Table 1 Studies of MMF in AIH
Author | Year | Type of study | Patients, n | Population (age group, variant, cirrhosis, indication for MMF) | Biochemical remission | Definition endpoint |
|---|---|---|---|---|---|---|
Richardson [22] | 2000 | Retrospective | 7 | Population: adult and children Variant: n=0 Cirrhosis: n=1 Indication: refractory disease (n=4), intolerance AZA (n=3) | 71% | Normalisation of ALT at 3 months |
Devlin [24] | 2004 | Retrospective | 5 | Population: adult Variant: n=0 Cirrhosis: n=2 Indication: refractory disease (n=3), side-effects (n=1), other reasons (n=1) | 100% | Normalised ALT at any timepoint |
Chatur [21] | 2005 | Retrospective | 16 (11/16 on MMF monotherapy) | Population: adult Variant: n=0 Cirrhosis: unknowna Indication: unknowna | 64% | Normalisation of serum aminotransferases at any timepoint |
Czaja [25] | 2005 | Retrospective | 8 | Population: adult Variant: n=0 Cirrhosis: n=1 Indication: treatment naïve (n=1), incomplete response (n=4), treatment failure (n=2), multiple relapses (n=1) | 0% | Normalisation of AST, bilirubin, and IgG at any timepoint |
Inductivo-Yu [20] | 2007 | Retrospective | 15 | Population: adult Variant: n=2 Cirrhosis: n=8 Indication: biochemical or histologic non-response (n=11), significant side effects (n=14) | 73% | Normalisation of ALT at unknown time point |
Hlivko [19] | 2008 | Retrospective | 29 | Population: adult Variant: unknowna Cirrhosis: unknowna Indication: intolerance or nonresponse (n=12 ➔ 9 prednisone + MMF; 1 MMF alone; 1 prednisone + MMF + tacrolimus; 1 MMF + cyclosporine), first-line therapy (n=17) | 55% | Resolution of symptoms, reduction in serum aminotransferase levels to <2 ULN, normalisation of serum bilirubin and γ-globulin levels (and improvement in liver histology to normal or only mild portal hepatitis) at unknown time point |
Hennes [18]b | 2008 | Retrospective | 36 | Population: unknown Variant: unknown Cirrhosis: unknown Indication: side effects (n=28), insufficient response (n=9), pregnancy (n=1) | 39% | AST <2x ULN at unknown timepoint |
Wolf [17] | 2009 | Retrospective | 16 | Population: unknown Variant: n=4 Cirrhosis: unknown Indication: intolerance (n=7), refractory disease (n=6), other reasons (n=3) | 31% | Reduction of ALT from greater than twice normal to less than twice normal |
Baven-Pronk [15] | 2011 | Retrospective | 45 | Population: adult Variant: (n=15) Cirrhosis: (n=24) Indication: AZA-non-responders (n=22), AZA-intolerance (n=23) | 47% | Normalisation of AST and/or ALT at any timepoint after starting MMF |
Zachou [28] | 2011 | Prospective | 59 | Population: adult + children Variant: n=0 Cirrhosis at presentation: n=14 Indication: treatment-naïve (n=59) | 88% | Normalisation of AST, ALT, and γ-globulins within 12 months |
Jothimani [16] | 2014 | Retrospective | 20 | Population: adult Variant: n=3 Cirrhosis: n=5 Indication: AZA intolerance (n=18), refractory disease (n=2) | 74% | Normalisation of ALT and/or AST at any timepoint |
Zachou [29] | 2016 | Prospective | 109 | Population: adult + children Variant: n=0 Cirrhosis at presentation: n=26 Indication: treatment-naïve (n=109) | 72% | Normalisation of ALT, AST, and IgG, symptoms improved or disappeared and liver histology, if performed, showed minimal or no inflammation at 3 months treatment |
Roberts [14] | 2018 | Retrospective | 105 | Population: adult Variant: n=0 Cirrhosis: n=38 Indication: suboptimal response (n=42), treatment intolerance (n=63) | 60% | Normalisation of ALT, AST, and IgG, with or without normal liver histology within the first 2 years of treatment |
Nicoll [13] | 2019 | Retrospective | 105 | Population: adult Variant: n=0 Cirrhosis: n=38 Indication: intolerance (n=63), nonresponse (n=42) | 60% | ALT, AST and IgG <ULN, with or without normal liver histology, within the first 2 years of treatment |
Giannakopoulos [12] | 2019 | Retrospective | 22 | Population: adult Variant: n=0 Cirrhosis: n=6 Indication: intolerance (n=14), non-response (n=5), intolerance + non-response (n=3) | 45% | Normalisation of ALT and AST within 3 to 30 weeks |
Liberal [23] | 2021 | Retrospective | 18 | Population: adult Variant: n=0 Cirrhosis: n=4 Indication: intolerance (n=9), refractory disease (n=9) | 39% | Normalisation of AST, ALT, and IgG at 12 months |
Dalekos [30] | 2021 | Prospective | 32 | Population: adult + children Variant: n=0 Cirrhosis: n=6 Indication: treatment-naïve (n=32) | 93.8%c | Normalisation of AST, ALT, and IgG at 6, 12 months, and at the end of follow-up |