Long-term Methimazole Therapy in Juvenile Graves' Disease: A Randomized Trial.

Background And Objectives: Recent studies show that long-term (LT) antithyroid drugs reduce relapse of hyperthyroidism in patients with Graves' disease. Our objective was to evaluate the effectiveness and safety of LT methimazole treatment and to compare remission rates in Graves' disease patients after LT and short-term (ST) therapy.

Methods: In this randomized, parallel group trial, 66 consecutive patients with untreated juvenile Graves' hyperthyroidism were enrolled. After a median 22 months of methimazole treatment, 56 patients were randomly assigned to either continue low-dose methimazole treatment ( = 24, LT group) or to discontinue treatment ( = 24, ST group). Twenty-four patients in LT group completed 96 to 120 months of methimazole treatment. Patients in both groups were managed for 48 months after discontinuation of treatment.

Results: Except for 3 cases of cutaneous reactions, no other adverse events were observed throughout 120 months of methimazole therapy. Serum free thyroxine, triiodothyronine, thyrotropin, and thyrotropin receptor antibody remained normal, and the required daily dosage of methimazole was gradually decreased from 5.17 ± 1.05 mg at 22 months to 3.5 ± 1.3 mg between 96 and 120 months of treatment ( < .001). Hyperthyroidism was cured in 92% and 88% of LT patients and in 46% and 33% of ST patients, 1 and 4 years after methimazole withdrawal, respectively.

Conclusions: LT methimazole treatment of 96 to 120 months is safe and effective for treatment of juvenile Graves' disease. The four-year cure rate of hyperthyroidism with LT methimazole treatment is almost 3 times more than that of ST methimazole treatment.