What is the treatment approach for Paget's disease of bone?

From the Guidelines

The treatment approach for Paget's disease of bone primarily involves bisphosphonates as first-line therapy, with specific medications including pamidronate, which has been shown to be effective in maintaining bone mass and preventing fractures in patients with Paget's disease 1.

Key Considerations

• Bisphosphonates, such as pamidronate, work by inhibiting osteoclastic bone resorption, which helps normalize the accelerated bone turnover characteristic of Paget's disease 1.
• Treatment is generally indicated for patients with symptoms such as bone pain, active disease near joints, neurological complications, or those at risk for pathological fractures.
• Asymptomatic patients with normal alkaline phosphatase levels may not require immediate treatment but should be monitored.
• Before starting bisphosphonates, calcium and vitamin D levels should be normalized to prevent hypocalcemia.

Alternative Therapies

• For patients who cannot tolerate bisphosphonates, calcitonin is an alternative, though less effective, with documented efficacy in the treatment of Paget’s disease 1.
• Calcitonin acts by inhibiting osteoclast action and may also stimulate osteoblast function.

Monitoring and Adjunctive Therapy

• Regular monitoring of alkaline phosphatase levels every 3-6 months helps assess treatment response, with the goal of reducing levels to normal or near-normal range.
• Pain management with NSAIDs or acetaminophen may be needed as adjunctive therapy.

Surgical Intervention

• Surgical intervention may be necessary for complications like fractures, severe arthritis, or spinal stenosis.

From the FDA Drug Label

2.5 Treatment of Paget's Disease of Bone The recommended treatment regimen is 40 mg once a day for six months. Re-treatment of Paget's Disease Re-treatment with alendronate sodium tablets, may be considered, following a six-month post-treatment evaluation period in patients who have relapsed, based on increases in serum alkaline phosphatase, which should be measured periodically Re-treatment may also be considered in those who failed to normalize their serum alkaline phosphatase.

The treatment approach for Paget's disease of bone is to take alendronate sodium tablets at a dose of 40 mg once a day for six months 2.

• Re-treatment may be considered after a six-month post-treatment evaluation period in patients who have relapsed or failed to normalize their serum alkaline phosphatase.
• The treatment is indicated in patients with Paget's disease of bone who have alkaline phosphatase at least two times the upper limit of normal, or those who are symptomatic, or those at risk for future complications from their disease 2.
• Alendronate sodium was effective in reducing alkaline phosphatase levels and was similarly effective regardless of age, gender, race, prior use of other bisphosphonates, or baseline alkaline phosphatase within the range studied 2.

From the Research

Treatment Approach for Paget's Disease of Bone

The treatment approach for Paget's disease of bone typically involves the use of bisphosphonates, which are highly effective in suppressing the elevations in bone turnover characteristic of the disease 3, 4, 5.

• Bisphosphonate Therapy: The mainstay of treatment for Paget's disease of bone is bisphosphonate therapy, with zoledronic acid being the most effective agent 3, 6, 7.
• Zoledronic Acid: A single infusion of zoledronic acid leads to a sustained reduction in bone pain and markers of bone turnover 3, 6, 7.
• Oral Bisphosphonates: Low-dose, once-weekly, oral bisphosphonate preparations, such as alendronate and risedronate, can also be effective in achieving remission in patients with Paget's disease of bone 4.
• Treatment Reservation: Bisphosphonates should be reserved for symptomatic patients, as treatment with these agents has been associated with an increase in rates of fracture in patients with asymptomatic Paget's disease of bone 3.
• Remission and Relapse: Remission can be achieved in a large proportion of patients with Paget's disease of bone using bisphosphonate therapy, and remission can be maintained for an extended period 4, 7.

Key Considerations

• Symptomatic Patients: Treatment with bisphosphonates is typically reserved for symptomatic patients with Paget's disease of bone 3.
• Asymptomatic Patients: Asymptomatic patients may not require treatment with bisphosphonates, as the risk of fracture may be increased 3.
• Long-term Effects: The long-term effects of bisphosphonate therapy on complications of Paget's disease of bone, such as deformity, pathological fractures, and deafness, have not been adequately studied 5.