Ursodeoxycholic acid in the treatment of primary biliary cirrhosis

Keith Lindor, E. Rolland Dickson, William P. Baldus, Roberta A. Jorgensen, Jurgen Ludwig, Paul A. Murtaugh, Jay M. Harrison, Russell H. Wiesner, Monte L. Anderson, Stephen M. Lange, Gene Lesage, Steven S. Rossi, Alan F. Hofmann

Research output: Contribution to journalArticle

384 Citations (Scopus)

Abstract

Background/Aims: A double-blind, placebo-controlled trial of ursodeoxycholic acid (UDCA) was conducted in 180 patients with primary biliary cirrhosis (PBC) to define the efficacy and safety of UDCA. Efficacy was assessed by time to treatment failure defined as death; liver transplantation; histological progression; development of varices, ascites, or encephalopathy; doubling of total serum bilirubin levels; progression of fatigue or pruritus; drug toxicity; or voluntary withdrawal. Methods: Patients with well-defined PBC underwent complete history, physical examination, liver chemistries, ultrasonography, upper endoscopy, and liver biopsy at entry as well as at 2 years. Liver chemistries were determined every 3 months. Results: In patients receiving UDCA, treatment failure was delayed compared with the placebo-treated group (P = 0.0003, log rank test). Seven patients receiving UDCA died or required transplantation compared with 12 in the placebo group (P = 0.18). No patients discontinued UDCA because of side effects or toxicity. Conclusions: UDCA was extraordinarily safe and well tolerated, and its use was associated with delayed progression of the disease as defined in this study. However, the lack of effects on symptoms, histology, and the need for liver transplantation or survival indicate that further evaluation is necessary to determine the ultimate role of UDCA in the treatment of PBC.

Original languageEnglish (US)
Pages (from-to)1284-1290
Number of pages7
JournalGastroenterology
Volume106
Issue number5
StatePublished - 1994
Externally publishedYes

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Ursodeoxycholic Acid
Biliary Liver Cirrhosis
Placebos
Treatment Failure
Liver Transplantation
Therapeutics
Liver
Varicose Veins
Brain Diseases
Pruritus
Drug-Related Side Effects and Adverse Reactions
Bilirubin
Ascites
Endoscopy
Physical Examination
Fatigue
Disease Progression
Ultrasonography
Histology
Transplantation

ASJC Scopus subject areas

  • Gastroenterology

Cite this

Lindor, K., Dickson, E. R., Baldus, W. P., Jorgensen, R. A., Ludwig, J., Murtaugh, P. A., ... Hofmann, A. F. (1994). Ursodeoxycholic acid in the treatment of primary biliary cirrhosis. Gastroenterology, 106(5), 1284-1290.

Ursodeoxycholic acid in the treatment of primary biliary cirrhosis. / Lindor, Keith; Dickson, E. Rolland; Baldus, William P.; Jorgensen, Roberta A.; Ludwig, Jurgen; Murtaugh, Paul A.; Harrison, Jay M.; Wiesner, Russell H.; Anderson, Monte L.; Lange, Stephen M.; Lesage, Gene; Rossi, Steven S.; Hofmann, Alan F.

In: Gastroenterology, Vol. 106, No. 5, 1994, p. 1284-1290.

Research output: Contribution to journalArticle

Lindor, K, Dickson, ER, Baldus, WP, Jorgensen, RA, Ludwig, J, Murtaugh, PA, Harrison, JM, Wiesner, RH, Anderson, ML, Lange, SM, Lesage, G, Rossi, SS & Hofmann, AF 1994, 'Ursodeoxycholic acid in the treatment of primary biliary cirrhosis', Gastroenterology, vol. 106, no. 5, pp. 1284-1290.
Lindor K, Dickson ER, Baldus WP, Jorgensen RA, Ludwig J, Murtaugh PA et al. Ursodeoxycholic acid in the treatment of primary biliary cirrhosis. Gastroenterology. 1994;106(5):1284-1290.
Lindor, Keith ; Dickson, E. Rolland ; Baldus, William P. ; Jorgensen, Roberta A. ; Ludwig, Jurgen ; Murtaugh, Paul A. ; Harrison, Jay M. ; Wiesner, Russell H. ; Anderson, Monte L. ; Lange, Stephen M. ; Lesage, Gene ; Rossi, Steven S. ; Hofmann, Alan F. / Ursodeoxycholic acid in the treatment of primary biliary cirrhosis. In: Gastroenterology. 1994 ; Vol. 106, No. 5. pp. 1284-1290.
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AU - Dickson, E. Rolland

AU - Baldus, William P.

AU - Jorgensen, Roberta A.

AU - Ludwig, Jurgen

AU - Murtaugh, Paul A.

AU - Harrison, Jay M.

AU - Wiesner, Russell H.

AU - Anderson, Monte L.

AU - Lange, Stephen M.

AU - Lesage, Gene

AU - Rossi, Steven S.

AU - Hofmann, Alan F.

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AB - Background/Aims: A double-blind, placebo-controlled trial of ursodeoxycholic acid (UDCA) was conducted in 180 patients with primary biliary cirrhosis (PBC) to define the efficacy and safety of UDCA. Efficacy was assessed by time to treatment failure defined as death; liver transplantation; histological progression; development of varices, ascites, or encephalopathy; doubling of total serum bilirubin levels; progression of fatigue or pruritus; drug toxicity; or voluntary withdrawal. Methods: Patients with well-defined PBC underwent complete history, physical examination, liver chemistries, ultrasonography, upper endoscopy, and liver biopsy at entry as well as at 2 years. Liver chemistries were determined every 3 months. Results: In patients receiving UDCA, treatment failure was delayed compared with the placebo-treated group (P = 0.0003, log rank test). Seven patients receiving UDCA died or required transplantation compared with 12 in the placebo group (P = 0.18). No patients discontinued UDCA because of side effects or toxicity. Conclusions: UDCA was extraordinarily safe and well tolerated, and its use was associated with delayed progression of the disease as defined in this study. However, the lack of effects on symptoms, histology, and the need for liver transplantation or survival indicate that further evaluation is necessary to determine the ultimate role of UDCA in the treatment of PBC.

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