Table 1.
Therapeutic agents that have shown benefits in NASH
| Therapeutic agent | Indication | Summary of benefits | Adverse effects and concerns |
|---|---|---|---|
| Metformin50,51 | No current indication | Improved aminotransferases, BMI, and insulin resistance; possible positive effect on ballooning in those who lose weight | AEs: diarrhea, nausea, and vomiting Concerns: Contraindicated in patients with renal failure |
| Orlistat53,53 | No current indication | Two trials; one showed improvement in ALT, BMI, and hepatic steatosis based on ultrasound; the other failed to show this | AEs: fatty diarrhea, abdominal pain–discomfort flatulence and fecal urgency |
| Pentoxifylline57–59 | No current indication | One trial showed improvements in steatosis and lobular inflammation, but not in ballooning; another showed improvements in steatosis and ballooning | AEs: nausea, vomiting |
| Ursodeoxycholic acid54,55 | No current indication | Borderline benefits in aminotransferases; only one trial showed histological benefit | AEs: GI upset, headache, and dizziness |
| Pioglitazone56 | Biopsy proven NASH with or without DM | Improved histology of NASH; one trial showed significant reductions in steatosis, lobular inflammation, and aminotransferases | AEs: weight gain, GI upset, edema, and fatigue Concerns: contraindicated in patients with bladder cancer and postmenopausal women with increase risk of fracture, long-term efficacy, and safety |
| Vitamin E56 | Biopsy proven NASH without DM and without cirrhosis | Improved histology of NASH; one trial showed significant reductions in steatosis, lobular inflammation, and aminotransferases, and significant improvement in ballooning | Concerns: Long-term efficacy and safety, lack of studies in patients with DM, cirrhosis, and posttransplant |
| Obeticholic acid63 | (Not currently available) Biopsy proven NASH without cirrhosis | Improved histology of NASH | AEs: itching (23%), increased total cholesterol and LDL cholesterol, and a modest decrease in HDL cholesterol Concerns: Dyslipidemia, long-term efficacy and safety, lack of studies in patients with cirrhosis, and posttransplant |
Note: BMI, body mass index; ALT, alanine aminotransferase; AEs, adverse effects; DM, diabetes mellitus; GI, gastrointestinal; NASH, nonalcoholic steatohepatitis; LDL, low-density lipoprotein; HDL, high-density lipoprotein.