Treatment of Paget disease of bone (osteitis deformans). Results of a one-year study with sodium etidronate.

A double-blind crossover study was performed in which 50 symptomatic patients with Paget disease were treated with sodium etidronate (EHDP) doses of 1, 2.5, 5, 10, or 20 mg/kg or were given placebo. After six months, the patients were reassigned to treatment with placebo or sodium etidronate, 5,10, or 20 mg/kg. Sodium etidronate in dosages of 5,10, or 20 mg/kg/day resulted in significant improvement in levels of serum alkaline phosphatase and urinary hydroxyproline, as well as clinical symptoms and bone scans. Bone scans and urinary determinations showed statistically greater improvement with sodium etidronate 20 mg/kg, in comparison to patients who received sodium etidronate 5 mg/kg for 12 months. After withdrawal of effective therapy, five of nine patients continued to have remission of their symptoms with placebo for an additional six months. ( JAMA 230:562-567, 1974)