Table 1.
Baseline characteristics of the study population
| CBDR (N=88) |
Placebo (N=81) |
Total (N=169) |
|
|---|---|---|---|
| Weight stratum | |||
| ≤65 kg | 24 (27%) | 23 (28%) | 47 (28%) |
| >65–80 kg | 14 (16%) | 10 (12%) | 24 (14%) |
| >80 kg | 50 (57%) | 48 (59%) | 98 (58%) |
| Demographics | |||
| Age (years) | 13.8 (2.9) | 13.6 (2.5) | 13.7 (2.7) |
| Male | 63 (72%) | 56 (69%) | 119 (70%) |
| Race | |||
| American Indian/Alaska Native | 5 (6%) | 6 (7%) | 11 (7%) |
| Asian | 0 (0%) | 2 (2%) | 2 (1%) |
| Black or African-American | 3 (3%) | 3 (4%) | 6 (4%) |
| White | 56 (64%) | 46 (57%) | 102 (60%) |
| More than one race | 3 (3%) | 1 (1%) | 4 (2%) |
| Refusal/not stated* | 21 (24%) | 23 (28%) | 44 (26%) |
| Hispanic ethnicity | 66 (75%) | 58 (72%) | 124 (73%) |
| Self-Reported Pediatric QOL† | |||
| Physical health | 81 (15) | 82 (19) | 81 (17) |
| Psychosocial health | 75 (16) | 77 (16) | 76 (16) |
| Parent/guardian-Reported Pediatric QOL† | |||
| Physical health | 68 (21) | 69 (24) | 68 (23) |
| Psychosocial health | 67 (19) | 68 (18) | 68 (19) |
| Liver enzymes | |||
| Alanine aminotransferase (U/L) | |||
| Median (IQR) | 93 (67–175) | 80 (61–120) | 87 (62–151) |
| Mean (SD) | 140 (118) | 104 (76) | 123 (101) |
| Aspartate aminotransferase (U/L) | |||
| Median (IQR) | 55 (40–91) | 49 (38–69) | 52 (39–79) |
| Mean (SD) | 82 (71) | 59 (38) | 71 (59) |
| Alkaline phosphatase (U/L) | 224 (116) | 214 (101) | 220 (109) |
| γ-glutamyl transpeptidase (U/L) | 50 (33) | 44 (29) | 47 (31) |
| Total bilirubin (mg/dL) | 0.54 (0.34) | 0.50 (0.26) | 0.52 (0.30) |
| Lipids | |||
| Total cholesterol (mg/dL) | 165 (40) | 163 (37) | 164 (38) |
| HDL cholesterol (mg/dL) | 39 (9) | 41 (9) | 40 (9) |
| LDL cholesterol (mg/dL) | 95 (32) | 92 (31) | 94 (31) |
| Non-HDL cholesterol (mg/dL) | 126 (40) | 122 (37) | 124 (39) |
| Triglycerides (mg/dL) | 160 (81) | 157 (77) | 158 (79) |
| Metabolic factors | |||
| Weight (kg) | 85 (26) | 84 (25) | 85 (25) |
| Body-mass index (kg/m2) | 33 (7) | 32 (6) | 32 (6) |
| Body-mass index z-score | 2.2 (0.5) | 2.2 (0.4) | 2.2 (0.4) |
| Waist circumference (cm) | 104 (15) | 103 (15) | 103 (15) |
| Fasting serum glucose (mg/dL) | 87 (10) | 88 (14) | 88 (12) |
| Insulin (µU/mL)‡ | 35 (31) | 38 (34) | 36 (32) |
| HOMA-IR (glucose [mmol/L] x insulin [µU/mL]/22.5)§ |
7.7 (7.5) | 8.4 (7.7) | 8.0 (7.6) |
| Systolic blood pressure (mm Hg) | 120 (11) | 120 (12) | 120 (11) |
| Diastolic blood pressure (mm Hg) | 68 (8) | 67 (10) | 67 (9) |
| Comorbidities | |||
| Diabetes¶ | 1 (1%) | 7 (9%) | 8 (5%) |
| Hypertension | 9 (10%) | 6 (7%) | 15 (9%) |
| Hyperlipidemia | 13 (15%) | 12 (15%) | 25 (15%) |
| Liver histology findings | |||
| NAFLD activity score∥ | 4.7 (1.4) | 4.6 (1.4) | 4.7 (1.4) |
| Steatosis score | 2.3 (0.8) | 2.5 (0.7) | 2.4 (0.7) |
| Lobular inflammation score¶ | 1.8 (0.7) | 1.6 (0.7) | 1.7 (0.7) |
| Hepatocellular ballooning score | 0.6 (0.7) | 0.6 (0.8) | 0.6 (0.7) |
| Portal inflammation score** | 1.1 (0.5) | 1.1 (0.5) | 1.1 (0.5) |
| Fibrosis stage | |||
| 0 - None | 24 (27%) | 25 (31%) | 49 (29%) |
| 1a - Mild, zone 3 perisinusoidal | 9 (10%) | 7 (9%) | 16 (9%) |
| 1b - Moderate, zone 3 perisinusoidal | 6 (7%) | 5 (6%) | 11 (7%) |
| 1c - Portal/periportal only | 20 (23%) | 20 (25%) | 40 (24%) |
| 2 - Zone 3 and periportal, any combination | 9 (10%) | 13 (16%) | 22 (13%) |
| 3 - Bridging | 19 (22%) | 11 (14%) | 30 (18%) |
| 4 - Cirrhosis | 1 (1%) | 0 (0%) | 1 (1%) |
| Fibrosis stage†† | 1.3 (1.1) | 1.1 (1.0) | 1.2 (1.1) |
| Steatohepatitis | |||
| No | 25 (28%) | 19 (23%) | 44 (26%) |
| Borderline Zone 3 pattern | 16 (18%) | 10 (12%) | 26 (15%) |
| Borderline Zone 1 pattern | 23 (26%) | 29 (36%) | 52 (31%) |
| Definite | 24 (27%) | 23 (28%) | 47 (28%) |
Data are n (%) or mean (SD), unless otherwise noted.
Patients who refused or did not report race were all of Hispanic/Latino ethnicity (N=44).
Pediatric Quality of Life Inventory (version 4.0), Child and Parent Reports for Children (ages 8–12) and Teens (ages 13–18). Scores are transformed on a scale from 0 to 100, where higher scores indicate better quality of life.
One outlier with insulin of 718.7 umol/mL was excluded.
HOMA-IR=homeostasis model assessment-estimated insulin resistance.
The only statistically significant differences in baseline characteristics by treatment group were diabetes status (P=0.03) and lobular inflammation score (P=0.02).
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.
Mean fibrosis stage assessed on a scale of 0–4, with higher scores showing more severe fibrosis.