Clinical and biochemical effects of EHDP in Paget's disease of bone: patterns of response to initial treatment and to long-term therapy.

Studies regarding the efficacy of EHDP in the treatment of Paget's disease of bone conducted at our institution are described. An initial placebo-controlled clinical trial established that doses of 5 mg/kg/day for 6 months produced symptomatic improvement and a 50% lowering of serum alkaline phosphatase and urinary hydroxyproline. A subsequent study indicated that the biochemical suppression was maintained during the ensuing 6 months without therapy. Current results of long-term intermittent EHDP treatment in 40 patients studied for 5-8 years demonstrate that a small minority of individuals experience a sustained remission after a single six-month course of therapy, while a majority require repeated courses of 5 mg/kg/day for 6 of every 12 months to maintain their initial improvement. Data are also presented describing the successful use of EHDP at a dose of 20 mg/kg/day for 1 of every 4 months in patients refractory to conventional low doses of therapy. The possible role of parathyroid hormone in the expression of Paget's disease is also discussed, and recommendations regarding the pharmacologic management of patients with this disorder are provided.

RCT Entities:   Population Interventions Outcomes