Assessment and Treatment

Treatment may be recommended, particularly if the affected bones are painful. Zoledronate is often the drug of choice. Given as a single dose into the bloodstream, it can be effective for several years. See below for more treatment options.

Referral for assessment: In some cases, Paget’s disease is found by chance, does not cause any symptoms and requires no treatment. Referral to a hospital consultant, however, is recommended so that a full assessment can take place. See below for more detail regarding referral including Paget's Association Centres of Excellence.

Referral for assessment

When Paget’s disease is suspected, it is important that there is a detailed assessment process, ideally carried out by a hospital consultant who understands the condition. The consultant will consider symptoms, enquire about any family history of the condition, and ensure that appropriate investigations are carried out.

Many rheumatologists and endocrinologists have the expertise to treat patients with Paget’s disease and it is likely that most hospitals in the UK have a specialist who can deal with the condition. If there is no specialist locally, then referral to one of the Paget’s Association Centres of Excellence might be worthwhile.

In some cases Paget’s disease does not cause any symptoms and requires no treatment, however, a good assessment is still necessary.

  • Treatment may be recommended if the affected bones are painful
  • Sometimes treatment is given if Paget's disease affects a site that might be expected to cause complications

The website of your local hospital may list your local specialists. They are often (not always) found in the rheumatology or endocrinology department. In some areas, there may be a metabolic bone centre or clinic.

Centres of Excellence

The Paget's Association has awarded Centre of Excellence status to several hospital and university departments which demonstrate excellence in both the treatment of Paget’s disease and research into the condition. Details can be found by following this link.

Treatment with Bisphosphonates

Paget’s disease does not always cause any symptoms and not everyone needs treatment. The main reason for treatment is if the affected bones are painful. If the pain is directly from Paget’s disease, it often improves with treatment. Pain, however, can arise from complications.

Bisphosphonates

Bisphosphonates are drugs which work by inhibiting the bone remodelling process. These drugs reduce abnormal bone destruction by the osteoclasts, thereby restoring a more normal remodelling process.

For those who require treatment, the current first-line bisphosphonate, due to its potency and prolonged duration of action, is zoledronic acid. It is the bisphosphonate most likely to relieve pain from active Paget’s disease.

The main treatment goal is to control bone pain. The clinical benefit of giving bisphosphonates to patients who have a raised level of alkaline phosphatase in their blood, but do not have symptoms, is unknown.

Log-in or register to view the Members' Area of this website where you will find further information regarding treatment and the potential side-effects of Bisphosphonates.

New Paget's Guideline

A new clinical Guideline, for the diagnosis and management of Paget’s Disease of Bone in adults, was commissioned by the Paget's Association. The full Guideline has now been published in the Journal of Bone and Mineral Research. It has been endorsed by the European Calcified Tissues Society, the International Osteoporosis Foundation, the American Society of Bone and Mineral Research, the Bone Research Society (UK), and the British Geriatric Society.

You can read the new Guideline by following this link.

New Facts Booklet. The Paget's Association has updated its booklet,  'Paget's - The Facts', in line with the new Guideline. This will be available in the coming weeks to download from the member's area of this website and also by contacting the Paget's Association on 0161 7994646 or email [email protected]

Intravenous Preparations
Zoledronic acid

For those who require treatment, the current first-line bisphosphonate, due to its potency and prolonged duration of action, is zoledronic acid. It is the bisphosphonate most likely to relieve pain from active Paget’s disease. It is usually given in hospital, as an outpatient. A single dose of 5mg is given through an infusion (a drip) directly into the bloodstream (intravenous), over 15 minutes. Over the following months, this treatment often normalises the abnormal bone remodelling and one dose can be effective for many years.

Pamidronate

Pamidronate is an effective treatment but has largely been superseded by zoledronic acid which lasts longer and is easier to administer. Pamidronate is given in several doses, intravenously (an infusion into the bloodstream), and repeated when necessary, dependant on symptoms. Doses can vary, but commonly 60mg is given by an infusion over a period of four hours and this is repeated on three consecutive days.

What about side effects?

A comprehensive review on the effects of bisphosphonates in the treatment of Paget’s disease, published in 2017, concluded that serious side effects were rare and that the most common side effects experienced were a flu-like illness in people given bisphosphonates by infusion, and stomach upset in those given tablets. Severe side effects causing treatment discontinuation were rare.

The most common side effect with zoledronic acid is a flu-like reaction that lasts a day or two in most people who experience it. It is usually of mild or moderate severity, but more severe reactions can sometimes occur. Pamidronate can also cause a flu-like reaction but there is less information on the frequency with which this occurs or its severity. If a flu-like reaction does occur with either drug, it can usually be controlled with paracetamol or ibuprofen.

Both Zoledronic acid and pamidronate may cause a decrease in calcium levels. The risk of this is reduced in people who have a good dietary calcium intake and those with normal levels of vitamin D. Sometimes vitamin D levels (25-hydroxy vitamin D) might be measured before treatment to determine if supplements are required. Depending on the results, calcium and vitamin D supplements may be given prior to treatment as a preventative measure.

The most common side effect of taking risedronate is stomach upset.

On rare occasions, bisphosphonates are associated with osteonecrosis of the jaw (ONJ). This is a condition which may present after dental surgery when exposed bone fails to heal. ONJ is rarely seen in those with Paget’s disease and the risk of ONJ is greater if bisphosphonates are given for cancer. As a precaution however, if possible, complete any extensive dental treatment prior to bisphosphonate treatment. If you have to undergo extensive dental procedures, it is important to inform your dentist that you are having, or have had treatment with a bisphosphonate.

You should discuss your individual circumstances with your doctor, but if bisphosphonate treatment is required, the benefits often outweigh the risk of any potential side effects.

When should bisphosphonates be avoided?

The decision to give treatment may be modified if you have another medical problem which could be aggravated by bisphosphonates. As bisphosphonates are excreted by the kidneys they cannot be used if there is significant kidney disease. If you are being considered for bisphosphonate treatment the doctor will usually organise a blood test to check kidney function, to see if it is safe to go ahead. Although bisphosphonates are rarely given to younger people, they should be avoided during pregnancy as their effects on the foetus are unknown.

Booklets containing more detailed information are available to download in the Member's Area of this website. Alternatively, they can be posted to you. Please telephone 0161 799 4646 or use the contact form.

Oral Preparations

Intravenous treatment with zoledronic acid is usually the first consideration, as it acts quicker and lasts longer, however, treatment can be given orally.

Alternatives to Zoledronic acid
Risedronate

Bisphosphonates can also be given as tablets; these are slightly less effective than an infusion of zoledronic acid, at reducing bone remodelling, and the effect doesn’t last quite as long. The most commonly used oral treatment is 30mg of risedronate sodium, taken daily, for two months. If necessary, the course can be repeated.

For risedronate to be effective, it is important that you do not take it with food, other medicines or drinks (other than plain water). If taken at the same time, medicines containing any of the following lessen the effect of risedronate: calcium, magnesium, aluminium (e.g. some indigestion mixtures) or iron. To ensure risedronate is absorbed properly, it should be taken with a full glass of water, on an empty stomach, first thing in the morning. These tablets can irritate your oesophagus (the tube that takes food into your stomach) therefore, sit upright or stand for at least 30 minutes after taking it. Wait at least 30 minutes before eating or drinking (other than water), and before taking other medication.

Further treatment may be given after 6 months.

What about side effects?

A comprehensive review on the effects of bisphosphonates in the treatment of Paget’s disease, published in 2017, concluded that serious side effects were rare and that the most common side effects experienced were a flu-like illness in people given bisphosphonates by infusion, and stomach upset in those given tablets. Severe side effects causing treatment discontinuation were rare.

The most common side effect with zoledronic acid is a flu-like reaction that lasts a day or two in most people who experience it. It is usually of mild or moderate severity, but more severe reactions can sometimes occur. Pamidronate can also cause a flu-like reaction but there is less information on the frequency with which this occurs or its severity. If a flu-like reaction does occur with either drug, it can usually be controlled with paracetamol or ibuprofen.

Both Zoledronic acid and pamidronate may cause a decrease in calcium levels. The risk of this is reduced in people who have a good dietary calcium intake and those with normal levels of vitamin D. Sometimes vitamin D levels (25-hydroxy vitamin D) might be measured before treatment to determine if supplements are required. Depending on the results, calcium and vitamin D supplements may be given prior to treatment as a preventative measure.

The most common side effect of taking risedronate is stomach upset.

On rare occasions, bisphosphonates are associated with osteonecrosis of the jaw (ONJ). This is a condition which may present after dental surgery when exposed bone fails to heal. ONJ is rarely seen in those with Paget’s disease and the risk of ONJ is greater if bisphosphonates are given for cancer. As a precaution, however, if possible, complete any extensive dental treatment prior to bisphosphonate treatment. If you have to undergo extensive dental procedures, it is important to inform your dentist that you are having, or have had treatment with a bisphosphonate.

You should discuss your individual circumstances with your doctor, but if bisphosphonate treatment is required, the benefits often outweigh the risk of any potential side effects.

When should bisphosphonates be avoided?

The decision to give treatment may be modified if you have another medical problem which could be aggravated by bisphosphonates. As bisphosphonates are excreted by the kidneys they cannot be used if there is significant kidney disease. If you are being considered for bisphosphonate treatment the doctor will usually organise a blood test to check kidney function, to see if it is safe to go ahead. Although bisphosphonates are rarely given to younger people, they should be avoided during pregnancy as their effects on the foetus are unknown.

Booklets containing more detailed information are available to download in the Member's Area of this website. Alternatively, they can be posted to you. Please telephone 0161 799 4646 or use the contact form.

Other medication

Painkillers may be required. Not all those with Paget’s disease experience pain. Pain is however, the commonest presenting symptom of Paget’s disease.

PAIN

Pain can be a warning that something is wrong and should not be ignored. It is therefore important to have pain assessed by your doctor. Pain in Paget’s disease may be directly related to the disease itself as a consequence of increased bone activity. Pain can also occur as a result of damage to the adjacent joints (osteoarthritis), a break in the bone (fracture), pressure on the nerves from enlarged bone or as the result of deformity of the bones, which can put stress on joints and soft tissues. In rare cases (less than 1%) pain can arise from a cancerous change within the bone (sarcoma).

You will find useful information about dealing with pain in our Booklet "Paget's Disease and Pain". This can be downloaded from the Member's Area of this website, or you can contact us to request a copy.

Surgery

As Paget’s disease near a major joint may increase the risk of osteoarthritis, joint replacement surgery may be required.

Fractures: If a fracture cannot be stabilised with a plaster cast, surgery may then be required.

Surgery is occasionally required to correct marked deformity.

For more information see our Paget's Disease - The Facts booklet and our Newsletters available in the Member's Area of this website.