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

Stephan Vom Dahl, Ludger Poll, Maja Di Rocco, Giovanni Ciana, Carmencita Denes, Giuliano Mariani, Mario Maas

Research output: Contribution to journalArticle

64 Citations (Scopus)

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 marrow infiltration by Gaucher cells; semi-quantitative methods for assessing bone marrow infiltration in routine clinical practice should use readily available technology, include an assessment of Gaucher cell infiltration in the lumbar spine and femur, and be validated for inter-rater reliability and in comparison to other methods; a multidisciplinary approach is required for the treatment of Gaucher patients; all Gaucher patients should receive a comprehensive initial radiologic evaluation for bone disease and ongoing radiological monitoring at least once every 2 years.

Original languageEnglish
Pages (from-to)1045-1064
Number of pages20
JournalCurrent Medical Research and Opinion
Volume22
Issue number6
DOIs
Publication statusPublished - Jun 2006

Fingerprint

Enzyme Replacement Therapy
Bone Diseases
Gaucher Disease
Bone Marrow
Bone and Bones
Biomedical Technology Assessment
Practice Guidelines
Femur
Spine
Biomarkers
X-Rays
Pain
Therapeutics

Keywords

  • Bone disease
  • Enzyme replacement therapy
  • Gaucher disease
  • Imiglucerase
  • Monitoring

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Evidence-based recommendations for monitoring bone disease and the response to enzyme replacement therapy in Gaucher patients. / Vom Dahl, Stephan; Poll, Ludger; Di Rocco, Maja; Ciana, Giovanni; Denes, Carmencita; Mariani, Giuliano; Maas, Mario.

In: Current Medical Research and Opinion, Vol. 22, No. 6, 06.2006, p. 1045-1064.

Research output: Contribution to journalArticle

Vom Dahl, Stephan ; Poll, Ludger ; Di Rocco, Maja ; Ciana, Giovanni ; Denes, Carmencita ; Mariani, Giuliano ; Maas, Mario. / Evidence-based recommendations for monitoring bone disease and the response to enzyme replacement therapy in Gaucher patients. In: Current Medical Research and Opinion. 2006 ; Vol. 22, No. 6. pp. 1045-1064.
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