Table 1.
Baseline characteristics of the study population
| Obeticholic acid (n=141) | Placebo (n=142) | |
|---|---|---|
|
Demographics
| ||
| Age (years) | 52 (11) | 51 (12) |
| Male | 43 (30%) | 53 (37%) |
| Race | ||
| Asian | 6 (4%) | 10 (7%) |
| Black or African-American | 2 (1%) | 4 (3%) |
| White | 123 (87%) | 111 (78%) |
| Other | 10 (7%) | 17 (12%) |
| Ethnic origin | ||
| Hispanic | 22 (16%) | 21 (15%) |
|
| ||
|
SF-36 Quality of life
| ||
| Physical component summary | 45 (11) | 44 (11) |
| Mental component summary | 48 (12) | 48 (12) |
|
| ||
|
Liver enzymes
| ||
| Alanine aminotransferase (U/L) | 83 (49) | 82 (51) |
| Aspartate aminotransferase (U/L) | 64 (38) | 58 (34) |
| Alkaline phosphatase (U/L) | 82 (29) | 81 (25) |
| γ-glutamyl transpeptidase (U/L) | 78 (85) | 76 (97) |
| Total bilirubin (μmol/L) | 11·5 (5·9) | 11·3 (7·5) |
|
| ||
|
Lipids
| ||
| Total cholesterol (mmol/L) | 4·9 (1·2) | 4·8 (1·2) |
| HDL cholesterol (mmol/L) | 1·1 (0·3) | 1·1 (0·4) |
| LDL cholesterol (mmol/L) | 2·9 (1·0) | 2·9 (1·1) |
| Trigylcerides (mmol/L) | 2·2 (1·5) | 2·0 (1·7) |
|
| ||
|
Haematology
| ||
| Haemoglobin (g/L) | 140 (15) | 140 (14) |
| Haematocrit (proportion of 1·0) | 0·41 (0·04) | 0·41 (0·04) |
| Mean corpuscular volume (fL) | 88·7 (4·8) | 89·0 (5·3) |
| White blood cell count (×109 per L) | 7·3 (1·9) | 6·9 (2·3) |
| Platelet count (×109 per L) | 237 (59) | 237 (65) |
|
| ||
|
Chemistries
| ||
| Bicarbonate (mmol/L) | 25·9 (2·5) | 26·2 (2·6) |
| Calcium (mmol/L) | 2·4 (0·1) | 2·4 (0·1) |
| Phosphate (mmol/L) | 1·1 (0·2) | 1·1 (0·2) |
| Creatinine (μmol/L) | 71 (18) | 70 (16) |
| Uric acid (μmol/L) | 375 (89) | 366 (86) |
| Albumin (g/L) | 43 (4) | 43 (4) |
| Total protein (g/L) | 73 (5) | 74 (5) |
|
| ||
|
Other laboratory results
| ||
| Prothrombin time (s) | 11·7 (2·1) | 11·7 (2·2) |
| International normalised ratio | 1·01 (0·08) | 1·00 (0·07) |
|
| ||
|
Metabolic factors
| ||
| Fasting serum glucose (mmol/L) | 6·5 (1·8) | 6·4 (2·2) |
| Insulin (pmol/L) | 201 (226) | 138 (129) |
| HOMA-IR (glucose [mmol/L] × insulin [pmol/L]/22·5) | 61 (74) | 40 (42) |
| Glycated haemoglobin A1c (mmol/mol) | 48 (12) | 47 (11) |
| Weight (kg) | 100 (23) | 96 (18) |
| Body-mass index (kg/m2) | 35 (7) | 34 (6) |
| Waist circumference (cm) | 112 (15) | 109 (14) |
| Waist-to-hip ratio | 0·96 (0·07) | 0·95 (0·09) |
| Systolic blood pressure (mm Hg) | 132 (17) | 132 (15) |
| Diastolic blood pressure (mm Hg) | 77 (11) | 78 (10) |
|
| ||
|
Comorbidities
| ||
| Hyperlipidaemia* | 87 (62%) | 86 (61%) |
| Hypertension | 87 (62%) | 85 (60%) |
| Cardiovascular disease | 7 (5%) | 8 (6%) |
| Diabetes | 75 (53%) | 74 (52%) |
|
| ||
|
Concomitant medications in the past 6 months
| ||
| Antilipidaemic | 72 (51%) | 64 (45%) |
| Cardiovascular | 97 (69%) | 92 (65%) |
| Antidiabetic | 67 (48%) | 73 (51%) |
| Metformin | 55 (39%) | 62 (44%) |
| Pioglitazone | 1 (1%) | 6 (4%) |
| Vitamin E | 29 (21%) | 32 (23%) |
| Thiazolidinedione | 3 (2%) | 5 (4%) |
| Aspirin (81 mg) | 37 (26%) | 33 (23%) |
|
| ||
|
Liver histology findings
| ||
| Definite steatohepatitis | 114 (81%) | 111 (79%) |
| Fibrosis stage† | 1·9 (1·1) | 1·8 (1·0) |
| Total NAFLD activity score‡ | 5·3 (1·3) | 5·1 (1·3) |
| Hepatocellular ballooning score | 1·4 (0·7) | 1·3 (0·7) |
| Steatosis score | 2·1 (0·8) | 2·0 (0·8) |
| Lobular inflammation score | 1·8 (0·7) | 1·8 (0·7) |
| Portal inflammation score§ | 1·2 (0·6) | 1·1 (0·6) |
| Biopsy length (mm) | 21 (10) | 21 (10) |
Data are n (%) or mean (SD). HOMA-IR=homoeostasis model assessment–estimated insulin resistance.
History of cholesterol or triglyceride elevations as determined by the site investigator.
Fibrosis was assessed on a scale of 0–4, with higher scores showing more severe fibrosis.
Total non-alcoholic fatty liver disease (NAFLD) activity was assessed on a scale of 0–8, with higher scores indicating 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.