Efficacy and Safety of Mycophenolate Mofetil in Patients With Autoimmune Hepatitis and Suboptimal Outcomes After Standard Therapy

Stuart K. Roberts, Ricky Lim, Simone Strasser, Amanda Nicoll, Alessia Gazzola, Joanne Mitchell, Way Siow, Tiffany Khoo, Zaki Hamarneh, Martin Weltman, Natasha Janko, Edmund Tse, Gauri Mishra, En Hsiang Cheng, Miriam Levy, Wendy Cheng, Siddharth Sood, Richard Skoien, Jonathan Mitchell, Amany ZekryJacob George, Gerry MacQuillan, Alan Wigg, Katherine Stuart, William Sievert, Geoffrey McCaughan, ALA Clinical Research Network

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29 Citations (Scopus)


Background & Aims: Little is known about outcomes of patients with autoimmune hepatitis (AIH) who have a suboptimal outcome to standard therapy and are then given mycophenolate mofetil as rescue therapy. We evaluated the efficacy and safety of mycophenolate mofetil in patients failed by or intolerant to corticosteroids, with or without azathioprine.

Methods: We performed a retrospective study of 105 patients with AIH who received mycophenolate mofetil therapy after an inadequate response or intolerance to standard therapy (98% received combination therapy with corticosteroids plus thiopurines). Patients were recruited from 17 liver clinics via the Australian Liver Association Clinical Research Network. We reviewed records for baseline demographic features and characteristics of liver disease, initial therapy, mycophenolate mofetil indications, treatment outcome, and side effects. The primary outcome was biochemical remission, defined as levels of alanine and aspartate transferase and IgG level within the normal reference range, with or without normal liver histology within the first 2 years of treatment.

Results: The indication for mycophenolate mofetil therapy was non-response to treatment for 40% of cases and intolerance to therapy for 60%. Overall, 63 patients (60%) achieved biochemical remission following a median 12 weeks treatment with mycophenolate mofetil. The proportion of patients who achieved biochemical remission was similar between patients receiving mycophenolate mofetil for non-response to standard therapy (57%) and patients with intolerance to standard therapy (62%). However, a lower proportion of patients with cirrhosis achieved biochemical remission (47%) than patients without cirrhosis (6%) (P =.07). Serious adverse events occurred in 3 patients (2.7%) including 1 death, and 10 patients (9.2%) discontinued mycophenolate mofetil because of adverse events.

Conclusion: In this retrospective study of patients with AIH who received mycophenolate mofetil as a rescue therapy, we found the drug to be well tolerated and moderately effective, inducing biochemical remission in 60% of subjects. Rates of response are lower and rates of infection are higher in patients with AIH and cirrhosis. Prospective studies of mycophenolate mofetil are warranted for this population.

Original languageEnglish
Pages (from-to)268-277
Number of pages10
JournalClinical Gastroenterology and Hepatology
Issue number2
Publication statusPublished - Feb 2018
Externally publishedYes


  • Immune Suppressant
  • Inflammation
  • Periportal Hepatitis
  • TAPESTRY Study


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