What is Paget's disease (Osteitis Deformans)?

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From the Guidelines

Paget's disease should be treated with bisphosphonates, specifically zoledronic acid or oral options like alendronate, to slow bone turnover and prevent complications, as supported by the most recent and highest quality studies 1.

Treatment Overview

The treatment of Paget's disease focuses on relieving symptoms, preventing complications, and improving quality of life. Bisphosphonates are the primary treatment option, with zoledronic acid being the most effective due to its potent inhibition of osteoclast activity.

  • Zoledronic acid (5mg IV once) is a highly effective treatment option for Paget's disease, as it significantly reduces bone turnover and alleviates symptoms.
  • Oral bisphosphonates like alendronate (40mg daily for 6 months) or risedronate (30mg daily for 2 months) are alternative treatment options, although they may not be as potent as zoledronic acid.

Supporting Evidence

The use of bisphosphonates in treating Paget's disease is supported by studies, including the K/DOQI clinical practice guidelines for bone metabolism and disease in chronic kidney disease 1. These guidelines highlight the effectiveness of bisphosphonates in increasing bone mass and preventing fractures in patients with osteoporosis, which is also relevant to Paget's disease.

Additional Considerations

Adequate calcium (1000-1500mg daily) and vitamin D (800-1000 IU daily) supplementation should accompany bisphosphonate treatment to support bone health. Pain management may include NSAIDs or acetaminophen, and regular monitoring with alkaline phosphatase levels helps assess disease activity and treatment response. Surgery may be necessary for complications like fractures or severe joint damage.

From the Research

Diagnosis of Paget's Disease

  • Paget's disease of bone is characterized by focal increased bone remodeling activity, resulting in sclerotic or lytic lesions and poor bone quality at one or more sites 2
  • Diagnosis is through plain film imaging, with radionuclide bone scan to determine the extent 2
  • Biochemical evaluation of a patient should be done using serum total ALP (alkaline phosphatase) or with the use of a more specific marker of bone formation: PINP (intact N-terminal type 1 procollagen propeptide) or CTX (cross-linked C-telopeptide) 3

Treatment of Paget's Disease

  • Bisphosphonates are the mainstay of the treatment of Paget's disease of bone (PDB) 4, 2, 5
  • Treatment with a potent bisphosphonate is indicated in patients with Paget's disease who have symptoms likely to respond to reduced bone turnover at pagetic sites 5
  • A single dose of 5 mg i.v. zoledronate is the current standard therapy for Paget's disease 2, 6, 3
  • Oral bisphosphonates, such as alendronate and risedronate, are less potent than zoledronic acid but can still achieve remission in a large proportion of patients 4, 5, 6

Management and Follow-up

  • Measurement of total ALP or other baseline disease activity markers at 6 to 12 weeks after treatment is an acceptable and cost-effective option to assess response to treatment 3
  • Maximum suppression of high bone turnover may require measurement at 6 months after administration 3
  • Biochemical follow-up is recommended to be used as a more objective indicator of relapse rather than symptoms 3
  • The prolonged response after zoledronate treatment should be assessed every 1-2 years after normal bone turnover, while with less potent drugs, every 6 to 12 months is appropriate 3

References

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Research

Paget's disease of bone: updates for clinicians.

Current opinion in endocrinology, diabetes, and obesity, 2019

Research

Medical management of Paget's disease of bone: indications for treatment and review of current therapies.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research, 2006

Research

Long-term control of bone turnover in Paget's disease with zoledronic acid and risedronate.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research, 2007

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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