Treatment for my patient

The Paget's Association supports all those involved in the care of patients with Paget’s disease. We hope you find the information provided helpful. Please consider becoming a member to receive our quarterly newsletter and access the resources in the Professional Member's Area of this website.

Information Sheet: Download an information sheet to give your patient: Download Paget's Essential Facts 2019

Who needs treatment for Paget’s disease?

In many cases, Paget’s disease is found by chance, does not cause any symptoms and does not require any treatment. Referral to a consultant is recommended in order to carry out a full assessment and decide management. Assessment usually includes an isotope bone scan and family history.

Centres of Excellence for care of patients and research: You can find the list of centres by following this link

Clinical Guideline

26th Feb 2019 - The new Guideline for the diagnosis and management of Paget’s Disease of Bone, in adults, has now been published - follow this link for details. You can download a summary of the Guideline here Paget's Guideline Handout 2019

Treatment with Bisphosphonates

Paget’s disease does not always cause any symptoms and not everyone needs treatment. When treatment is required, Bisphosphonates are used and Zoledronate is often the drug of choice as it acts quickly and can be effective for many years. 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. 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. It can take several months for bisphosphonates to have their full effect and for the individual to feel the maximum benefit.

Alkaline Phosphatase (ALP) is an indicator of disease activity and should be measured prior to starting treatment and repeated 2-3 months later when it should have decreased. It should be noted that Paget's disease can be active and yet the alkaline phosphatase (ALP) may remain within the normal range, particularly if the area involved is small.

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 can be obtained by contacting the Paget's Association on 0161 7994646 or email [email protected]

Zoledronate
Zoledronic acid (zoledronate)

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.
The current first-line bisphosphonate used to treat Paget’s disease is zoledronic acid.

5 mg of Zoledronic acid intravenously over 15-30 minutes 

• One dose may be effective for many years therefore often patients may not require further treatment

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. You can read the new Guideline by following this link.

Pamidronate

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

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.

Pamidronate: given intravenously over 2-4 hours.  Dosage (60 – 90 mg) and frequency will depend on the severity of symptoms.

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

    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.

Case Histories

Considerations in Diagnosis & Management of Paget’s Disease

Dr Clive Kelly is a Consultant Rheumatologist in Gateshead. Speaking at our Paget's Information Day in Middlesbrough, in 2014, Dr Kelly discussed three case histories of patients with Paget’s disease. None were straight forward and so they show the difficulties that can be encountered when diagnosing and treating Paget’s disease. Dr Kelly has kindly provided summaries of the individual cases below.

Experienced clinicians often look upon Paget’s disease as an easy diagnosis to make and a condition that is readily treated. Although treatment of the condition can be much easier these days, there are however, still some pitfalls in both the diagnosis and management of the condition. These are illustrated by the three patient summaries below which highlight some of these issues.

Case 1

A 76 year old gentleman had suffered with back pain for three months, associated with sleep disturbance and difficulty in micturition. Examination showed tenderness in the lower back on deep palpation, although the spinal architecture was normal. His blood tests showed an abnormal level of calcium, raised alkaline phosphatase (ALP) and a prostatic specific antigen (PSA) of 10 (normal 0-5). Plain x-rays of the lumbar spine demonstrated bony sclerosis in a vertebrae (L4) which was not present on his previous x-ray, taken five years before.
The trap here is to assume that the features represent Paget’s disease without exclusion of other possibilities. This patient may have prostatic cancer with bony metastasis as this may mimic Paget’s disease. The elevated PSA and micturition difficulty makes it important to refer him to a Urologist for further investigation. If a biopsy of the prostate or an MRI scan of the spine show evidence of cancer, then the treatment would be with hormonal manipulation therapy to treat the prostatic cancer. If however, investigations show monostotic Paget’s disease (Paget’s in just one bone), then therapy with a single infusion of 5 mg of intravenous Zoledronic acid may suffice to control his symptoms indefinitely.

Case 2

An 83 year old lady presented with increasing pain in her right hip causing limited mobility. She could hardly climb the stairs in her house and the pain was disturbing her sleep. Examination showed features of generalised osteoarthritis in her fingers,while her right hip demonstrated marked reduction in rotation and pain on attempted flexion. Blood tests showed a slight rise in alkaline phosphatase, while a plain x-ray of her hip confirmed severe osteoarthritis with narrowing of the joint space and sclerosis. There was however, also widening of the trabeculae around the neck and head of femur with some expansion of the femoral head and variation in bone density at the same site.
The mistake here would be to assume that all her symptoms were due to osteoarthritis of the hip and to anticipate that a total hip replacement would solve her problems. In this situation, co-existing Paget’s disease is important to recognise and treat, as surgery may be likely to fail otherwise as a consequence of bony softening and / or fracture. A single infusion of Zoledronic acid may suffice to relieve symptoms and improve bone quality and density in the region of the hip, permitting elective surgery three to six months later if pain persists. The intravenous bisphosphonate alone, however, may lead to significant symptomatic improvement.

Case 3

An 88 year old man presented to his GP with low back pain, radiating down the left leg. Examination showed a reduction in range of movement of the lumbar spine with little tenderness. Straight leg lift was reduced on the left and his ankle jerk and strength at the ankle were both reduced. Blood tests including alkaline phosphatase, calcium and PSA were all normal although a reduction in renal function was noted. A plain x-ray showed features of degenerative disc disease, most marked at the lumbosacral junction. There was also an area of abnormal bone in the right ilium, with sclerosis and coarsened trabeculae, strongly suggestive of Paget's disease, but no fracture was seen. These features were present on a previous x-ray 3 years earlier.
The patient appeared to have a disc prolapse at the lumbosacral junction with a resulting left radiculopathy causing symptoms. The radiographic features of his Paget's disease were unlikely to be associated with any symptoms, and the normal blood tests and stability of the radiographic changes made it most likely that his Paget's disease was neither active nor rapidly progressive. Treatment in this situation may actually carry more risk than benefit as there is no evidence that the treatment of asymptomatic lesions at this site alter outcome.

Summary

In summary, Pagets disease is common among the elderly and is easily treated in most cases with a single infusion of Zoledronic acid if renal function, calcium and vitamin d levels are adequate. Although further infusions can be given, in practice these are rarely needed at intervals of less than three years. Failure to correct vitamin D can precipitate hypocalcaemia and administration to patients with a glomerular filtration rate of less than 30 mls/minute, can also precipitate toxicity. Hence treatment of asymptomatic lesions in non-weight bearing bones may be avoided if the bone chemistry suggests low disease activity.