Efficacy and Safety of Obeticholic Acid for Treating Hepatic Steatosis in Patients With Familial Partial Lipodystrophy
Authors' conclusion
OCA is safe and effective in lowering hepatic TG levels in patients with FPLD2.
Study context
- Population
- Women aged 19–60 years with FPLD (FPLD2) (pathogenic heterozygous lamin A/C variants) and hepatic steatosis (liver fat >5.6% by MRI proton-density fat fraction)all completed
- Participants
- 10 women
- Comparison
- Obeticholic acid (OCA) 25 mg daily vs matched placebo
- Primary endpoint
- Liver fat
Methodological detailsdouble-blind, placebo-controlled, crossover, single academic referral center
- Double-blind
- Placebo-controlled
- Crossover
- Single academic referral center
Primary result
As reported in the abstract
Reduction in liver fat
39.6%
P value for treatment × month interaction = .03
By group
Secondary outcomes
- No significant differences in serum TG or transaminase levels during OCA vs placebo
Safety
Serum LDL-C
increase 24% · P = 0.0016
Events
| Outcome | OCA | Placebo |
|---|---|---|
| Itching | 4 | 2 |
Itching
- OCA overall well tolerated except for itching
Original publication
Efficacy and Safety of Obeticholic Acid for Treating Hepatic Steatosis in Patients With Familial Partial Lipodystrophy. J Clin Endocrinol Metab. Published 13 March 2025. doi:10.
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