From the Guidelines
Paget's disease of bone should be treated with bisphosphonates, such as zoledronic acid, to reduce the risk of complications, including osteosarcoma, which can develop as a secondary malignancy in adults over 65 years with Paget's disease 1.
Diagnosis and Treatment
Paget's disease of bone is a chronic disorder characterized by abnormal bone remodeling, resulting in enlarged, weakened bones that may cause pain, deformities, and complications. The disease often involves the pelvis, skull, spine, and long bones.
- Treatment is recommended for patients with symptoms, those at risk for complications, or those with active disease in high-risk sites.
- First-line therapy consists of bisphosphonates, with zoledronic acid (5mg IV once) being the most effective option, providing remission for up to 6-7 years.
- Alternative treatments include risedronate (30mg daily for 2 months) or alendronate (40mg daily for 6 months).
Disease Characteristics and Complications
- The disease results from excessive osteoclast activity followed by disorganized bone formation, often triggered by viral infections in genetically predisposed individuals.
- Regular monitoring with alkaline phosphatase levels helps assess disease activity and treatment response.
- Complications can include fractures, hearing loss, and rarely, osteosarcoma, making early diagnosis and appropriate management essential, as osteosarcoma may develop as a secondary malignancy related to Paget disease of the bone, particularly in adults over 65 years 1.
From the Research
Overview of Paget's Disease
- Paget's disease of bone is characterized by focal increased bone remodeling activity, resulting in sclerotic or lytic lesions and poor bone quality at one or more sites 2.
- Patients may be symptomatic with bone pain, or may be asymptomatic, and identified through unexplained elevations in serum alkaline phosphatase 2.
Treatment Options
- Bisphosphonates are the mainstay of the treatment of Paget's disease of bone (PDB) 3, 2.
- A single 5-mg infusion of zoledronic acid is the current standard therapy for Paget's disease, resulting in extended suppression of bone turnover and amelioration of bone pain 4, 2, 5.
- Oral bisphosphonates such as alendronate and risedronate can also be effective in achieving remission, with once-weekly doses used for treating osteoporosis being sufficient for a large proportion of patients 3.
- Treatment with a potent bisphosphonate is indicated in patients with Paget's disease who have symptoms likely to respond to reduced bone turnover at pagetic sites, as well as in asymptomatic patients with active disease at sites susceptible to local progression and late complications 6.
Efficacy and Safety
- Zoledronic acid has been shown to maintain biochemical remission for at least 2 years, with a rapid and complete control of the increased bone turnover of Paget's disease 4, 5.
- Risedronate and alendronate have also been shown to be effective in reducing bone turnover and achieving remission, although the duration of remission may be shorter compared to zoledronic acid 3, 5.
- Common side effects of bisphosphonate therapy include esophageal irritation, acute phase reaction with fever and flu-like symptoms, and hypocalcemia, which can be managed with calcium and vitamin D repletion 6.