Table 2.
Treatment results in 11 hemizygous patients who had Fabry disease and received EOW (2 to 4 yr) versus weekly (2 yr) ERT with 0.2 mg/kg agalsidase alfaa
| Patient | ERT Frequency | eGFR Slope (ml/min per 1.73 m2/mo) | Agalsidase alfa IgG Antibody Statusb | MAP (mmHg) | ACEi/ARB Statusc | Protein Excretion (mg/d) |
|---|---|---|---|---|---|---|
| 1 | EOW | −0.85 | 2 | 94 | 0 | 1785 |
| Weekly | −0.22 | 1 | 93 | 1 | 3302 | |
| 2 | EOW | −0.56 | 0 | 64 | 0 | 1025 |
| Weekly | −0.57 | 0 | 71 | 1 | 715 | |
| 3 | EOW | −0.41 | 1 | 94 | 0 | 123d |
| Weekly | 0.29 | 0 | 87 | 0 | 215 | |
| 6 | EOW | −0.54 | 2 | 84 | 0 | 3123 |
| Weekly | −1.14 | 2 | 80 | 1 | 1344 | |
| 7 | EOW | −0.43 | 2 | 104 | 0 | 264 |
| Weekly | 0.14 | 2 | 104 | 1 | 504 | |
| 8 | EOW | −0.78 | 0 | 76 | 0 | 199d |
| Weekly | −0.12 | 0 | 74 | 0 | 99 | |
| 9 | EOW | −0.59 | 0 | 96 | 1 | 225 |
| Weekly | 0.07 | 0 | 94 | 1 | 220 | |
| 10 | EOW | −0.51 | 0 | 73 | 0 | 1023 |
| Weekly | −0.30 | 0 | 89 | 1 | 3172 | |
| 11 | EOW | −0.97 | 0 | 95 | 1 | 2577 |
| Weekly | −0.58 | 0 | 89 | 1 | 999 | |
| 12 | EOW | −1.12 | 2 | 86 | 0 | 3774 |
| Weekly | −0.26 | 2 | 72 | 1 | 1476 | |
| 14 | EOW | −0.56 | 0 | 78 | 0 | 2220 |
| Weekly | −0.29 | 0 | 93 | 1 | 1938 |
a
ACEi/ARB, angiotensin-converting enzyme inhibitors/angiotensin receptor blockers; ERT, enzyme replacement therapy; MAP, mean arterial pressure.
b
0, none; 1, transiently positive; 2, permanently positive.
c
0, no; 1, yes.
d
Previous observation carried forward.