Table 4.
Efficacy endpoints
| Outcome | Pioglitazone, mean (95% CI) | Placebo, mean (95% CI) | Mean difference (95% CI) between periods | P-value |
|---|---|---|---|---|
| TKV (% change) | 4.35 (0.84–7.86) | 7.85 (3.60–12.11) | −3.5 (−8.39–1.38) | 0.15 |
| Kidney cyst volume (% change) | 5.64 (1.48–9.79) | 7.82 (3.57–12.08) | −2.19 (07.68–3.3) | 0.41 |
| Kidney cyst index (% change) | 571 (490–652) | 552 (475–629) | −25 (121–70) | 0.58 |
| Liver volume (% change) | 0.31 (−6.12 to 6.73) | −2.46 (−8.27–3.36) | 2.7 (−6.42–11.94) | 0.53 |
| Liver cysts volume (% change) | 30.4 (7.8–53.1) | 23.7 (11.2–36.26) | 6.69 (−19.7–33.1) | 0.59 |
| Liver cysts index (% change) | 27.1 (0.52–53.73) | 21.29 (8.0–34.6) | 5.83 (−20.5–32.1) | 0.63 |
| Pain score AUC (scale 1–10) | 69.9 (32.8–107.1) | 46.9 (20.3–63.2) | 21.6 (−15.45–58.7) | 0.19 |
| eGFR AUC (CKD-EPI) (mL/min/m2) | 956 (805–1108) | 975 (795–1155) | −103 (295–89) | 0.27 |
| eGFR (CKD-EPI) (mL/min/m2), mean (95% CI) | 75.5 (61.99–89.1) | 78.1 (64.3–91.9) | −2.59 (6.24–1.05) | 0.15 |
| MAC ratio AUC (μg/mg) | 37 (17–56) | 38 (24–52) | −03.8 (−22–14) | 0.66 |
| MAC ratio (μg/g), mean (95% CI) | 0.16 (0.12–0.21) | 0.24 (0.13–0.35) | −0.08 (−0.16–0.01) | 0.09 |
| Systolic BP AUC (mmHg) | 1610 (1577–1643) | 1648 (1579–1727) | −125 (−319–70) | 0.19 |
| Systolic BP (mmHg), mean (95% CI) | 127 (124–130) | 129 (124–134) | −2.07 (7.1–3.0) | 0.40 |
| Diastolic BP AUC (mmHg) | 1053 (1015–1091) | 1094 (1052–1134) | −95 (−216–26) | 0.12 |
| Diastolic BP (mmHg), mean (95% CI) | 83 (81–86) | 86 (82–89) | −2.4 (−5.1–0.3) | 0.08 |
| Mean arterial BP (mmHg), mean (95% CI) | 97.9 (95.2–100.5) | 100.2 (96.3–104.1) | −2.29 (−5.5–1.52) | 0.1 |
The 15 subjects who completed both periods are included in the comparison. The mean difference (paired t-test) is the difference between pioglitazone minus placebo for treatment; a negative number represents a lower value with pioglitazone.