Table. Changes in kidney volume and renal function in five clinical studies involving 100 patients or more.
| Study | No. of treated patients | Age (years) | Baseline TKV (mL) | Baseline eGFR (mL/min/1.73 m²) | Δ TKV vs. placebo (% per year) | Δ eGFR vs. placebo (mL/min/1.73 m2 per year) | Relative stage*2 |
| Pravastatin (29) | 56 | 16 | 571 | 135*1 | –2.7(*) | 0.0 | 1 |
| Sirolimus (17) | 50 | 31 | 907 | 92 | 0.2 | 2.5 | 3 |
| HALT-PKD (30) | 274 | 37 | 1185 | 91 | –1.0(*) | 0.4 | 5 |
| TEMPO 3/4 (24) | 961 | 39 | 1705 | 81 | –2.7(*) | 1.0(*) | 8 |
| Everolimus (18) | 213 | 45 | 2028 | 53 | –2.2(*) | –2.0 | 17 |
Significantly lower TKV (*) led to a significant delay in loss of renal function (*) in the TEMPO 3/4 study only. The difference between the intervention and placebo groups was 1.0 mL/min/1.73 m² per year. The relative stage was ascertained by multiplying kidney volume (TKV in dL) by age and dividing by renal function; it illustrates the various stages of ADPKD at which the studies were conducted (1 = early stage, 17 = advanced stage). This difference should be taken into account when comparing different ADPKD studies. ADPKD: Autosomal dominant polycystic kidney disease; TKV: Total kidney volume; eGFR: Estimated glomerular filtration rate; HALT-PKD: Halt Progression of Polycystic Kidney Disease; TEMPO 3/4: Tolvaptan Efficacy and Safety in Management of ADPKD and Its Outcomes 3/4 (*): Denotes significance *1 Creatinine clearance *2 Relative stage = TKV (dL) × age (years)/eGFR (mL/min/1.73 m²)