From the Guidelines
Paget's disease of bone is a chronic disorder that requires prompt treatment with bisphosphonates, such as zoledronic acid or oral alendronate, to manage symptoms, prevent complications, and improve quality of life. The condition is characterized by abnormal bone remodeling, leading to enlarged and deformed bones, and typically affects the skull, spine, pelvis, and long bones, causing pain, fractures, and deformities 1.
Diagnosis and Differential Diagnosis
The diagnosis of Paget's disease can be challenging, and it is essential to consider other conditions that may present with similar symptoms, such as osteomalacia, hypophosphatasia, and fibrous dysplasia 1. A thorough medical history, physical examination, and imaging studies, including bone scans and X-rays, can help establish the diagnosis.
Treatment Options
Treatment depends on symptom severity and often includes bisphosphonates, which inhibit bone resorption. Zoledronic acid (5mg IV once) or oral alendronate (40mg daily for 6 months) are commonly used options 1. For pain management, NSAIDs or acetaminophen may be prescribed. In severe cases with significant deformities, orthopedic surgery might be necessary.
Monitoring and Prevention
Regular monitoring with bone scans, alkaline phosphatase levels, and X-rays helps track disease progression. Patients should maintain adequate calcium and vitamin D intake to support bone health. It is also essential to address any underlying conditions, such as vitamin D deficiency or hypogonadism, that may contribute to bone disease 1.
Key Considerations
The disease results from overactive osteoclasts (bone-destroying cells) followed by disorganized bone formation by osteoblasts, creating structurally weak bone. While Paget's disease cannot be cured, proper treatment can effectively manage symptoms, prevent complications, and improve quality of life. A multidisciplinary approach, including orthopedic surgery, physical therapy, and medical management, may be necessary to optimize patient outcomes 1.
From the Research
Paget's Disease of Bone Deformity
- Paget's disease of bone is a condition characterized by localized areas of excessive bone resorption coupled with accelerated bone formation, resulting in new bone that is less structurally organized and weaker than normal bone 2.
- Complications of Paget's disease can include bone pain, osteoarthritis, skeletal deformity, hearing loss, and fractures 2.
- Bisphosphonates are the mainstay of treatment for Paget's disease of bone, and they work by suppressing the elevations in bone turnover that are characteristic of the disease 3, 4, 5.
Treatment Options
- Clinical practice guidelines recommend treatment with intravenous zoledronic acid or high-dose oral nitrogen bisphosphonates (N-BPs) 3.
- A study found that a single infusion of zoledronic acid produces more rapid, more complete, and more sustained responses in Paget's disease than does daily treatment with risedronate 4.
- Another study found that remission can be achieved with once-weekly, "osteoporosis doses" of alendronate or risedronate in a large proportion of "real world" PDB patients 3.
Effects of Bisphosphonates on Complications
- The effects of bisphosphonates on complications of Paget's disease such as deformity, pathological fractures, and deafness have not been adequately studied 5.
- A randomized trial found that intensive bisphosphonate therapy aimed at normalizing ALP was no more effective than symptom-directed treatment with bisphosphonates at preventing complications of PDB 6.
- The Zoledronate in the Prevention of Paget's Disease (ZiPP) trial is currently in progress to determine whether early intervention with zoledronic acid might be effective in preventing disease progression 5.