Dashed lines show Kaplan–Meier estimates of time to any clinical event (composite outcome). Solid lines show curves with adjustment for baseline proteinuria (urine protein–creatinine ratio), derived from a Cox regression analysis with baseline proteinuria added as a covariate to the regression equation (22, 23). Top. The time-to-event analyses for the composite end point for both treatment groups in the intention-to-treat population are shown. The unadjusted treatment-related hazard ratio associated with agalsidase beta was 0.57 (95% CI, 27.0 to 1.22; P = 0.14). With adjustment for baseline proteinuria, the hazard ratio was 0.47 (CI, 0.21 to 1.03; P = 0.06). Bottom. The time-to-event analyses for the composite end point for both treatment groups in the per-protocol population are shown. The unadjusted treatment-related hazard ratio associated with agalsidase beta was 0.54 (CI, 0.25 to 1.19; P = 0.12). With adjustment for baseline proteinuria, the hazard ratio was 0.39 (CI, 0.16 to 0.93; P = 0.034).

Proteinuria denotes the urine protein–creatinine ratio. The P values for formal tests for treatment interaction were as follows: baseline serum creatinine level, P = 0.16; baseline eGFR, P = 0.09; and baseline proteinuria, P = 0.54.

Longitudinal analysis of proteinuria in both groups.

Data are shown as increases or decreases from baseline proteinuria (urinary protein–urinary creatinine ratio) for the placebo group (solid line) and the agalsidase-beta group (dashed line). A mixed model with fixed effects for treatment group, time, and treatment-by-time interactions, as well as random intercepts and slopes, for each patient over time was fit. The overall P value for treatment effect is 0.32.