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

Abstract

An evidence-based clinical guideline for the diagnosis and management of Paget's disease of bone (PDB) was developed using GRADE methodology, by a Guideline Development Group (GDG) led by the Paget's Association (UK). A systematic review of diagnostic tests and pharmacological and nonpharmacological treatment options was conducted that sought to address several key questions of clinical relevance. Twelve recommendations and five conditional recommendations were made, but there was insufficient evidence to address eight of the questions posed. The following recommendations were identified as the most important: 1) Radionuclide bone scans, in addition to targeted radiographs, are recommended as a means of fully and accurately defining the extent of metabolically active disease in patients with PDB. 2) Serum total alkaline phosphatase (ALP) is recommended as a first-line biochemical screening test in combination with liver function tests in screening for the presence of metabolically active PDB. 3) Bisphosphonates are recommended for the treatment of bone pain associated with PDB. Zoledronic acid is recommended as the bisphosphonate most likely to give a favorable pain response. 4) Treatment aimed at improving symptoms is recommended over a treat-to-target strategy aimed at normalizing total ALP in PDB. 5) Total hip or knee replacements are recommended for patients with PDB who develop osteoarthritis in whom medical treatment is inadequate. There is insufficient information to recommend one type of surgical approach over another. The guideline was endorsed by the European Calcified Tissues Society, the International Osteoporosis Foundation, the American Society of Bone and Mineral Research, the Bone Research Society (UK), and the British Geriatric Society. The GDG noted that there had been a lack of research on patient-focused clinical outcomes in PDB and identified several areas where further research was needed. © 2019 The Authors. Journal of Bone and Mineral Research Published by Wiley Periodicals Inc.

Keywords: ALP; ANTIRESORPTIVES; BISPHOSPHONATES; PAGET'S DISEASE OF BONE; RADIONUCLIDE BONE SCANS.

Figure 1

X‐ray features of PDB. Pelvic radiograph from a patient with PDB affecting the upper right femur showing alternating areas of osteolysis and osteosclerosis in the greater and lesser trochanters and femoral neck; loss of distinction between the cortex and medulla in the upper femur; bone expansion and deformity of the affected femur; and a pseudofracture on the lateral aspect of the femur opposite the lesser trochanter.

Figure 2

Diagnosis and monitoring of Paget's disease. ALP = total alkaline phosphatase; BALP = bone‐specific alkaline phosphatase; PINP = procollagen type I N‐terminal propeptide; uNTX = urinary cross‐linked N‐terminal telopeptide of type I collagen.

Figure 3

Management of Paget's disease.