Paget Disease of Bone
ICD-10 M88 · ICD-11 FB85

Treatment of Symptomatic Paget's Disease of Bone with Bone Pain

This protocol covers symptomatic Paget's disease of bone presenting with bone pain, headache due to skull involvement, back pain due to Pagetic arthropathy, or hypercalcaemia due to immobilisation — in patients without fracture in pagetic bone.

Current management guidelines recommend reserving treatment for symptomatic patients. The focus is on controlling symptoms — in particular bone pain — rather than suppression of bone turnover alone.

First-line therapy involves a bisphosphonate agent, which acts by directly inhibiting osteoclasts to suppress the accelerated bone turnover characteristic of Paget's disease. One available option is delivered as a single intravenous infusion; oral alternatives exist for appropriate patients. The specific agent, route, and full regimen are detailed in the complete protocol.

  • Normalisation of serum total alkaline phosphatase at 3–6 months after treatment
  • Resolution of bone pain
  • Improvement of osteolytic bone lesions on plain radiograph at 12 months

References

The current management guidelines for PDB recommend that treatment should be reserved for symptomatic patients, while those who are asymptomatic be observed.

This has led to the recommendation that bisphosphonate therapy should be reserved for use in symptomatic PDB and focus on symptom management rather than suppression of bone turnover.

The treatment of choice for PDB is bisphosphonates, which are highly effective at suppressing the accelerated bone turnover by directly inhibiting osteoclasts.

Treatment response is best assessed by measuring serum total ALP 3–6 months after treatment and then annually once levels are normalised.

If there are osteolytic lesions, the plain film should be repeated at 12 months to assess for improvement.

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