Mithramycin therapy in Paget's disease.

Nine patients with symptomatic Paget's disease were treated with mithramycin in doses of 15 mug/kg/day for 10 days. All but one had considerable symptomatic improvement and, in some, pain relief was dramatic after only a few days. A progressive decrease in serum alkaline phosphatase levels and urinary hydroxyproline excretion was seen at the same time. Bone scans with technetium-99m stannous polyphosphate were performed in six patients and were grossly abnormal in all, showing increased uptake in areas approximately co-extensive with the radiological changes. In five cases improvement in the scans was seen during therapy. In those cases in which the scans had not returned to normal by the end of therapy the improvement continued and was associated with a further decrease in serum alkaline phosphatase levels. We suggest that mithramycin is effective therapy for Paget's disease and, in the dose used, is safe.