The effect of ursodesoxycholic acid on duodenal adenomas in familial adenomatous polyposis: a prospective randomized placebo-control trial

Colorectal Dis. 2012 Jul;14(7):854-60. doi: 10.1111/j.1463-1318.2011.02816.x.

Abstract

Aim: Duodenal adenomas occur in about 90% of patients with familial adenomatous polyposis (FAP) and are the second cause of death of patients who have had a prophylactic proctocolectomy. Studies suggest that biliary acids have a role in the development of duodenal adenomas. The aim of this study was to evaluate the impact of ursodesoxycholic acid (UDCA) on duodenal adenoma formation in patients with FAP.

Method: A randomized, double-blinded, placebo-controlled study was carried out of 71 patients (20-65 years) who already had a restorative proctocolectomy. Subjects received either 10 mg/kg of UDCA orally per day or a placebo tablet for 24 months. The Spigelman severity score was determined after duodenal axial and lateral view endoscopy at 12 and 24 months.

Results: At 2 years 55 patients had completed the entire period of treatment. At the end of the follow-up period, nine (25%) patients in the UDCA group and seven (20%) in the placebo group had a decrease in the Spigelman score (P = 0.6142). Patients receiving UDCA had no side-effects (0%) compared with four (14%) in the placebo group (P = 0.0392).

Conclusion: UDCA had no effect on the development of duodenal adenomas in FAP patients (NCT: 00134758).

Trial registration: ClinicalTrials.gov NCT00134758.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenoma / complications
  • Adenoma / pathology
  • Adenoma / prevention & control*
  • Adenomatous Polyposis Coli / complications*
  • Adult
  • Aged
  • Analysis of Variance
  • Chi-Square Distribution
  • Cholagogues and Choleretics / adverse effects
  • Cholagogues and Choleretics / therapeutic use*
  • Double-Blind Method
  • Duodenal Neoplasms / complications
  • Duodenal Neoplasms / pathology
  • Duodenal Neoplasms / prevention & control*
  • Female
  • Humans
  • Male
  • Middle Aged
  • Proctocolectomy, Restorative
  • Severity of Illness Index
  • Statistics, Nonparametric
  • Treatment Failure
  • Ursodeoxycholic Acid / adverse effects
  • Ursodeoxycholic Acid / therapeutic use*
  • Young Adult

Substances

  • Cholagogues and Choleretics
  • Ursodeoxycholic Acid

Associated data

  • ClinicalTrials.gov/NCT00134758