Principles of managing Paget disease of bone

Bisphosphonates are the mainstay of treatment for Paget disease of bone. Analgesics and nonsteroidal anti-inflammatory drugs (NSAIDs) can also be used to ease symptoms related to bone pain and secondary osteoarthritis (see Oral paracetamol and NSAIDs for osteoarthritis).

Most patients with symptomatic Paget disease require a bisphosphonate to relieve symptoms and to prevent complications. Asymptomatic Paget disease does not usually warrant treatment; there is no evidence that bisphosphonates prevent bony deformities in asymptomatic patients. However, treatment should be considered for any patient at increased risk of disease progression or complications, including patients who:

  • are young (eg less than 50 years)
  • have a lesion at a critical site (eg skull, vertebrae)
  • have active disease (as indicated by elevated serum total alkaline phosphatase) particularly in weight-bearing bones, near major joints or in the skull or vertebrae
  • have neurological symptoms
  • have hypercalcaemia (which is aggravated by bed rest)
  • require orthopaedic surgery of or near a pagetic bone.

The primary aims of treatment of Paget disease are to relieve symptoms and prevent complications. Normalisation of the serum total alkaline phosphatase concentration is a secondary goal, which usually occurs incidentally with symptom relief. Pagetic lesions do not always normalise with treatment, but symptom relief and normalisation of alkaline phosphatase indicate that the lesions have become inactive.

If the patient remains symptomatic despite normalisation of alkaline phosphatase, consider an alternative cause of the symptoms.

Surgery is sometimes required to manage complications of Paget disease:

  • Secondary osteoarthritis can require joint replacement or another orthopaedic procedure.
  • Neurological syndromes associated with spinal Paget disease often respond to bisphosphonates, but surgical decompression of the spinal cord or cauda equina may be required.
  • Cosmetic or dental deformities related to a pagetic lesion may require oral maxillofacial surgery.