Evidence-based recommendations for monitoring bone disease and the response to enzyme replacement therapy in Gaucher patients

Publisher Website
Google Scholar
Abstract
Background: Bone disease is a serious complication of Gaucher disease. Untreated, it can result in pain, permanent bone damage and disability. Enzyme replacement therapy reverses many of the clinical signs of Gaucher bone disease but early assessment and treatment, and regular monitoring, are essential in optimising outcomes.Scope: In September 2005, a group of European experts met to review current knowledge and identify best practice and unmet needs in the monitoring of Gaucher bone disease and the response to enzyme replacement therapy.Methods: Medline searches of peer-reviewed literature (no date restrictions) were conducted and supplemented by additional information considered relevant by panellists to furthering discussions.Findings and conclusions: The group's recommendations included: currently used biochemical bone markers are not clinically practical or reliable; plain X‐rays should not be the sole method of assessing bone disease; MRI is the most sensitive method for monitoring bone mar...