Table 3.
Baseline Demographic and Clinical Characteristics for Prompt versus Delayed Agalsidase Alfa Initiation Cohorts, Based on Time from Diagnosis (Analysis B)
| Variable | Prompt Treatment (n=1006) | Delayed Treatment (n=1045) | Total (N=2051) | P-value |
|---|---|---|---|---|
| Sex | ||||
| N | 1006 | 1045 | 2051 | 0.808a |
| Male, n (%) | 557 (55.4) | 573 (54.8) | 1130 (55.1) | |
| Age at symptom onset, years | ||||
| N | 644 | 716 | 1360 | <0.001b |
| Mean (SD) | 25.3 (18.9) | 19.2 (16.2) | 22.1 (17.8) | |
| Age at diagnosis, years | ||||
| N | 1006 | 1045 | 2051 | <0.001b |
| Mean (SD) | 40.6 (17.4) | 29.6 (17.1) | 35.0 (18.1) | |
| Age at agalsidase alfa initiation, years | ||||
| N | 1006 | 1045 | 2051 | 0.261b |
| Mean (SD) | 41.5 (17.4) | 40.6 (16.2) | 41.0 (16.8) | |
| Time from symptom onset to diagnosis, years | ||||
| N | 644 | 716 | 1360 | <0.001b |
| Mean (SD) | 13.9 (14.0) | 9.7 (12.7) | 11.7 (13.5) | |
| Time from diagnosis to agalsidase alfa initiation, years | ||||
| N | 1006 | 1045 | 2051 | <0.001b |
| Mean (SD) | 0.9 (0.5) | 11.0 (9.8) | 6.1 (8.7) | |
| Time receiving agalsidase alfa treatment, years | ||||
| N | 1006 | 1045 | 2051 | <0.001b |
| Mean (SD) | 6.0 (4.2) | 6.7 (4.7) | 6.4 (4.4) | |
| eGFR at baseline, mL/min/1.73 min2 | ||||
| N | 651 | 652 | 1303 | 0.256b |
| Mean (SD) | 94.4 (31.1) | 96.3 (30.7) | 95.3 (30.9) | |
| LVMI at baseline, g/m2.7 | ||||
| N | 352 | 346 | 698 | 0.762b |
| Mean (SD) | 54.0 (21.3) | 53.5 (21.5) | 53.7 (21.4) | |
| History of cardiovascular event | ||||
| N | 1006 | 1045 | 2051 | 0.002a |
| Yes, n (%) | 385 (38.3) | 469 (44.9) | 854 (41.6) | |
| History of renal event | ||||
| N | 1006 | 1045 | 2051 | 0.001a |
| Yes, n (%) | 273 (27.1) | 353 (33.8) | 626 (30.5) | |
| Family history of Fabry disease | ||||
| N | 902 | 886 | 1788 | <0.001a |
| Yes, n (%) | 778 (86.3) | 815 (92.0) | 1593 (89.1) | |
| Mutation classificationc | ||||
| N | 224 | 221 | 445 | <0.001a |
| Classical, n (%) | 152 (67.9) | 197 (89.1) | 349 (78.4) | |
| Late-onset, n (%) | 72 (32.1) | 24 (10.9) | 96 (21.6) |
Notes: The prompt treatment cohort initiated agalsidase alfa <24 months after diagnosis; the delayed treatment cohort initiated agalsidase alfa ≥24 months after diagnosis. Baseline was defined as the date closest to agalsidase alfa initiation within a window of –6 months to +3 months. eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration. Values were restricted to 10–160 mL/min/1.73 m2 for eGFR and 10–120 g/m2.7 for LVMI; values outside these ranges were considered missing. History of cardiovascular and/or renal events refers to respective events that occurred prior to or at the date of agalsidase alfa initiation. P-values were derived from aChi-square test and bt-test. cMutation classification was reported for patients both with genetic informed consent form and who provided genetic data.
Abbreviations: eGFR, estimated glomerular filtration rate; LVMI, left ventricular mass index.