EFFECTIVE ORAL TREATMENT OF SEVERE PAGET'S DISEASE OF BONE WITH APD (3‐AMINO‐I‐HYDROXYPROPYLIDENE‐1,1‐BISPHOSPHONATE); A COMPARISON WITH COMBINED CALCITONIN + EHDP (1 ‐HYDROXYETHYLIDENE‐1,1 ‐BISPHOSPHONATE)

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Abstract
Ten patients with severe Paget''s disease of bone and serum alkaline phosphatase (sAP) > 900 IU/l were treated for 6 mo. with the oral diphosphonate APD, (3-amino-1-hydroxypropylidene-1, 1-bisphosphonate). By the end of the treatment period there was a reduction in the log mean urine hydroxyproline (uHP) and the log mean sAP of 92% and 87%, respectively. In 4 patients both sAP and uHP fell to within the normal range and remained normal for at least 6 mo. after therapy was stopped. Bone scintigraphy showed a fall in 99mTc-MDP uptake in sites of active Paget''s disease in all patients and histomorphometry showed no increase in osteoid. Repair of radiological osteolytic lesions was observed in 6/6 patients and progression of tibial osteolytic wedges was arrested in 5/5 patients and reversed in 4. This improvement persisted 6 mo. after completion of therapy but further wedge progression occurred in 1 patient whose urine HP remained elevated. There were no serious effects though 5 patients complained of nausea. The clinical and biochemical responses to APD were equivalent to those observed in the same patients during a previous 6 mo. course of combined therapy with human calcitonin (CT) + EHDP except that there was additional biochemical and radiological evidence of bone healing. This study confirms PAD as an effective treatment of severe Paget''s disease of bone.