Guidelines for Paget’s Disease of the Bone Released

Guidelines for Paget's Disease of the Bone Released
Guidelines for Paget’s Disease of the Bone Released
The Endocrine Society has released a clinical practice guideline for the diagnosis and treatment of Paget's disease of the bone.

A one-time intravenous infusion of the osteoporosis drug zoledronate may be the treatment of choice for Paget’s disease of the bone, according to a new clinical practice guideline issued by the Endocrine Society.

“We’ve long known that bisphosphonates — a class of drugs often used to treat osteoporosis and other bone conditions — work well for treating Paget’s disease,” Frederick R. Singer, MD, of the John Wayne Cancer Institute in Santa Monica, California, and chair of the task force that authored the guideline, said in a press release from the Endocrine Society.

“One particular option — a one-time IV infusion of zoledronate — has emerged as the preferred option. The medication can put Paget’s disease into remission for up to 6 years, and many patients prefer the one-time infusion to oral medications that can cause gastrointestinal side effects and must be taken over the course of several months,” Dr. Singer said.


In the guideline, the task force recommends that bisphosphonates be prescribed for patients with active Paget’s disease at risk for future complications. In particular, a single 5-mg dose of IV zoledronate is suggested as first-line therapy unless it is contraindicated, such as in patients with reduced kidney function.

Other recommendations include:

  • Plain radiographs should be taken of pertinent regions of the skeleton in patients with suspected Paget’s disease as part of the diagnostic process.
  • If diagnosis is confirmed, a radionucleotide bone scan should be performed to determine the extent of the disease.
  • After diagnosis, measurement of total serum alkaline phosphatase (ALP) levels or, if warranted, a more specific marker of bone formation or bone resorption should be performed to evaluate treatment response or evolution of disease in untreated patients.
  • In patients with monostatic disease with normal serum total ALP levels, a specific marker of bone formation or bone resorption should be measured, although these may also be normal.
  • Patients should undergo testing for bone markers to determine if they are relapsing, as many patients do not report feeling pain when disease activity recurs.

Additionally, the task force suggest that bisphosphonate treatment may help prevent or slow hearing loss or osteoarthritis in the joints adjacent to Paget’s disease and potentially reverse paraplegia associated with spinal Paget’s disease.

They also suggest treatment with a bisphosphonate before surgery on pagetic bone.

Paget’s Disease of the Bone

Normally, the body breaks down old bone tissue and replaces it with new bone, but in Paget’s disease of the bone, the new tissue can grow too large and weak, according to the release.

About one million people nationwide have Paget’s disease, according to information from the NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases, with an estimated 2% to 3% of people aged older than 55 years having the condition. However, it is rare in people aged younger than 40 years.

The goal of the clinical practice guideline, which was co-sponsored by the European Society of Endocrinology, was to formulate recommendations for the diagnosis and treatment of Paget’s disease. It will be published in the December 2014 issue of the Journal of Clinical Endocrinology & Metabolism.


  1. Singer FR et al. J Clin Endocrinol Metab. 2014;doi:10.1210/jc.2014-2910.