Table 2.

Progression rates, baseline proteinuria and GFR, and ERT dosing in Fabry nephropathy

Reference/Patient Nos.No. of PatientsMale/FemaleERTDuration (Months)Baseline ValuesProteinuria during ERT (mg/d)Progression Ratea
Age (Years)MDRD eGFRProteinuria (mg/d)
Warnock (16)11Agalsidase-alfa 0.2 mg/kg EOW1640.472  1510350−13.70 ± 1.50
Schwarting et al. (23), 7, 10, 12, and 1441/3Agalsidase-alfa 0.2 mg/kg EOW1257 ± 545 ± 5203 ± 162295 ± 208−5.00 ± 4.08
Schiffmann et al. (15), 3, 7, 8, and 944/0Agalsidase-alfa 0.2 mg/kg EOW4646 ± 790 ± 15216 ± 79287 ± 175−6.63 ± 2.06
Feriozzi et al. (18)40?Agalsidase-alfa 0.2 mg/kg EOW3637 ± 8; males 50 ± 12; females85 ± 26<500 <500 −1.68 ± 3.59
Feriozzi et al. (18)14?Agalsidase-alfa 0.2 mg/kg EOW3637 ± 8; males 50 ± 12; females92 ± 27>500 >500 −3.98 ± 7.86
West et al. (19)5858Agalsidase-alfa 0.2 mg/kg EOW>4834 ± 990 ± 31<1000<1000−2.10 ± 1.30
West et al. (19)2222Agalsidase-alfa 0.2 mg/kg EOW>4834 ± 990 ± 31>1000>1000−6.30 ± 1.80
Warnock (16)11Agalsidase-beta 1.0 mg/kg EOW6541.948.7350555 ± 237−2.92 ± 0.44
Breunig et al. (24), 9, 10, 11, 12, 20, 21, and 2274/3Agalsidase-beta 1.0 mg/kg EOW2240 ± 1295 ± 20152 ± 188146 ± 171−4.66 ± 6.86
Schiffmann et al. (15), 3, 7, 8, and 944/0Agalsidase-alfa 0.2 mg/kg per wk2450 ± 762 ± 15203 ± 60260 ± 17254.20 ± 5.40
Germain et al. (21)4240/2Agalsidase-beta 1.0 mg/kg EOW5229 ± 10137 ± 50277 ± 213245 ± 247−1.01 ± 0.97
Tahir et al. (29), 3, 4, 5, and 842/2Agalsidase-beta 1.0 mg/kg EOW3032 ± 1097 ± 17449 ± 433361 ± 2451.18 ± 2.78
  • eGFR calculated with the MDRD equation (ml/min per 1.73 m2). EOW, every other week.

  • a Annualized rate of change of the slope of the linear regression of eGFR over time (ml/min per 1.73 m2/yr).