Table 3.
Change from baseline to week 16 in non-invasive markers of fibrosis, ELF score and its components, and markers of liver injury.
| Placebo (n = 10) |
Efruxifermin (n = 20) |
p vs. placebo | |||
|---|---|---|---|---|---|
| Value | p vs. baseline | Value | p vs. baseline | ||
| Liver stiffness (kPa) | |||||
| Baseline mean (SD) | 25.8 (13.2) | 22.6 (10.9) | |||
| LS mean absolute CFB (95% CI)1 | −1.9 (−7.0, 3.2) | 0.4519 | −5.7 (−9.4, −2.0) | 0.0036 | 0.2226 |
| LS mean percent CFB (95% CI)1 | −7.6 (−25.6, 10.5) | 0.3970 | -24.4 (−37.5, −11.4) | 0.0007 | 0.1326 |
| Pro-C3 (μg/L) | |||||
| Baseline mean (SD) | 22.6 (11.8) | 28.9 (30.8) | |||
| LS mean absolute CFB (95% CI)1 | −3.4 (−6.9, 0.2) | 0.0620 | −9.0 (−11.5, −6.5) | <0.0001 | 0.0130 |
| LS mean percent CFB (95% CI)1 | 3.9 (−17.7, 25.4) | 0.7168 | −16.2 (−31.4, −1.0) | 0.0379 | 0.1309 |
| ELF score | |||||
| Baseline, median | 9.5 | 10.4 | |||
| Median absolute CFB (IQR)2 | 0.4 (0.0, 0.6) | NC | −0.4 (−0.9, −0.0) | NC | |
| Median difference vs. placebo2 | −0.8 (−1.3, −0.4) | 0.0036 | |||
| Hyaluronic acid, μg/L | |||||
| Baseline, median | 63.8 | 116.9 | |||
| Median absolute CFB (IQR)2 | 16.4 (1.8, 69.9) | NC | −19.4 (−58.3, 6.4) | NC | |
| Median difference vs. placebo2 | −59.3 (−86.6, −11.1) | 0.0139 | |||
| P3NP, μg/L | |||||
| Baseline, median | 8.2 | 11.6 | |||
| Median absolute CFB (IQR)2 | 1.8 (0.3, 3.4) | NC | −3.2 (−5.7, −1.2) | NC | |
| Median difference vs. placebo2 | −5.9 (−9.0, −2.7) | 0.0019 | |||
| TIMP-1, μg/L | |||||
| Baseline, median | 230.2 | 279.5 | |||
| Median absolute CFB (IQR)2 | 17.6 (−14.3, 43.0) | NC | −35.7 (−74.1, 3.6) | NC | |
| Median difference vs. placebo2 | −52.3 (−112.9, −6.9) | 0.0415 | |||
| ALT, U/L | |||||
| Baseline mean (SD) | 32.7 (20.0) | 31.8 (17.2) | |||
| LS mean absolute CFB (95% CI)1 | −1.3 (−6.7, 4.1) | 0.6289 | −10.3 (−14.3, −6.4) | <0.0001 | 0.0098 |
| LS mean percent CFB (95% CI)1 | 3.0 (−14.5, 20.4) | 0.7301 | −22.1 (−34.8, −9.5) | 0.0014 | 0.0244 |
| AST, U/L | |||||
| Baseline mean (SD) | 28.9 (21.1) | 31.6 (14.0) | |||
| LS mean absolute CFB (95% CI)1 | −4.5 (−9.0, 0.1) | 0.0566 | −9.6 (−12.9, −6.2) | <.0001 | 0.0752 |
| LS mean percent CFB (95% CI)1 | −5.5 (−17.9, 6.8) | 0.3654 | −25.8 (−34.8, −16.9) | <.0001 | 0.0112 |
| GGT, U/L | |||||
| Baseline mean (SD) | 46.7 (17.7) | 76.2 (40.2) | |||
| LS mean absolute CFB (95% CI)1 | 21.3 (−18.4, 61.0) | 0.2808 | −14.5 (−42.6, 13.6) | 0.2996 | 0.1569 |
| LS mean percent CFB (95% CI)1 | 42.2 (−16.1, 100.5) | 0.1487 | −23.0 (−64.2, 18.3) | 0.2624 | 0.0817 |
| ALP, U/L | |||||
| Baseline mean (SD) | 67.2 (19.0) | 77.9 (24.6) | |||
| LS mean absolute CFB (95% CI)1 | 4.6 (−9.2, 18.4) | 0.4988 | −2.9 (−12.9, 7.0) | 0.5468 | 0.3748 |
| LS mean percent CFB (95% CI)1 | 6.5 (−10.6, 23.7) | 0.4412 | −2.4 (−14.7, 10.0) | 0.6956 | 0.3999 |
| Urate, mg/dl | |||||
| Baseline mean (SD) | 5.87 (1.41) | 6.04 (1.22) | |||
| LS mean absolute CFB (95% CI)3 | −0.34 (−0.82, 0.14) | 0.1526 | -0.98 (−1.32, −0.63) | <.0001 | 0.0357 |
| LS mean percent CFB (95% CI)3 | −4.56 (−11.95, 2.83) | 0.2164 | −16.19 (−21.52, −10.86) | <.0001 | 0.0143 |
ALP, alkaline phosphatase; ALT, alanine aminotransferase; AST, aspartate aminotransferase; CFB, change from baseline; ELF, enhanced liver fibrosis; GGT, gamma-glutamyltransferase; NC, not calculated; LS, least squares; Pro-C3, N-terminal type III collagen propeptide; P3NP, procollagen type III N-terminal propeptide; TIMP-1, tissue inhibitor of metalloproteinase-1.
1
p values from ANCOVA.
2
p values from Wilcoxon rank-sum test.
3
p values from mixed-effects model of repeated measure.