Baseline characteristics of adult patients with Fabry disease receiving agalsidase β (1 mg/kg every 2 weeks) as their initial source of ERT and included in the study
| Characteristic | All patients (n=1044) | Age <40 years at first treatment (n=526) | Age ≥40 years at first treatment (n=518) | p Value (comparing age groups) |
|---|---|---|---|---|
| Men, n (%) | 641 (61) | 390 (74) | 251 (49) | <0.01 |
| Severe clinical event pre-ERT,* n (%) | 172 (17) | 35 (7) | 137 (26) | <0.01 |
| Renal factors | ||||
| eGFR,† n | 695 | 349 | 346 | |
| Mean±SD (mL/min/1.73 m2) | 85±34 | 100±33 | 70±27 | <0.01 |
| <60 mL/min/1.73 m2, n (%) | 166 (24) | 51 (15) | 115 (33) | <0.01 |
| Protein:creatine ratio, n | 461 | 227 | 234 | |
| >0.5 g/g, n (%) | 213 (46) | 87 (38) | 126 (54) | <0.01 |
| Cardiac factors | ||||
| Echocardiography (pre-ERT), n | 667 | 293 | 374 | |
| LVH (pre-ERT),†‡ n (%) | 406 (61) | 109 (37) | 297 (79) | <0.01 |
| Arrhythmia (pre-ERT),§ n (%) | 247 (24) | 90 (17) | 157 (30) | <0.01 |
| Systolic blood pressure,† n | 727 | 365 | 362 | |
| Mean±SD (mm Hg) | 125±16 | 123±15 | 127±17 | <0.01 |
| ≥130 mm Hg, n (%) | 297 (41) | 127 (35) | 170 (47) | <0.01 |
| Diastolic blood pressure,† n | 727 | 365 | 362 | |
| Mean±SD (mm Hg) | 75±11 | 73±11 | 77±11 | <0.01 |
| ≥80 mm Hg, n (%) | 274 (38) | 109 (30) | 165 (46) | <0.01 |
*Non-renal event (stroke or cardiac event).
†Baseline eGFR values and blood pressure values were measured −3/+3 months of first ERT, and wall thickness values were measured within 3 months of the first ERT.
‡LVH criterion was wall thickness ≥12 mm. The frequency of occurrence of LVH was determined using available left posterior wall thickness values in the range 5–35 mm and/or a ‘yes’ response to the checkbox question ‘LVH present?’.
§Arrhythmia was based on the treating physician's judgement and indicated by a ‘yes’ or ‘no’ response.
eGFR, estimated glomerular filtration rate; ERT, enzyme replacement therapy; LVH, left ventricular hypertrophy.