Approach
Treatment is aimed at decreasing excessive osteoclastic activity and bone turnover. Treatment is indicated for symptomatic patients (e.g., those with bone pain, deformities, and elevated bone turnover) and in patients at risk of complications, particularly with disease in facial bones (malocclusion, deformities), skull (cranial nerve compression, entrapment), vertebrae (compression fractures), and locations adjacent to a joint (functional impairment, pain).[3] Treatment may be considered in patients undergoing surgery on a pagetic bone: for example, corrective osteotomy for tibial bowing or total joint arthroplasty for osteoarthritis. Some studies suggest that treatment decreases blood flow and reduces risk of bleeding during surgery, although results are conflicting.[30]
The goal of treatment is to reduce the disease metabolic activity and bring it close to normal. Biochemical markers of bone turnover are monitored after 6 months of treatment, and subsequently every 6 to 12 months, or until symptoms reappear. In one trial, treatment based on activity markers was not superior to symptom-directed treatment, and it is not clear if re-treatment based solely on increased serum bone turnover markers is warranted.[40] Clinical guidelines suggest treatment should be aimed at improving symptoms rather than normalising alkaline phosphatase.[30]
Asymptomatic patients, in whom PDB has been diagnosed as an incidental finding and who are at low risk for complications, can be monitored without active pharmacological treatment.
Bisphosphonates
Bisphosphonates are potent inhibitors of bone resorption and are the mainstay of treatment for PDB. There is moderate-quality evidence that bisphosphonates reduce bone pain in people with PDB compared with placebo.[41]
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Zoledronic acid, alendronic acid, and risedronate sodium are used as first-line agents.[28]
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For those without contraindications, a single intravenous dose of zoledronic acid is recommended.[28] Compared with oral risedronate, a single intravenous dose of zoledronic acid had more rapid onset of action and resulted in superior effects on quality of life.[42] Daily use of high-dose oral bisphosphonates is frequently associated with gastrointestinal side effects, and needs to be used with caution in patients with a past medical history of gastrointestinal reflux disease. The choice of bisphosphonate depends on: individual doctor and patient preference concerning route of administration (oral or intravenous), likely compliance with medicine, and the monitoring required; previous experience of particular bisphosphonates and resulting effect; and any contraindications.
Calcium and vitamin D levels should be normal before bisphosphonate treatment to reduce the risk of hypocalcaemia or worsening osteomalacia.[3]
Calcitonin
Calcitonin has been shown to partially heal lytic lesions in PDB, but bone turnover markers rarely normalise.[28][43] Calcitonin is generally indicated only in people who are unable to tolerate bisphosphonates or who have contraindications to bisphosphonates.[44]
Supportive therapy
Physiotherapy provides an opportunity for education about high-risk activities and promotes range of motion, muscular strength, and ambulation skills. If long-bone deformity ensues, orthoses can be added to the therapy, as well as training using walking aids such as walking sticks, crutches, and walkers.
The use of hearing aids is recommended at a very early stage for people with hearing deficits because of the progressive nature of the disease.
Analgesics are indicated for pain and inflammatory symptoms.
Surgery
Surgical intervention is rarely required and is usually only recommended for the treatment of complications related to advanced disease and/or concomitant pathologies (e.g., reduction and fixation for femoral fracture, or total hip or knee arthroplasty for patients with hip or knee end-stage osteoarthritis associated with PDB). Laminectomy is recommended for the treatment of patients with spinal stenosis.
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