Diagnosis and Management of Paget's Disease of Bone in Adults: A Clinical Guideline

This guideline differs from previous guidelines published on this subject(58–60) in that we considered both pharmacological and nonpharmacological treatment options; in that we had patient representation on the guideline development group and sought feedback from patients in the peerreview process; and in that we have provided information on the key questions used to develop the guideline, as well as details of the search strategy and numbers of publications that were reviewed for each key question

Stuart H Ralston

2019

Scholarcy highlights

  • Paget’s disease of the bone is a nonmalignant skeletal disorder characterized by focal abnormalities in bone remodeling at one or more skeletal sites
  • This guideline differs from previous guidelines published on this subject in that we considered both pharmacological and nonpharmacological treatment options; in that we had patient representation on the guideline development group and sought feedback from patients in the peerreview process; and in that we have provided information on the key questions used to develop the guideline, as well as details of the search strategy and numbers of publications that were reviewed for each key question
  • Specific anti-Pagetic treatment involves the use osteoclast inhibitors to reduce the elevations in bone turnover that are characteristic of active disease, whereas other treatments such as analgesics, nonsteroidal antiinflammatory drugs, and anti-neuropathic agents are used for symptom control. We have mainly focused on the use of bisphosphonates, which are currently considered to be the treatment of choice for PDB and which are the only agents that have been evaluated in randomized controlled trials
  • In one observational study of PDB patients treated with pamidronate, healing of lytic lesions was demonstrated in some cases at 6 months, but longer-term follow-up of these patients after 2 years showed progression of lytic lesions once again, even though biochemical markers of bone turnover were normal at this point. The effects of bisphosphonates on lytic lesions have been studied in two randomized controlled trials
  • This guideline is the result of a comprehensive systematic review on the diagnosis and management of PDB, which considered both pharmacological and nonpharmacological treatment options
  • The bone resorption markers ubCTX, sbCTX, uNTX, and sNTX significantly predicted lesion extent assessed by scintigraphy after bisphosphonate treatment with values of 0.563, 95% CI 0.297–0.748 for ubCTX; 0.639, 95% CI for sbCTX 0.401–0.796; and 0.674, 95% CI 0.518–0.787 for uNTX
  • We have made recommendations in 12 areas and conditional recommendations in five, the Guideline Development Group noted that for several outcomes of clinical importance to patients, there was insufficient evidence to answer the questions posed in this guideline due to the fact that most clinical trials in PDB had been short term and focused on biochemical markers as the primary outcome, rather than patient-reported outcome measures
  • The Guideline Development Group felt that further research into PDB is warranted and identified the following topics as areas where research would be warranted

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