Table 2.

Mean baseline data and duration of ERT for all patients and separately for low-dose (group 1) and high-dose (group 2) patients.

VariableAll Patients (n=12)Group 1 (n=6) (ERT, 0.2 mg/kg Every Other Week)Group 2 (n=6) (ERT, 0.4 or 1.0 mg/kg Every Other Week)P Value
Patients (n)1266
Age (yr)17.6 (12.7–22.5)18.2 (10.6–25.7)17.0 (7.7–26.3)0.81
α-Galactosidase A activity (μkat/kg protein)a4.3 (1.6–7.1)4.1 (1.6–6.6)4.6 (−1.8 to 10.9)0.26
pGL3 (μmol/L)a8.7 (6.9–10.5)9.2 (8.3–10.0)8.2 (4.0–12.4)0.57
uGL3 (mol/mol)a5.1 (1.4–8.7)3.6 (0.6–6.6)6.5 (−1.6 to 14.7)0.63
ACR (mg/mmol)5.4 (2.9–7.9)4.8 (−0.3 to 10.0)5.9 (2.6–9.3)0.66
PCR (mg/mmol)17.0 (11.9–12.1)15.6 (6.7–24.5)18.4 (10.0–26.7)0.20
mGFR (ml/min per 1.73m2)105.8 (100.1–111.6)105.5 (92.8–118.2)106.2 (100.2–112.2)0.91
ERT (mo)60.3 (50.7–70.0)56.8 (34.2–79.4)63.8 (60.1–67.6)0.87
  • Values are means and 95% confidence intervals in parentheses. Group 1: patients 2, 4, 6, 8, 10, and 11. Group 2: patients 1, 3, 5, 7, 9, and 12. ACR reference: <2.5 mg/mmol; PCR reference: < 20 mg/mmol; mGFR reference: > 90 ml/min per 1.73 m2; pGL3 reference: 1.6–3.3 μmol/L; uGL3 reference: < 0.6 mol/mol; α-galactosidase A activity (without inhibitor) reference: 17.7–26.4 μkat/kg protein. pGL3, plasma GL3; uGL3, urine GL3; ACR, albumine-to-creatinine-ratio; PCR, protein-to-creatinine-ratio.

  • a All α-galactosidase A and GL3 values are from before start of ERT.