Clinical and translational pharmacological aspects of the management of fibrous dysplasia of bone.
Marlous RotmanNeveen Agnes Therese HamdyNatasha M Appelman-DijkstraPublished in: British journal of clinical pharmacology (2018)
Fibrous dysplasia (FD) is a genetic, noninheritable rare bone disease caused by a postzygotic activating mutation of the α subunit of the stimulatory G-protein causing increased abnormal bone formation leading to pain, deformity and fractures. To date, no cure has been identified for FD/McCune-Albright syndrome (MAS) and treatment is symptomatic and aimed at decreasing pain and/or local bone turnover. Various drugs have been used to achieve clinical improvement in FD/MAS patients including bisphosphonates and denosumab, however further translational studies are also warranted to address unresolved pathophysiological issues and explore novel pharmacological targets for the management of FD/MAS. In this article, we review literature on the medical treatment of FD/MAS, discuss the unresolved pathophysiological issues and explore novel pharmacological targets for the management of FD/MAS.
Keyphrases
- bone mineral density
- chronic pain
- end stage renal disease
- postmenopausal women
- ejection fraction
- healthcare
- chronic kidney disease
- neuropathic pain
- newly diagnosed
- bone regeneration
- signaling pathway
- body composition
- peritoneal dialysis
- genome wide
- gene expression
- patient reported outcomes
- spinal cord
- case report
- drug induced
- smoking cessation
- giant cell