Table 6.

Change of podocyte scores and albumin-to-creatinine ratio after 5 years of ERT

Treatment (agalsidase)GL3 Inclusion Score (Toluidine Semithin Sections)Vacuolization Score (PAS Sections)Composite Score (Inclusions and Vacuolization)Albumin-to-Creatinine Ratio
Change (95% CI)P ValueChange (95% CI)P ValueChange (95% CI)P ValueChange (95% CI)P Value
0.2 mg/kg EOW01.0−0.03 (−0.23 to 0.16)1.0−0.05 (−0.25 to 0. 15)0.54−0.88 (−2.1 to 0.3)0.12
(6 patients, group 1)
0.4–1.0 mg/kg EOW−1.89 (−3.6 to −0.16)a0.037−1.1 (−2.3 to 0.08)a0.062−3.0 (−5.9 to −0.1) b0.044−0.13 (−8.9 to 8.6)0.35
(6 patients, group 2)
1.0 mg/kg EOW−2.84 (−4.37 to−1.3)c0.0100.1.66 (−3.18 to −0.14)c0.040−4.5 (−7.5 to −1.5)c0.018−4.2 (−6.0 to −2.5)c *0.005
(4 patients)
  • Score reduction is given as mean value with 95% confidence intervals in parentheses. The GL3 inclusions are given in values of 0–4, the vacuolization scores are given in values 0–3, and the combined inclusion and vacuolization scores are given in values of 0–7.17 Albumin-to-creatinine reference: <2.5 mg/mmol. CI, confidence interval; EOW, every other week.

  • a Group 2 had higher GL3 clearance compared with group 1 (P=0.022), as well as greater reduction of vacuolization (P=0.022).

  • b Group 2 had borderline higher GL3 clearance compared with group 1 (P=0.050).

  • c The subgroup of four patients (patients 1, 3, 5, and 9) with 1.0 mg/kg every other week had greater GL3 reduction in the podocytes (P=0.004), greater reduction of vacuolization (P=0.010), greater reduction of composite score (P=0.010), and greater reduction of albumin-to-creatinine ratio (P=0.011) than group 1 (0.2 mg/kg every other week).