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Treatment of Paget disease of bone

Author
Julia F Charles, MD, PhD
Section Editors
Ethel Siris, MD
Marc K Drezner, MD
Deputy Editor
Paul L Romain, MD

INTRODUCTION

Paget disease of bone, also known as osteitis deformans, is a focal disorder of bone metabolism that is characterized by an accelerated rate of bone remodeling, resulting in overgrowth of bone at selected sites and impaired integrity of affected bone. The main clinical manifestations of Paget disease are pain and deformities in affected areas and a heightened risk of fracture. Osteoarthritis in proximal joints and neurologic disease are also common complications; high-output heart failure and abnormalities in calcium and phosphate balance are unusual, and osteosarcomas arising in pagetic bone are rare. Because of the vascularity of pagetic bone, excessive bleeding may occur during orthopaedic surgery on pagetic bone.

Many patients with Paget disease of bone are asymptomatic. The diagnosis in such patients is usually made incidentally following a routine chemistry screen showing an elevated serum concentration of alkaline phosphatase of bone origin, or by an imaging study obtained for some other reason that shows pagetic changes in bone. (See "Clinical manifestations and diagnosis of Paget disease of bone".)

In patients with Paget disease, the newer generations of nitrogen-containing bisphosphonates can ease pain, slow bone turnover, and promote deposition of normal lamellar bone, which in turn results over time in the normalization of serum alkaline phosphatase and other markers of bone turnover (see 'Use of nitrogen-containing bisphosphonates' below). Effective therapy may also lead to radiographic healing of lytic lesions and a lessening of increased isotope uptake on bone scan [1-5]. Analgesic, rehabilitative, surgical, and other measures may also be of benefit.

The treatment of Paget disease of bone will be reviewed here. The pathogenesis, clinical manifestations, and diagnosis of this disorder are discussed separately. (See "Clinical manifestations and diagnosis of Paget disease of bone".)

GOALS AND INDICATIONS FOR THERAPY

The goals of treatment are to ease pain and to reduce the rate of bone remodeling toward normal, which will enhance the deposition of normal lamellar bone, decrease the vascularity of bone, and slow disease progression. Treatment is indicated in patients with symptomatic Paget disease and in selected asymptomatic patients with significant biochemical abnormalities or imaging changes indicating risk of complications from untreated disease. (See 'Treatment indications' below.)

                                   

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Literature review current through: Nov 2016. | This topic last updated: Wed Jun 15 00:00:00 GMT+00:00 2016.
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