https://scholars.lib.ntu.edu.tw/handle/123456789/195651
Title: | Management of Non-Neuronopathic Gaucher Disease with Special Reference to Pregnancy, Splenectomy, Bisphosphonate Therapy, Use of Biomarkers and Bone Disease Monitoring | Authors: | Cox, T. M. Aerts, J. M. F. G. Belmatoug, N. Cappellini, M. D. Dahl, S. vom Goldblatt, J. Grabowski, G. A. Hollak, C. E. M. WUH-LIANG HWU Maas, M. Martins, A. M. Mistry, P. K. Pastores, G. M. Tylki-Szymanska, A. Yee, J. Weinreb N. |
Issue Date: | 2008 | Start page/Pages: | 319-336 | Source: | Journal of Inherited Metabolic Disease | Abstract: | Enzyme replacement was introduced as treatment for non- neuronopathic Gaucher disease more than 15 years ago. To ensure the best use of this costly ultra-orphan agent, a systematic disease management approach has been proposed by an international panel; this includes the development, by consensus, of achievable treatment goals. Here we critically review these goals and monitoring guidelines and incorporate emerging experience of the disease in the therapeutic era, as well as contemporary clinical research. This review makes recommendations related specifically to the management of pregnancy; the appropriate use of splenectomy and bisphosphonate treatment; the relevance of biochemical markers to disease monitoring; and the use of semi-quantitative methods for assessing bone marrow infiltration. In addition, we identify key areas for development, including the requirement for a validated index of disease severity; the need to correlate widely used biomarkers with long-term disease outcomes, and the desirability of establishing agreed standards for monitoring of bone disease particularly in infants and children with Gaucher disease. |
URI: | http://ntur.lib.ntu.edu.tw//handle/246246/186831 | DOI: | 10.1007/s10545-008-0779-z | SDG/Keyword: | alendronic acid; alglucerase; biological marker; bisphosphonic acid derivative; calcitonin; calcitriol; calcium; chitotriosidase; denosumab; desmopressin acetate; estrogen; ibandronic acid; imiglucerase; methoxy isobutyl isonitrile technetium tc 99m; miglustat; pamidronic acid; parathyroid hormone[1-34]; selective estrogen receptor modulator; strontium ranelate; zoledronic acid; adjuvant therapy; avascular necrosis; bleeding; bone disease; bone marrow transplantation; bone necrosis; bone pain; bone radiography; clinical trial; disease severity; drug efficacy; drug safety; dual energy X ray absorptiometry; enzyme replacement; femur head necrosis; Gaucher disease; human; infection sensitivity; jaw disease; low drug dose; nonhuman; nuclear magnetic resonance imaging; osteogenesis imperfecta; osteopenia; osteoporosis; portal hypertension; practice guideline; pregnancy; prognosis; quantitative analysis; review; scoring system; sensitivity and specificity; splenectomy; splenomegaly; thrombocyte transfusion; Absorptiometry, Photon; Biological Markers; Bone Diseases; Diphosphonates; Female; Gaucher Disease; Humans; Magnetic Resonance Imaging; Pregnancy; Pregnancy Complications; Splenectomy |
Appears in Collections: | 醫學系 |
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