Table 3.
Annualised GFR from baseline to month 18
| ANCOVA* | Migalastat, mean±SEM† (95% CI), n=34 |
ERT, mean±SEM† (95% CI), n=18 |
Means within 2.2 mL/min/1.73 m2/year |
>50% overlap of the 95% CI |
|---|---|---|---|---|
| eGFRCKD-EPI | −0.40±0.93 (−2.27 to 1.48) |
−1.03±1.29 (−3.64 to 1.58) |
Yes | Yes |
| mGFRiohexol | −4.35±1.64 (−7.65 to −1.06) |
−3.24±2.27 (−7.81 to 1.33) |
Yes | Yes |
| eGFRMDRD | −1.51±0.95 (−3.43 to 0.40) |
−1.53±1.32 (−4.20 to 1.13) |
NA | NA |
GFR=mL/min/1.73 m2/year.
*mITT (randomised patients with amenable mutations receiving at least one dose of study medication and having a baseline and postbaseline efficacy measures of eGFRCKD-EPI and mGFRiohexol).
†Least-squares means.
ANCOVA, analysis of covariance; eGFRCKD-EPI, estimated GFR using the Chronic Kidney Disease Epidemiology Collaboration formula; eGFRMDRD, annualised change in eGFR using the Modification of Diet in Renal Disease; ERT, enzyme replacement therapy; GFR, glomerular filtration rate; mGFRiohexol, measured GFR using iohexol clearance; mITT, modified intention-to-treat population; NA, not assessed.