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The Following is a summary of “EFFICACY AND SAFETY OF OBETICHOLIC ACID FOR TREATING HEPATIC Steatos in Patients with Familial Partial Lipodystrophy,” Published in the March 2025 ISUE OF OF Journal of Clinical Endocrinology & Metabolism by Garg et al.
Individuals with family partial lipodystrophy (FPLD) Faced a Higher Risk of hepatic steatosis and ITS complications, with in the Approved Treatment Available.
Researchers Drive the Retrospective Study to Evaluate the Effficacy and Safety of Obeticholic Acid, A Farnesoid x Receiver Agonist, In Reducing Hepatic Steatosis in Individuals with FPLD.
They Caried Out a Randomized, Double-Blind, Placebo-Controlled, Cross-Over trial at an academic referral center. The Study Included 10 Females AGED 19–60 Years with Dunnigan Variety of Familial Partial Lipodystrophy (FPLD2), All Carrying Pathogenic Heterozygous Varies in the Lamin A/C Gene and Present with hepatic steatosis (Liver Fat> 5.6% BY Proton-Density Fat Fraction Mapping Using Magnetic Resonance Imaging). Participants Received Obeticholic Accident 25 mg Daily or Matched placebo for 4 months, separate by a 4-moonth Washout Period. The Primary Outcome was Liver Fat, While Secondary Outcomes Included Serum Triglyceride Levels and Aminotransferase Levels.
The Results Showed that obeticholic accident therapy led to 39.6% Reduction in Liver Fat Compred to Placebo (MINIMUM): 6.4% (2.4% – 18.0%) vs 10.6% (3.4% – 29.3%); P Value for Treatment x Month Interaction = 0.03). No Significant Differences Were observed in serum triglyceride or aminotransferase levels Between obeticholic accidents and placebo. OBETICHOLIC ACID WAS GENERALLY WELL TOLERATED, THOUGH ITCHING OCCURRED IN 4 INDIVIDUALS VS 2 ON PLACEBO. Compred to placebo, obeticholic accidents increased serum low-density lipoprotein cholesterol by 24% (Mean 129 mg/dl vs 104 mg/dl; P = 0.0016).
Investigators Concluded that obeticholic accidents Safely and effectively reduced hepatic triglyceride levels in patients with fpld2.
Source: academic.oup.com/jcem/advance-article-abstract/doi/10.1210/clinem/dgaf173/8075157