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Paget disease: when to treat and when not to treat

Nature Reviews Rheumatology volume 5, pages 483–489 (2009)Cite this article

Abstract

Paget disease of bone is a focal disorder of the skeleton that can affect one or more bones. Many patients are discovered accidentally because of elevated serum alkaline phosphatase activity or an abnormal skeletal radiograph intended to evaluate an unrelated condition. Patients are often asymptomatic, but a subset experience considerable morbidity that can include bone pain and skeletal deformity, as well as a variety of regional complications, such as hearing loss associated with cranial involvement, degenerative arthritis of the hip or knee, fractures of the lower extremities and, rarely, sarcoma or giant cell tumors. Bisphosphonates have proven to be effective in controlling disease activity because they inhibit osteoclast function. Administration of these agents can relieve bone pain, decrease biochemical markers of bone resorption and bone formation, and retard or reverse the early osteolytic phase of the disease. Future studies are needed to determine whether these drugs, if used in an early stage of the disease, can prevent complications in asymptomatic patients.

Key Points

  • Paget disease evolves over many years

  • Many patients are asymptomatic

  • Bone pain, deformity and local complications such as degenerative arthritis might require drug therapy or orthopedic surgery, or both

  • Bisphosphonates are highly effective in relieving bone pain and suppressing the increased metabolic activity in the skeleton

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Figure 1: Cortical changes in an untreated patient with Paget disease.
Figure 2: Effects of salmon calcitonin on osteolytic lesions in a patient with Paget disease.

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Acknowledgements

The author appreciates the long-term support of the Edythe and Eli Broad Foundation, and of Lois Rosen. Charles P. Vega, University of California, Irvine, CA, is the author of and is solely responsible for the content of the learning objectives, questions and answers of the MedscapeCME-accredited continuing medical education activity associated with this article.

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  1. John Wayne Cancer Institute at Saint John's Health Center, Santa Monica, CA, USA

    Frederick R. Singer

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Correspondence to Frederick R. Singer.

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F. R. Singer has acted as a consultant for Amgen, and has acted as a consultant, been involved in speakers bureaus (honoraria) and has received grant/research support (including for clinical trials) for Merck, Novartis and Proctor and Gamble.

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Singer, F. Paget disease: when to treat and when not to treat. Nat Rev Rheumatol 5, 483–489 (2009). https://doi.org/10.1038/nrrheum.2009.149

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