Paget's Disease of Bone

Publication Date: December 1, 2014
Last Updated: October 22, 2024

Diagnosis

Imaging

In patients with suspected Paget’s disease, the Endocrine Society (ES) recommends obtaining plain radiographs of the suspicious regions of the skeleton. ( 1 , H )
700
In patients diagnosed with Paget’s disease, the ES suggests a radionuclide bone scan to determine the extent of the disease and identify possible asymptomatic sites. ( 2 , M )
700

Biochemistry

The ES recommends that after radiological diagnosis of Paget’s disease, the initial biochemical evaluation of a patient should be done using serum total alkaline phosphatase (ALP) or with the use of a more specific marker of bone formation when appropriate. ( 1 , H )
700
The ES recommends measuring a specific marker of bone formation or resorption in patients with Paget’s disease and abnormal liver or biliary tract function to assess response to treatment or follow evolution of the disease in untreated patients. ( 1 , M )
700

Treatment

Indications

The ES recommends treatment with a bisphosphonate (see Table 2) for most patients with active Paget’s disease who are at risk of future complications. ( 1 , M )
700

Choice of Medication

The ES suggests a single 5-mg dose of IV zoledronate as the treatment of choice in patients without contraindications. (2, M)
700

Assessing the Response to Treatment

If there is urgency in the control of symptoms or the disease is particularly active, the ES suggests the use of short-term response of bone resorption markers before and shortly after treatment to indicate that an adequate therapeutic response is likely. ( 2 , L )
700
The ES suggests that patients who have osteolytic lesions of Paget’s disease have a repeat x-ray approximately 1 year after radiological diagnosis to determine whether there has been improvement with therapy or worsening in the absence of therapy. Subsequent x-rays may be considered in the event of persistent elevations of biochemical markers of bone turnover or the presence of bone pain and to determine when there is resolution of the lesion. ( 2 , L )
700

Maintaining Remission

The ES suggests that to maximize the duration of remission, bone turnover should be reduced below the midpoint of the reference range for the chosen monitoring bone turnover marker. ( 2 , L )
700

Relapse and Retreatment

The ES recommends that in patients with increased bone turnover, biochemical follow-up should be used as a more objective indicator of relapse than symptoms. ( 1 , M )
700

Monostotic Paget’s Disease

The ES suggests that amino-terminal propeptide of type 1 collagen (P1NP) or bone-specific ALP (BSAP) and βC-terminal propeptide of type 1 collagen (βCTx) or N-terminal propeptide of type 1 collagen (NTx) should be used for assessing the activity of untreated monostotic Paget’s disease, although these may be normal when evidence of disease activity is still clearly demonstrated on scintigraphy. ( 2 , L )
700

Management of the Complications of Paget’s Disease

Hearing Loss

The ES suggests treatment with a potent bisphosphonate to prevent worsening of a hearing deficit. ( 2 , L )
700

Osteoarthritis

The ES suggests the use of analgesics as adjunctive therapy for mild-to-moderate joint pain due to joint cartilage deterioration in patients with Paget’s disease adjacent to the painful joint. ( 2 , L )
700
For patients with severe osteoarthritis adjacent to Paget’s disease of bone, the ES suggests bisphosphonate therapy before undergoing elective total joint replacement to prevent intraoperative hemorrhaging and postoperative loosening of the prosthesis. ( 2 , L )
700

Bowing of Lower Extremity

The ES suggests treatment with a potent bisphosphonate before elective surgery for patients who require an osteotomy to correct severe bowing of the lower extremity associated with impaired ambulation and/or severe joint pain. ( 2 , L )
700

Paralysis

In cases of paraplegia associated with Paget’s disease of the spine, the ES suggests immediate treatment with a potent IV bisphosphonate associated with neurosurgical consultation. Surgical intervention may not be necessary after effective medical treatment unless there is severe structural damage. ( 2 , L )
700

Neoplasms

The ES suggests that patients with osteosarcoma or a giant cell tumor be evaluated by an orthopedic surgeon (ungraded recommendation). If surgery is planned, the ES suggests pretreatment with a potent bisphosphonate to reduce bleeding from adjacent pagetic bone. ( 2 , L )
700

Congestive Heart Failure

The ES suggests treatment with a bisphosphonate in patients with Paget’s disease and congestive heart failure. ( 2 , L )
700

Recommendation Grading

Overview

Title

Paget's Disease of Bone

Authoring Organization

Endocrine Society

Publication Month/Year

December 1, 2014

Last Updated Month/Year

November 5, 2024

Supplemental Implementation Tools

Document Type

Guideline

External Publication Status

Published

Country of Publication

US

Document Objectives

The aim of this guideline was to formulate practice guidelines for the diagnosis and treatment of Paget's disease of the bone.

Target Patient Population

Patients with Paget's disease of bone

Inclusion Criteria

Male, Female, Adult, Older adult

Health Care Settings

Ambulatory

Intended Users

Nurse, nurse practitioner, physician, physician assistant

Scope

Diagnosis, Treatment, Management

Diseases/Conditions (MeSH)

D010144 - Paget's Disease, Mammary, D010145 - Paget Disease, Extramammary

Keywords

Paget's disease of bone, bone disorders, paget's disease

Source Citation

Frederick R. Singer, Henry G. Bone, III, David J. Hosking, Kenneth W. Lyles, Mohammad Hassan Murad, Ian R. Reid, Ethel S. Siris, Paget's Disease of Bone: An Endocrine Society Clinical Practice Guideline, The Journal of Clinical Endocrinology & Metabolism, Volume 99, Issue 12, 1 December 2014, Pages 4408–4422, https://doi.org/10.1210/jc.2014-2910