
A recent proof-of-concept study investigated the use of obeticholic acid (OCA), a selective agonist of the farnesoid-X receptor (FXR), in the treatment of low phospholipid-associated cholelithiasis (LPAC) syndrome. LPAC is a rare genetic cause of hepatolithiasis, with an ABCB4 gene variant identified in half of the affected patients. While ursodeoxycholic acid (UDCA) is the approved treatment, it sometimes fails to prevent symptoms and complications. This retrospective study examined five LPAC patients treated with OCA alongside or independently of UDCA. Encouragingly, four patients showed symptom improvement, indicating OCA’s potential efficacy.
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