The role of mycophenolate mofetil in the management of autoimmune hepatitis and overlap syndromes

• Published in: Alimentary pharmacology & therapeutics 2011-08, Vol.34 (3), p.335-343
• Main Authors: Baven‐Pronk, A. M. C., Coenraad, M. J., van Buuren, H. R., de Man, R. A., van Erpecum, K. J., Lamers, M. M. H., Drenth, J. P. H., van den Berg, A. P., Beuers, U. H., den Ouden, J., Koek, G. H., van Nieuwkerk, C. M. J., Bouma, G., Brouwer, J. T., van Hoek, B.
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aged; azathioprine; Biological and medical sciences; Child; Cirrhosis; Cohort Studies; Digestive system; Female; Gastroenterology. Liver. Pancreas. Abdomen; Hepatitis; Hepatitis, Autoimmune - drug therapy; Hepatitis, Autoimmune - physiopathology; Humans; Immunosuppressive Agents - therapeutic use; Intolerance; Liver transplantation; Liver. Biliary tract. Portal circulation. Exocrine pancreas; Male; Medical sciences; Middle Aged; Mycophenolate mofetil; Mycophenolic Acid - analogs & derivatives; Mycophenolic Acid - therapeutic use; Netherlands; Other diseases. Semiology; Pharmacology. Drug treatments; Prednisolone; Remission; Retrospective Studies; Severity of Illness Index; Side effects; Syndrome; Treatment Outcome; Young Adult
• ISSN: 0269-2813; 1365-2036
• DOI: 10.1111/j.1365-2036.2011.04727.x

Aliment Pharmacol Ther 2011; 34: 335–343 Summary Background  Treatment failure occurs in 20% of autoimmune hepatitis patients on prednisolone and azathioprine (AZA). There is no established second line treatment. Aim  To assess the efficacy of mycophenolate mofetil as second line treatment after AZA‐intolerance or AZA‐nonresponse in autoimmune hepatitis and overlap syndromes. Methods  Consecutive patients from the Dutch Autoimmune Hepatitis Group cohort, consisting of 661 patients, with autoimmune hepatitis or overlap syndromes, AZA‐intolerance or AZA‐nonresponse and past or present use of mycophenolate mofetil were included. Primary endpoint of mycophenolate mofetil treatment was biochemical remission. Secondary endpoints were biochemical response (without remission), treatment failure and prevention of disease progression. Results  Forty‐five patients treated with mycophenolate mofetil were included. In autoimmune hepatitis remission or response was achieved in 13% and 27% in the AZA‐nonresponse group compared to 67% and 0% in the AZA‐intolerance group (P = 0.008). In overlap‐syndromes remission or response was reached in 57% and 14% in the AZA‐nonresponse group and 63% and 25% of the AZA‐intolerance group (N.S.); 33% had side effects and 13% discontinued mycophenolate mofetil. Overall 38% had treatment failure; this was 60% in the autoimmune hepatitis AZA‐nonresponse group. Decompensated liver cirrhosis, liver transplantations and death were only seen in the autoimmune hepatitis AZA‐nonresponse group (P