A review of Paget's disease of bone with a focus on the efficacy and safety of zoledronic acid 5 mg.

Background: Paget's disease of bone, the second most common metabolic bone disease in the United States, is characterized by localized areas of excessive bone resorption coupled with accelerated bone formation, resulting in new bone that is less structurally organized and is weaker than normal bone. Complications of Paget's disease can include bone pain, osteoarthritis, skeletal deformity, hearing loss, and fractures. The objective of this review is to provide a comprehensive overview of current standards of treatment in Paget's disease.

Scope: A review of literature from 1974 to 2007 was performed on topics such as epidemiology, etiology, treatment of Paget's disease of bone, and bisphosphonates.

Findings: Paget's disease affects an estimated 2-7% of persons of age 55 years or older in North America and western Europe. Antiresorptive treatment with bisphosphonates is the standard treatment, but there may be limitations to oral therapy. Intravenous pamidronate is efficacious and has long been available, but its use is hindered by an impractical recommended dosing regimen of 30 mg IV over 4 h for three consecutive days. In two identical, double-blind, 6-month trials, 96% of patients treated with a one-time intravenous treatment of zoledronic acid 5 mg achieved therapeutic response, compared with 74% treated with 60 days of daily oral treatment with risedronate 30 mg (p < 0.001). One limitation of this review is that historical data are not reviewed in the same level of detail as newer treatments, because recent advances in pharmacotherapy of Paget's disease have reduced the clinical utility of the older drugs.

Conclusion: The etiology of Paget's disease is unclear, but some evidence suggests genetic and viral components. Bisphosphonates restore normal bone turnover and relieve bone pain, but oral formulations may be limited by complicated dosing regimens and poor gastrointestinal absorption. The bisphosphonate, zoledronic acid is administered as a single intravenous infusion and offers antiresorptive efficacy and longer-lasting remission.