Treatment Overview
For many people, a diagnosis of Paget’s disease of bone comes as a surprise, often discovered during X-rays or blood tests for other issues. For others, it explains years of deep, aching bone pain, unexplainable warmth over a specific joint, or subtle changes in hearing. Living with this chronic condition can be worrying, as it involves the body’s recycling process of old bone into new bone going out of sync. This can lead to bones that are larger but weaker than normal, causing concerns about mobility and fracture risks.
Treatment is highly effective and focuses on regulating this bone remodeling process. The primary goals are to relieve bone pain and prevent the progression of the disease to avoid complications like osteoarthritis, fractures, or hearing loss. Because the disease can affect just one bone or several, and symptoms vary from mild to severe, treatment plans are tailored to the individual. Not every patient needs immediate treatment; those without symptoms may simply require monitoring, while those with active pain or critical bone involvement require medication (National Institute of Arthritis and Musculoskeletal and Skin Diseases, 2023).
Overview of treatment options for Paget’s Disease of Bone
The management of Paget’s disease relies almost entirely on pharmacological intervention. The central objective is to suppress the overactive breakdown of bone. By slowing this process down, the body can lay down new bone in a more organized, stronger pattern.
Medical therapy is typically recommended for anyone experiencing bone pain or for patients with involvement in critical areas such as the skull, spine, or weight-bearing bones even if they are asymptomatic. This preventative approach helps avoid nerve compression or joint damage. While surgery is sometimes necessary to repair fractures or replace damaged joints, medications are the primary tool used to treat the disease itself.
Medications used for Paget’s Disease of Bone
Bisphosphonates are the gold standard and first-line treatment for Paget’s disease. These are the same medications often used for osteoporosis, but they are used differently for Paget’s. They can be administered orally or intravenously.
The most common and effective option is often a single intravenous infusion of zoledronic acid. Clinical experience suggests that one dose can control the disease and provide remission for several years in many patients.
Oral bisphosphonates, such as alendronate or risedronate, are alternatives for patients who prefer pills. These are typically taken daily for a course of two to six months. While effective, they require strict adherence to dosing instructions to ensure absorption and protect the esophagus.
For patients who cannot tolerate bisphosphonates due to kidney issues or severe side effects, calcitonin is a second-line option. This is a synthetic hormone administered via injection. While it helps reduce pain and bone turnover, it is generally less potent than bisphosphonates and is used less frequently today.
Analgesics, including non-steroidal anti-inflammatory drugs (NSAIDs) like ibuprofen or acetaminophen, are often used alongside these specific treatments to help manage day-to-day joint or bone pain (Mayo Clinic, 2022).
How these medications work
Paget’s disease is caused by overactive osteoclasts (bone-breaking cells) that break down bone too quickly. In response, osteoblasts (bone-building cells) rush to replace it with disorganized, weak tissue.
Bisphosphonates treat this by binding to bone and stopping osteoclast function. This allows osteoblasts to build stronger, healthier bone at a normal pace. Calcitonin works similarly to inhibit osteoclasts, but its effect is less lasting (American College of Rheumatology, 2023).
Side effects and safety considerations
Intravenous bisphosphonates (like zoledronic acid) can cause a temporary flu-like reaction (fever, muscle aches, fatigue), usually minimized by good hydration. Oral bisphosphonates may cause GI irritation (heartburn/ulcers); they must be taken with water on an empty stomach, and the patient must remain upright for 30 minutes.
A rare, serious side effect of high-dose bisphosphonate therapy is osteonecrosis of the jaw (ONJ), often linked to invasive dental work; dental procedures should ideally be completed before starting treatment. Calcium and Vitamin D levels are monitored due to the risk of hypocalcemia. Doses may need adjustment or alternative therapies for patients with severe kidney disease.
Since everyone’s experience with the condition and its treatments can vary, working closely with a qualified healthcare provider helps ensure safe and effective care.
References
- American College of Rheumatology. https://www.rheumatology.org
- Mayo Clinic. https://www.mayoclinic.org
- National Institute of Arthritis and Musculoskeletal and Skin Diseases. https://www.niams.nih.gov
- MedlinePlus. https://medlineplus.gov
Medications for Paget's Disease of Bone
These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Paget's Disease of Bone.