Table 1.
Patient Baseline Characteristics by Treatment Cohort in the Full Analysis Set
| Characteristic | Tolvaptan (n = 149) | Historical Controls (n = 959) | Standardized Mean Difference |
|---|---|---|---|
| Age (y), n | 149 | 959 | |
| Mean ± SD | 43.1 ± 11.7 | 42.5 ± 12.0 | 0.05 |
| Range | 19.4-77.0 | 18.1-70.9 | |
| Sex, n (%) | 149 | 959 | |
| Female | 60 (40.3) | 494 (51.5) | −0.23 |
| Race/ethnicity, n (%) | 149 | 957 | |
| White | 93 (62.4) | 814 (85.1) | −0.53 |
| African American | 22 (14.8) | 48 (5.0) | 0.33 |
| Hispanic | 12 (8.1) | 61 (6.4) | 0.06 |
| Asian | 12 (8.1) | 15 (1.6) | 0.31 |
| Other | 10 (6.7) | 19 (2.0) | 0.23 |
| Age at ADPKD diagnosis (y), n | 149 | 957 | |
| Mean ± SD | 35.0 ± 12.7 | 30.3 ± 12.6 | 0.36 |
| Range | 2.7-77.0 | 0.0-68.3 | |
| CKD stage,a n (%) | 148 | 947 | |
| G1 | 15 (10.1) | 263 (27.8) | −0.46 |
| G2 | 38 (25.7) | 301 (31.8) | −0.14 |
| G3a | 53 (35.8) | 129 (13.6) | 0.53 |
| G3b | 22 (14.9) | 117 (12.4) | 0.07 |
| G4 | 20 (13.5) | 104 (11.0) | 0.08 |
| G5 | 0 (0.0) | 33 (3.5) | −0.27 |
| Baseline eGFRb in mL/min/1.73 m2, n | 148 | 947 | |
| Mean ± SD | 56.3 ± 26.7 | 68.6 ± 31.9 | −0.42 |
| Range | 8.5-142.1 | 4.3-146.6 | |
| History of nephrolithiasis, n (%) | 149 | 959 | |
| Yes | 14 (9.4) | 80 (8.3) | 0.04 |
| History of hypertension, n (%) | 149 | 959 | |
| Yes | 104 (69.8) | 819 (85.4) | −0.38 |
Abbreviations: ADPKD, autosomal dominant polycystic kidney disease; CKD, chronic kidney disease; eGFR, estimated glomerular filtration rate; SD, standard deviation.
a
Stage G1, ≥90 mL/min/1.73 m2; stage G2, 60 to < 90 mL/min/1.73 m2; stage G3a, 45 to < 60 mL/min/1.73 m2; stage G3b, 30 to < 45 mL/min/1.73 m2; stage G4, 15 to < 30 mL/min/1.73 m2; stage G5, < 15 mL/min/1.73 m2.
b
Because there were no baseline eGFR assessments for the tolvaptan patients, eGFR measured at month 1 (30-60 days after tolvaptan initiation) was considered as the baseline value.