass index; MSSI, Mainz severity score index; NA, not available; PWTD, left ventricular posterior wall thickness in diastole.