Table 3.
Urinary excretion of glycosaminoglycan biomarkers at baseline and after 2 years of IV-ERT augmentation using a generalized estimated equations model to assess intra-patient changes.
| Condition | n | Baseline GM (95% CI) | Month 24 GM (95% CI) | GM Ratio (95% CI) | p-value (ratio) |
|---|---|---|---|---|---|
| Heparan sulfate (mg/mmol creatinine) | |||||
| All | 10 | 0.91 (0.74, 1.12) | 0.66 (0.57, 0.76) | 0.72 (0.58, 0.90) | <0.01 |
| Low/No ADA | 7 | 0.87 (0.66, 1.14) | 0.45 (0.38, 0.54) | 0.52 (0.39, 0.69) | <0.01 |
| High ADA | 3 | 1.03 (0.80, 1.33) | 1.57 (1.27, 1.95) | 1.53 (1.06, 2.21) | 0.02 |
| I0S6 (mg/mmol creatinine) | |||||
| All | 10 | 0.16 (0.12, 0.20) | 0.07 (0.06, 0.09) | 0.48 (0.38, 0.60) | <0.01 |
| Low/No ADA | 7 | 0.15 (0.11, 0.21) | 0.05 (0.04, 0.06) | 0.29 (0.24, 0.37) | <0.01 |
| High ADA | 3 | 0.16 (0.11, 0.24) | 0.24 (0.17, 0.32) | 1.46 (0.83, 2.58) | 0.19 |
| I0S0 (mg/mmol creatinine) | |||||
| All | 10 | 0.022 (0.015, 0.032) | 0.014 (0.010, 0.020) | 0.627 (0.408, 0.963) | 0.03 |
| Low/No ADA | 7 | 0.019 (0.011, 0.031) | 0.008 (0.005, 0.013) | 0.431 (0.245, 0.759) | <0.01 |
| High ADA | 3 | 0.032 (0.022, 0.046) | 0.048 (0.028, 0.081) | 1.500 (0.859, 2.619) | 0.15 |
GM = geometric means from the generalized estimated equations model; GM ratio = ratio of geometric means at 24 months compared to baseline; CI = confidence interval; ADA = anti-drug drug antibody; p-values are from tests of the null hypothesis that the true GM ratio (month 24/baseline) is 1.