Clinical and statistical analyses of new and evolving therapies for primary biliary cirrhosis

R. H. Wiesner, P. M. Grambsch, Keith Lindor, J. Ludwig, E. R. Dickson

Research output: Contribution to journalArticle

51 Citations (Scopus)

Abstract

Primary biliary cirrhosis is a chronic, progressive, cholestatic liver disease thought to be related to abnormalities in immune regulation. The disease is associated with granulomatous bile duct destruction, cholestasis, hepatic copper overlaoding and the development of hepatic fibrosis or cirrhosis or both. There have been numerous therapeutic trials evaluating immunosuppressive, antifibrotic and cupruretic agents. Prednisolone, D-penicillamine, azathioprine, colchicine and chlorambucil have been evaluated in controlled clinical trials, and biochemical improvement of liver function has been noted with all of the agents, except D-penicillamine. Improved survival has also been reported in patients treated long-term with azathioprine and colchicine. However, none of the therapeutic agents has been demonstrated to halt histologic progression of the disease or to induce a complete clinical, biochemical and histologic remission as has been reported in patients with autoimmune chronic active hepatitis treated with corticosteroids. Many of the trials did not use a double-blind design, failed to use the 'intent to treat' rule or failed to define an objective time to analyze results. Many of the studies involved small numbers of patients with short-term follow-up and thus potentially were inadequate to appreciate drug effects that might be of clinical benefit. Currently, there is no totally effective therapy for primary biliary cirrhosis. We believe that well-designed clinical trials can provide important information to better understand this disease until a totally effective therapy is available. New clinical trials should use well-established methodologic guidelines in study design and well-accepted statistical standards in the analysis and interpretation of results.

Original languageEnglish (US)
Pages (from-to)668-676
Number of pages9
JournalHepatology
Volume8
Issue number3
StatePublished - 1988
Externally publishedYes

Fingerprint

Biliary Liver Cirrhosis
Penicillamine
Azathioprine
Colchicine
Liver
Fibrosis
Clinical Trials
Chlorambucil
Autoimmune Hepatitis
Cholestasis
Controlled Clinical Trials
Chronic Hepatitis
Therapeutics
Immunosuppressive Agents
Prednisolone
Bile Ducts
Disease Progression
Liver Diseases
Copper
Adrenal Cortex Hormones

ASJC Scopus subject areas

  • Hepatology

Cite this

Wiesner, R. H., Grambsch, P. M., Lindor, K., Ludwig, J., & Dickson, E. R. (1988). Clinical and statistical analyses of new and evolving therapies for primary biliary cirrhosis. Hepatology, 8(3), 668-676.

Clinical and statistical analyses of new and evolving therapies for primary biliary cirrhosis. / Wiesner, R. H.; Grambsch, P. M.; Lindor, Keith; Ludwig, J.; Dickson, E. R.

In: Hepatology, Vol. 8, No. 3, 1988, p. 668-676.

Research output: Contribution to journalArticle

Wiesner, RH, Grambsch, PM, Lindor, K, Ludwig, J & Dickson, ER 1988, 'Clinical and statistical analyses of new and evolving therapies for primary biliary cirrhosis', Hepatology, vol. 8, no. 3, pp. 668-676.
Wiesner, R. H. ; Grambsch, P. M. ; Lindor, Keith ; Ludwig, J. ; Dickson, E. R. / Clinical and statistical analyses of new and evolving therapies for primary biliary cirrhosis. In: Hepatology. 1988 ; Vol. 8, No. 3. pp. 668-676.
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