Table 6.
Baseline characteristics of patients receiving Agalsidase alfa or beta included in the assessment of disease burden and quality of life
| Agalsidase alfa | Agalsidase beta | Mann Whitney test (p value) | |
|---|---|---|---|
| Males | n = 6 | n = 23 | |
| Age at ERT commencement (yrs) | 30 (14–54) | 38 (21–69) | 0.12 |
| ERT duration (months) | 114 (22–118) | 87 (20–138) | 0.03 |
| LVH (mm) | 12.5 (9–20) | 14 (7.1–27) | 0.64 |
| Proteinuria (g/24 h) | 0.1 (0.06–0.63) | 0.23 (0–3.1) | 0.51a |
| eGFR (mL/min/1.73 m2) | 84 (49–137) | 69 (40–125) | 0.68a |
| Number with ESRF requiring RRT (%) | 1 | 6 | 1.00b |
| Number with antibody testing performed | 1 | 14 | |
| Number who were antibody positive (%) | 0 | 11 | |
| Peak antibody titre | – | 1:600 (0-1:3200) | |
| Females | n = 1 | n = 3 | |
| Age at ERT commencement (yrs) | 54 | 49 (43–62) | |
| ERT duration (months) | 114 | 36 (16–74) | |
| LVH (mm) | 11 | 16 (11–19) | |
| Proteinuria (g/24 h) | 1.2 | 0.27 (0.11–0.69) | |
| eGFR (mL/min/1.73 m2) | 63 | 76 (73–85) |
eGFR estimated glomerular filtration rate, ESRF end-stage renal failure, LVH left ventricular hypertrophy, RRT renal replacement therapy
Note: Results are presented as median with range in parentheses. No female patient had ESRF requiring RRT or assessment of their antibody status performed.
aPatients with ESRF were excluded from proteinuria and eGFR comparisons
bAssessed by Fisher’s exact t-test