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Paget's disease of bone in 51 patients was treated with ethane-1, hydroxy-1,1 diphosphonate (EHDP) for six months at a dosage of 5 mg/kg/day. Clinically, the analgesic effect on bone pain is clear, with parallel reduction in radioisotope uptake observed by quantitative bone scintigraphy. There is also a reduction of about 70% of the excess level of serum alkaline phosphatase and total 24-hour urinary hydroxyproline. Histologically, there is a significant reduction in both the osteoclastic resorption surfaces and the number of osteoclasts. In contrast to that obtained by calcitonin, these clinical, biologic, and histologic changes continue for at least one year beyond the end of the treatment. That is the main characteristic of this treatment, of which the clinical and biologic tolerance is admittedly very satisfactory. Accumulation of osteoid tissue, which is indicative of a mineralization defect, does not appear at low dosages under the prescribed conditions. EHDP (5 mg/kg/day) can be regarded as effective treatment for some patients with Paget's disease of bone.

Quantitative bone scans were performed with 99mTc-EHDP in 170 untreated pagetic patients (93 men, 77 women; mean age, 65.4 years). The distribution of 863 pagetic skeletal locations was analyzed. Bone scans demonstrated 8.3% more pagetic sites than roentgenograms. The extent of Paget's disease was evaluated in each patient by a scintigraphic skeletal index. This index correlated with serum alkaline phosphatase (SAP) and urinary hydroxyproline (HyPro) levels, and also with hypocalcemic acute response to calcitonin. The correlation of SAP with an index of activity (extent index adjusted by uptake ratios) was better than with the nonadjusted index. Only 30.6% of pagetic sites were responsible for clinical symptoms. No correlation was found between age and skeletal index of the disease.