In most patients with
autoimmune hepatitis, remission can be maintained with
prednisolone, usually in combination with
azathioprine, but the majority of patients have a relapse when treatment is stopped and therefore require long-term
therapy. Because prolonged
corticosteroid therapy may have serious toxic effects, in 1984 we undertook a controlled trial of maintenance
therapy with
azathioprine alone. None of the 25 patients in that trial had relapses during the follow-up period of one year. We have now followed these 25 patients for 10 years and have treated an additional 47 patients in a similar manner.
METHODS: RESULTS: Sixty patients (83 percent) remained in remission while receiving
azathioprine alone for a median of 67 months (range, 12 to 128). Of 48 follow-up liver biopsies in 42 patients, 45 showed inactive or minimal disease, and 3 showed moderate disease (2 after one year of
therapy and 1 after eight years). After the
prednisolone had been withdrawn, 26 patients lost their cushingoid
facies, and 32 patients lost weight (median loss, 6.4 kg; range, 1.5 to 22.3). The most common adverse effect was
arthralgia (in 38 patients). With the higher dose of
azathioprine, four patients had myelosuppression, defined as a decrease in the leukocyte and platelet counts to less than 4000 and 150,000 per cubic millimeter, respectively. Two of these patients (both with
pancytopenia) relapsed when the
azathioprine was withdrawn; in the other two, remission was maintained with the resumption of
prednisolone.
Lymphopenia developed in 32 of 56 patients treated with 2 mg of
azathioprine per kilogram per day for more than two years. During follow-up, nine patients died: one of
liver failure and eight of causes not directly related to their
liver disease.
CONCLUSIONS: