Mycophenolate mofetil: role in autoimmune hepatitis and overlap syndromes.

Martine A.M.C. Baven-Pronk (1) , Minneke J. Coenraad (1) , Henk van Buuren (2) , Robert A de Man (1) , Karel J van Erpecum (3) , Mieke M.H. Lamers (4) , Joost Drenth (1) , Aad A.P. van den Berg (5) , Ulrich Beuers (6) , Jannie den Ouden (7) , Ger H. Koek (8) , Carin M.J. van Nieuwkerk (9) , Gerd Bouma (1) , Johannes T. Brouwer (10) , Bart van Hoek (1)

1 Gastroenterology and Hepatology
2 Gastroenterology and Hepatology
3 Gastroenterology
4 Gastroenterology and Hepatology
5 UMCG - University Medical Center Groningen [Groningen]
6 Department of Gastroenterology & Hepatology
7 Gastroenterology and Hepatology
8 Gastroenterology and Hepatology
9 Gastroenterology and Hepatology
10 Gastroenterology and Hepatology

Background and Aims: Treatment failure occurs in 20% of autoimmune hepatitis patients on prednisolone and azathioprine (AZA). There is no established second line treatment. This retrospective observational study was conducted 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 (NS). 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 < 0.001). Conclusion: Mycophenolate mofetil induced response or remission in a majority of patients with autoimmune hepatitis and azathioprine-intolerance and with overlap syndromes, irrespective of intolerance or nonresponse for azathioprine. In autoimmune hepatitis with azathioprine nonresponse mycophenolate mofetil is less often efective.

Mots clés: Medicine

Martine A.M.C. Baven-Pronk, Minneke J. Coenraad, Henk van Buuren, Robert A de Man, Karel J van Erpecum, et al.. Mycophenolate mofetil: role in autoimmune hepatitis and overlap syndromes.. Alimentary Pharmacology and Therapeutics, 2011, 34 (3), pp.335. ⟨10.1111/j.1365-2036.2011.04727.x⟩. ⟨hal-00650658⟩