A new set of treatment guidelines for Paget’s disease was published in a recent issue of the Journal of Bone and Mineral Research by the Guideline Development Group (GDG), produced by a multidisciplinary panel representing the UK Paget’s Association, the European Calcified Tissues Society, and the International Osteoporosis Foundation.
Paget’s disease is a nonmalignant skeletal disorder that causes enlargement of the bones leading to possible hearing loss, basilar invagination of the skull, obstructive hydrocephalus, stenosis of the spinal canal and paraplegia. The underlying mechanisms are believed to involve abnormalities in bone remodeling due increased osteoclast and osteoblast activity at a cellular level. Patients with Paget’s disease may have genetic susceptibilities that can be triggered by environmental factors, although this has not been established.
The new guidelines offer recommendations in multiple areas related to the management of Paget’s disease, including:
• Bisphosphonates as the recommended treatment for bone pain associated with Paget’s disease.
• At the same time, the committee concluded there was insufficient evidence to recommend bisphosphonates for other features related to Paget’s disease, including improvement in quality of life, prevention of fractures and bone deformity, prevention of progression of osteoarthritis and hearing loss, and reduction of blood loss prior to elective orthopedic surgery.
• Although bisphosphonates were shown to significantly reduce biochemical markers of bone remodeling (and especially high levels of alkaline phosphatase), there was no clear clinical benefit determined in suppression of bone turnover.
• Symptomatic treatment was recommended over a treat-to-target strategy.
• It was recommended that complications such as spinal dysfunction may be treated with a trial of bisphosphonate or calcitonin, with spinal surgery considered for treatment of spinal stenosis or cord compression.
• While surgery was recommended for fixation of fractures, no one type of surgery was deemed superior to another.
• Joint replacement surgery and osteotomy were recommended for treatment-resistant osteoarthritis.
The guidelines also pointed to currently unresolved questions in Paget’s disease management, including treatment of asymptomatic patients, the use of genetic profiling in diagnosis and treatment, the benefits to joint replacement surgery, osteotomy, and fracture fixation, and the potential for less invasive nonpharmacologic therapies than surgery.
Ralston SH, Corral-Gudino L, Cooper C, et al. Diagnosis and Management of Paget's Disease of Bone in Adults: A Clinical Guideline. J Bone Miner Res 2019 Feb 25:e3657. doi: 10.1002/jbmr.3657. [Epub ahead of print]