In patients with IBD and PSC, does prophylactic ursodeoxycholic acid (UDCA) reduce the risk of progression to cholangiocarcinoma compared to no UDCA
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There is no evidence that prophylactic UDCA reduces progression to cholangiocarcinoma in PSC-IBD: a randomized trial of moderate-dose UDCA (17–23 mg/kg/day) found no significant difference in cholangiocarcinoma development between UDCA and placebo groups 1,2. In that trial, cholangiocarcinoma deaths occurred in both arms (two in the UDCA group, two in the placebo group) 3. 1–4
What is known: - A randomized trial of higher-dose UDCA (17–23 mg/kg/day) in PSC found no significant difference in death, need for transplant, or cholangiocarcinoma development versus placebo. 1 - There were no significant differences in cholangiocarcinoma incidence between the high-dose UDCA (17–23 mg/kg/day) and placebo groups. 2 - During the 5-year trial, cholangiocarcinoma occurred in both arms, two UDCA-group deaths and two placebo-group deaths from cholangiocarcinoma. 3 - No UDCA dose has shown convincing benefit in preventing PSC progression or altering its natural history; a highest-dose trial was halted early for increased adverse events. 1,4
What is unknown / caveats: - Most excerpts address colorectal neoplasia, not cholangiocarcinoma, as the primary endpoint - Cholangiocarcinoma cases were few, so the trial was not powered to detect a difference in this outcome - The available data specifically on cholangiocarcinoma are limited to small numbers of events within trials designed for other endpoints.
## References
1. Navaneethan U, Shen B. Hepatopancreatobiliary manifestations and complications associated with inflammatory bowel disease. Inflamm Bowel Dis. 2010;16(9):1598-619. PMID: 20198712.
2. Nakazawa T, Naitoh I, Hayashi K, Sano H, Miyabe K, Shimizu S, Joh T. Inflammatory bowel disease of primary sclerosing cholangitis: a distinct entity?. World J Gastroenterol. 2014;20:3245-54. PMID: 24696608.
3. Lindström L, Boberg KM, Wikman O, Friis-Liby I, Hultcrantz R, Prytz H, Sandberg-Gertzén H, Sangfelt P, Rydning A, Folvik G, Gangsøy-Kristiansen M, Danielsson A, Bergquist A. High dose ursodeoxycholic acid in primary sclerosing cholangitis does not prevent colorectal neoplasia. Aliment Pharmacol Ther. 2012. PMID: 22221173.
4. Kim YS, Hurley EH, Park Y, Ko S. Treatment of primary sclerosing cholangitis combined with inflammatory bowel disease. Intest Res. 2023. PMID: 37519211.
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⚠ Required context not in this corpus (severe). The high-dose UDCA (28-30 mg/kg/d) trial in PSC was TERMINATED at 6 years for futility, and reported HARM: a 2.3x higher risk of the primary endpoint, 2.1x higher death/transplantation/minimal listing criteria (P = 0.04), and serious adverse events in 63% vs 37% (Lindor 2009, PMID 19585548; Imam 2011, PMID 21957881).
_Why this matters:_ An answer that calls high-dose UDCA merely 'unproven', or leaves open that a higher dose might help, can push a clinician toward a dose an RCT stopped for harm.
_Added by the landmark interlock. This corpus is surgical in scope and cannot admit the source paper (PMID 19585548, 21957881). Not generated by the RAG._
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_Draft, generated by the IBDology RAG and not yet clinician-reviewed. Answers are grounded in the retrieved literature listed above; a high faithfulness score means the answer matches its sources, not that the sources are correct._
Reviewer notes