From the FDA Drug Label
Paget’s disease of bone (osteitis deformans) is an idiopathic disease characterized by chronic, focal areas of bone destruction complicated by concurrent excessive bone repair, affecting one or more bones. These changes result in thickened but weakened bones that may fracture or bend under stress Signs and symptoms may be bone pain, deformity, fractures, neurological disorders resulting from cranial and spinal nerve entrapment and from spinal cord and brain stem compression, increased cardiac output to the involved bone, increased serum alkaline phosphatase levels (reflecting increased bone formation) and/or urine hydroxyproline excretion (reflecting increased bone resorption)
A Paget's disease workup may involve:
- Evaluating serum alkaline phosphatase levels to assess bone formation
- Assessing urine hydroxyproline/creatinine ratios to evaluate bone resorption
- Monitoring for bone pain, deformity, and fractures
- Checking for neurological disorders resulting from nerve entrapment or compression
- Evaluating cardiac output to the involved bone
- Possibly using imaging studies to assess bone changes, although this is not explicitly mentioned in the provided text 1
- Clinical trials have used single or multi-day infusions of pamidronate disodium to treat Paget’s disease, with response rates and duration of response varying by dose 1
- Another study used a single 5 mg dose of zoledronic acid injection to treat Paget’s disease, with patients experiencing decreases in serum alkaline phosphatase and urine hydroxyproline/creatinine ratios 2
From the Research
A Paget's disease workup typically involves comprehensive blood tests, including serum alkaline phosphatase, and imaging studies such as plain X-rays and bone scans to confirm diagnosis and assess disease severity. The initial evaluation should include blood tests to measure serum alkaline phosphatase (ALP) levels, which are often elevated in patients with Paget's disease, as well as other markers of bone turnover such as calcium, phosphate, P1NP, and NTX 3. Imaging studies are essential, starting with plain X-rays of affected bones to identify characteristic findings like bone enlargement, cortical thickening, and lytic lesions. A bone scan using technetium-99m is particularly valuable to determine the extent of skeletal involvement, showing increased uptake in affected areas 4.
Some key components of a Paget's disease workup include:
- Comprehensive blood tests, including serum alkaline phosphatase and other markers of bone turnover
- Imaging studies, such as plain X-rays and bone scans, to assess the extent of skeletal involvement
- Additional assessments, such as hearing and vision tests, if the skull is involved, and cardiac evaluation if extensive disease is present
- In some cases, CT or MRI scans may be ordered to evaluate complications or rule out other conditions
- Bone biopsy is rarely needed but may be performed if the diagnosis remains uncertain or to exclude malignancy
According to the most recent and highest quality study, the use of plain radiography and serum alkaline phosphatase testing is recommended for initial diagnosis, and radionuclide scans for delineation of the extent of disease 3. This comprehensive workup helps clinicians not only confirm Paget's disease but also determine its extent and activity, which guides treatment decisions regarding bisphosphonate therapy or other interventions.