Many people with Paget's disease require no treatment because they have no symptoms. The most clear-cut reasons for using drugs to treat Paget’s disease are bone pain, bone fractures (such as vertebral fractures), and compression of nerves in the spine or elsewhere.

The FDA has approved injectable calcitonin, four oral bisphosphonate drugs (a class of drugs used to strengthen bone), and two intravenous bisphosphonate drugs for treatment of Paget’s disease. Calcitonin (a synthetic version of a hormone that acts on bone) has pain-relieving properties and may be used in conjunction with a bisphosphonate, especially if additional pain relief is desired. No matter which drug is started first, it can be discontinued and replaced with another agent if it proves ineffective.

Calcitonin

Calcitonin (Miacalcin), which inhibits bone breakdown (resorption), is available for subcutaneous (under the skin) or intramuscular injection. Although it is also available as a nasal spray, the nasal formulation of calcitonin is not approved by the FDA for treating Paget’s disease. Injections are initially given daily; after a month or so, symptoms may be controlled with less frequent injections (three times a week, for example).

After a variable period of time, calcitonin stops working in some people. Between 10 and 20% of those treated with calcitonin develop nausea, loss of appetite, and flushing of the face and ears.

Bisphosphonates

The bisphosphonate class of drugs includes the oral medications alendronate (Fosamax), etidronate (Didronel), risedronate (Actonel), and tiludronate (Skelid). These drugs are the first line of therapy for the treatment of Paget’s disease, and they are also used to pre- vent and treat osteoporosis.

For Paget’s disease, Fosamax (40 mg a day) and Didronel (200 to 400 mg a day) are both taken in a six-months-on, six-months-off cycle. Actonel (30 mg a day) is taken in a two-months-on, two-months-off cycle and Skelid (400 mg a day) is taken in a three-months-on, three- months-off cycle. If relapse occurs, a person may begin treatment again after two or three months off the drug.

If an oral bisphosphonate is no longer effective or cannot be tolerated because of gastrointestinal side effects or if Paget’s disease is particularly severe, the intravenous bisphosphonate drugs pamidronate (Aredia) and zoledronic acid (Reclast) may be used. Reclast was approved by the U.S. Food and Drug Administration in April 2007 for the treatment of Paget’s disease. Previously, zoledronic acid had been approved under the brand name Zometa for the treatment of cancer-related bone problems.

Aredia is administered using an injection schedule determined by the doctor. Possible side effects include abdominal cramps, chills, fever, nausea, pain, muscle spasms, sore throat, and muscle stiffness. Reclast is given in one 15-minute intravenous infusion. Potential side effects are similar to those of Aredia.

Additional Paget’s Disease Treatments

People with Paget’s disease should also take 1,000 to 1,200 mg of calcium and 400 IU of vitamin D each day. People with signs and symptoms of spinal stenosis resulting from Paget’s disease may require surgery if medication is ineffective in reducing the stenosis.

Publication Review By: Lee H. Riley III, M.D., and Suzanne M. Jan de Beur, M.D.

Published: 04 Aug 2011

Last Modified: 18 Nov 2011