Paget's Disease : Therapies, When to Treat?

by Food and Drug Administration (FDA)

(Page 3 of 3)

In clinical studies, patients receiving Fosamax had a 20 to 25 percent greater drop in serum alkaline phosphatase levels than those receiving Didronel. The drop was up to 65 percent greater compared with placebo, which had little effect on alkaline phosphatase levels. Bone tissue studies indicated that normal bone was produced during treatment with Fosamax, even where preexisting bone had the abnormally disorganized pattern characteristic of Paget's disease.

Fosamax and Didronel usually are taken for no longer than six months at a time. If symptoms worsen or laboratory tests indicate a worsening of the disease, the drugs may be restarted after at least a six-month break from the medications.

Additional Therapies

According to the Paget Foundation, several more bisphosphonate drugs are undergoing clinical tests. These drugs may offer greater ease of use, says Charlene Waldman, executive director of the Paget Foundation.

Because new bone formation occurs as part of the process of repair in pagetic bone, it is important that along with calcitonin and the bisphosphonates to inhibit abnormal bone breakdown, patients eat a diet that provides 1,000 to 1,500 milligrams of calcium and 400 International Units of vitamin D daily. These nutrients are needed for proper bone formation.

Calcium can be obtained by eating a well-balanced diet that includes foods that are good sources of calcium — for example, milk and milk products, dark-green leafy vegetables (such as mustard greens and kale), and canned fish with soft bones (such as sardines and salmon). Dietary supplements of calcium may be another source.

Some Paget's patients, especially those with severe bowing of legs, fractures, and degenerative arthritis, may need splints, braces, and other devices such as canes and walkers. Patients also may receive physical therapy.

Although uncommon, surgery may be required, especially in cases of fractured bones, severe arthritis, and progressive deformity of leg bones.

Exercise is important for patients with Paget's disease, just as it is for everyone. Because patients with Paget's disease are prone to bone fractures, they should consult their doctors or physical therapists before starting an exercise program.

Various laboratory tests monitor the progression of Paget's disease. The most common is the total alkaline phosphatase. FDA has cleared two tests — Hybritech Ostase in 1994 and Metra Alkphase-B in 1995 — that measure only the alkaline phosphatase from bone, since the enzyme in the blood can come from other organs, too.

A possible future test, which is still under research, would measure osteocalcin, a byproduct of osteoblasts, to determine bone turnover rates.

Deciding When to Treat

For many years, doctors generally treated patients with Paget's disease only if they had symptoms. In recent years, with the availability of a wider range of drugs, doctors have begun treating patients without symptoms, as well, hoping that the drugs may prevent the effects of Paget's disease. Factors to consider in deciding whether to treat patients without symptoms, according to Siris, are the location of the disease and the likelihood of its progression. Diseased bone near joints, in the spine or skull, or in the leg bones are particularly "bad spots," she says, and may indicate the need for drug therapy.

Patients who are told they have Paget's disease may want to seek a medical specialist in that condition. The Paget Foundation recommends endocrinologists (doctors who specialize in hormonal and metabolic disorders) or rheumatologists (doctors who specialize in joint and muscle disorders). Orthopedic doctors (who specialize in bone problems), neurologists (doctors who specialize in nerve disorders), and otolaryngologists (eye, ear, nose, and throat specialists) also may be called on to evaluate specific symptoms.

Siris and Michael McClung, M.D., an endocrinologist and director of the Oregon Osteoporosis Center in the Oregon Health Sciences University in Portland, say that too often doctors who aren't specialists in the disease fail to follow up on laboratory tests or x-rays that indicate Paget's disease. "They might tell patients: 'Forget about it. You'll just end up in a wheelchair,'" Siris says. She believes that many doctors aren't aware of current treatments because effective drugs for Paget's disease weren't available when they were trained.

Since Paget's disease often runs in families, medical experts recommend that people with a family history of Paget's disease have their serum alkaline phosphatase measured after age 40, since the disease rarely shows up in people under 40. The laboratory test can be done as part of the routine medical exam.

With prompt medical attention and treatment, when needed, people with Paget's disease may be able to avoid some of the disease's serious, often painful effects.

Maryland resident Brown hopes that will be true for her. "[The disease] is foremost in my mind," she says. "I wonder: 'Am I going to suffer any deformities from this?' I don't know. But I must be treated or so many things could happen."

About the Author

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