Table 1.
Baseline characteristics of CyNCh patients with baseline and end of treatment biopsies
| Histological Improvement | ||||
|---|---|---|---|---|
| Improved (N=43) |
Not improved (N=103) |
Total (N=146) |
P | |
| Demographics | ||||
| Age (years) | 13.2 (2.6) | 13.7 (2.6) | 13.6 (2.6) | 0.25 |
| Male | 30 (70%) | 73 (71%) | 103 (71%) | 1.00 |
| Race | 0.82 | |||
| White | 26 (60%) | 60 (58%) | 86 (59%) | |
| Non-white | 5 (12%) | 17 (17%) | 22 (15%) | |
| Refusal/not stated | 12 (28%) | 26 (25%) | 38 (26%) | |
| Hispanic ethnicity | 35 (81%) | 73 (71%) | 108 (74%) | 0.22 |
|
Treatment group CBDR Placebo |
25 (58%) 18 (42%) |
46 (45%) 57 (55%) |
71 (49%) 75 (51%) |
0.15 |
|
Weight stratum
≤65 kg >65–80 kg >80 kg |
15 (35%) 2 (5%) 26 (60%) |
27 (26%) 16 (16%) 60 (58%) |
42 (29%) 18 (12%) 86 (59%) |
0.15 |
| Liver chemistries – median (IQR) | ||||
| Alanine aminotransferase (U/L) | 94 (57–179) | 84 (62–130) | 86 (62–140) | 0.53 |
| Aspartate aminotransferase (U/L) | 53 (39–92) | 50 (38–74) | 51 (38–79) | 0.67 |
| γ-glutamyl transpeptidase (U/L) | 38 (27–67) | 34 (26–54) | 35 (27–62) | 0.30 |
| Metabolic factors | ||||
| Weight (kg) | 81 (23) | 86 (27) | 84 (26) | 0.28 |
| Body-mass index (kg/m2) | 31 (6) | 32 (7) | 32 (6) | 0.23 |
| Body-mass index z-score | 2.1 (0.4) | 2.2 (0.5) | 2.2 (0.4) | 0.40 |
| Liver histology findings | ||||
| NAFLD activity score* | 5.4 (1.2) | 4.4 (1.3) | 4.7 (1.4) | <0.001 |
| Steatosis score | 2.4 (0.8) | 2.4 (0.7) | 2.4 (0.7) | 0.92 |
| Lobular inflammation score | 2.1 (0.7) | 1.5 (0.6) | 1.7 (0.7) | <0.001 |
| Hepatocellular ballooning score | 0.9 (0.8) | 0.5 (0.7) | 0.6 (0.7) | 0.007 |
| Portal inflammation score† | 1.1 (0.5) | 1.2 (0.5) | 1.1 (0.5) | 0.16 |
| Fibrosis stage | 0.18 | |||
| 0, none | 8 (19%) | 37 (36%) | 45 (31%) | |
| 1a, mild, zone 3 perisinusoidal | 3 (7%) | 8 (8%) | 11 (8%) | |
| 1b, moderate, zone 3 perisinusoidal | 4 (9%) | 5 (5%) | 9 (6%) | |
| 1c, portal/periportal only | 12 (28%) | 25 (24%) | 37 (25%) | |
| 2, zone 3 and periportal, any combination | 8 (19%) | 10 (10%) | 18 (12%) | |
| 3, bridging | 7 (16%) | 18 (17%) | 25 (17%) | |
| 4, cirrhosis | 1 (2%) | 0 (0%) | 1 (1%) | |
| Steatohepatitis | 0.03 | |||
| No | 6 (14%) | 34 (33%) | 40 (27%) | |
| Borderline Zone 3 pattern | 8 (19%) | 14 (14%) | 22 (15%) | |
| Borderline Zone 1 pattern | 12 (28%) | 34 (33%) | 46 (32%) | |
| Definite | 17 (40%) | 21 (20%) | 38 (26%) | |
Data are n (%) or mean (SD), unless otherwise noted.
*
NAFLD activity score was assessed on a scale of 0–8, with higher scores showing more severe disease (the components of this measure are steatosis [assessed on a scale of 0–3], lobular inflammation [assessed on a scale of 0–3], and hepatocellular ballooning [assessed on a scale of 0–2]).
†
Portal inflammation was assessed on a scale of 0–2, with higher scores showing more severe inflammation.