The Journal of clinical endocrinology and metabolism · 2025
Efficacy and Safety of Obeticholic Acid for Treating Hepatic Steatosis in Patients With Familial Partial Lipodystrophy.
СтатьяРандомизированное исследованиеНазвание и аннотация: Перевод в очереди
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Garg Abhimanyu, Vasandani Chandna, Li Xilong, Quittner Claudia, Yokoo Takeshi
Аннотация
CONTEXT: Patients with familial partial lipodystrophy (FPLD) have increased risk of hepatic steatosis and its complications, for which there is no approved therapy.
OBJECTIVE: This work aimed to investigate the efficacy and safety of obeticholic acid (OCA), a farnesoid X receptor agonist, for reducing hepatic steatosis in patients with FPLD.
METHODS: A randomized, double-blind, placebo-controlled, crossover trial was conducted at an academic referral center. Ten women (age 19-60 years) with the Dunnigan variety of FPLD (FPLD2), harboring pathogenic heterozygous variants in the lamin A/C gene and hepatic steatosis (liver fat >5.6% by proton-density fat fraction mapping by magnetic resonance imaging), were included. Intervention included OCA 25 mg daily vs matched placebo for 4 months each with a 4-month washout period in between. The primary end point variable was liver fat. Secondary end point variables were serum triglycerides (TGs) and transaminase levels.
RESULTS: All patients completed the trial. OCA therapy caused significant (39.6%) reduction in liver fat as compared to placebo (median liver fat [minimum-maximum]; 6.4% [2.4%-18.0%] vs 10.6% [3.4%-29.3%], respectively; P value for treatment × month interaction = .03). There were no significant differences in serum TGs or transaminase levels during OCA and placebo therapy. Overall, OCA was well tolerated except for itching in 4 patients compared to 2 on placebo. OCA, as compared to placebo, caused 24% increase in serum low-density lipoprotein cholesterol (mean 129 mg/dL vs 104 mg/dL, respectively; P = .0016).
CONCLUSION: OCA is safe and effective in lowering hepatic TG levels in patients with FPLD2.
OBJECTIVE: This work aimed to investigate the efficacy and safety of obeticholic acid (OCA), a farnesoid X receptor agonist, for reducing hepatic steatosis in patients with FPLD.
METHODS: A randomized, double-blind, placebo-controlled, crossover trial was conducted at an academic referral center. Ten women (age 19-60 years) with the Dunnigan variety of FPLD (FPLD2), harboring pathogenic heterozygous variants in the lamin A/C gene and hepatic steatosis (liver fat >5.6% by proton-density fat fraction mapping by magnetic resonance imaging), were included. Intervention included OCA 25 mg daily vs matched placebo for 4 months each with a 4-month washout period in between. The primary end point variable was liver fat. Secondary end point variables were serum triglycerides (TGs) and transaminase levels.
RESULTS: All patients completed the trial. OCA therapy caused significant (39.6%) reduction in liver fat as compared to placebo (median liver fat [minimum-maximum]; 6.4% [2.4%-18.0%] vs 10.6% [3.4%-29.3%], respectively; P value for treatment × month interaction = .03). There were no significant differences in serum TGs or transaminase levels during OCA and placebo therapy. Overall, OCA was well tolerated except for itching in 4 patients compared to 2 on placebo. OCA, as compared to placebo, caused 24% increase in serum low-density lipoprotein cholesterol (mean 129 mg/dL vs 104 mg/dL, respectively; P = .0016).
CONCLUSION: OCA is safe and effective in lowering hepatic TG levels in patients with FPLD2.