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

PatientERT FrequencyeGFR Slope (ml/min per 1.73 m2/mo)Agalsidase alfa IgG Antibody StatusbMAP (mmHg)ACEi/ARB StatuscProtein Excretion (mg/d)
1EOW−0.8529401785
Weekly−0.2219313302
2EOW−0.5606401025
Weekly−0.570711715
3EOW−0.411940123d
Weekly0.290870215
6EOW−0.5428403123
Weekly−1.1428011344
7EOW−0.4321040264
Weekly0.1421041504
8EOW−0.780760199d
Weekly−0.12074099
9EOW−0.590961225
Weekly0.070941220
10EOW−0.5107301023
Weekly−0.3008913172
11EOW−0.9709512577
Weekly−0.580891999
12EOW−1.1228603774
Weekly−0.2627211476
14EOW−0.5607802220
Weekly−0.2909311938
  • 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.