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Blood, 1 August 2006, Vol. 108, No. 3, pp. 830-835. Prepublished online as a Blood First Edition Paper on March 9, 2006; DOI 10.1182/blood-2005-12-5072. This Article Full Text Full Text (PDF) All Versions of this Article: 2005-12-5072v1 108/3/830    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by de Fost, M. Articles by Dahl, S. v. Search for Related Content PubMed PubMed Citation Articles by de Fost, M. Articles by Dahl, S. v. Related Collections Phagocytes Clinical Trials and Observations Related Letter in Blood Online Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  CLINICAL TRIALS AND OBSERVATIONS Superior effects of high-dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels: a 2-center retrospective analysis Maaike de Fost, Carla E. M. Hollak, Johanna E. M. Groener, Johannes M. F. G. Aerts, Mario Maas, Ludger W. Poll, Maaike G. Wiersma, Dieter Häussinger, Sarah Brett, Nicole Brill, and Stephan vom Dahl From the Department of Internal Medicine, Endocrinology and Metabolism; Department of Biochemistry; and Department of Radiology, Academic Medical Center, Amsterdam, The Netherlands; Department of Gastroenterology; Hepatology and Infectious Diseases; and Department of Radiology, Heinrich-Heine University, Duesseldorf, Germany. Dosing of enzyme replacement therapy (ERT) for Gaucher disease type 1 is still a subject of debate and varies from 15 to 130 U/kg/mo, making a huge economic difference of US $70 000 to US $380 000 (55 000-300 000) per patient per year. To investigate whether this difference in dosing ultimately translates into a different response, we retrospectively compared long-term outcome of ERT at 2 large European treatment centers, Academic Medical Center, Amsterdam, The Netherlands (n = 49, median dose, 15-30 U/kg/4 wks) and Heinrich-Heine University, Duesseldorf, Germany (n = 57, median dose, 80 U/kg/4 wks). These adult cohorts had a similar genetic background. All follow-up parameters were matched separately at baseline, to avoid bias with respect to disease severity. Improvement in hemoglobin, platelet count, and hepatosplenomegaly was not significantly different between both cohorts, whereas plasma chitotriosidase and bone marrow involvement by magnetic resonance imaging improved more quickly and was more pronounced in the higher-dosed group. Major bone complications rarely occurred in both groups. In conclusion, different dosing regimens of ERT do not affect outcome of hematologic and visceral parameters, but higher dosing leads to accelerated decrease of chitotriosidase and better objective bone response in adult type 1 Gaucher disease. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? Related Letter in Blood Online: Low-dose versus high-dose therapy for Gaucher disease: goals and markers Carla E. M. Hollak, Maaike de Fost, Johannes M. F. G. Aerts, and Stephan vom Dahl Blood 2007 109: 387. [Full Text] [PDF] This article has been cited by other articles: M. de Fost, C. J.M. van Noesel, J. M.F.G. Aerts, M. Maas, R. G. Poll, and C. E.M. Hollak Persistent bone disease in adult type 1 Gaucher disease despite increasing doses of enzyme replacement therapy Haematologica, July 1, 2008; 93(7): 1119 - 1120. [Full Text] [PDF] P. L. Robertson, M. Maas, and J. Goldblatt Semiquantitative Assessment of Skeletal Response to Enzyme Replacement Therapy for Gaucher's Disease Using the Bone Marrow Burden Score Am. J. Roentgenol., June 1, 2007; 188(6): 1521 - 1528. [Abstract] [Full Text] [PDF] J. Schmitz, L. W. Poll, and S. v. Dahl Therapy of adult Gaucher disease Haematologica, February 1, 2007; 92(2): 148 - 152. [Full Text] [PDF] C. E. M. Hollak, M. d. Fost, J. M. F. G. Aerts, and S. vom Dahl Low-dose versus high-dose therapy for Gaucher disease: goals and markers Blood, January 1, 2007; 109(1): 387 - 387. [Full Text] [PDF] A. Zimran, D. Elstein, and E. Beutler Low-dose therapy trumps high-dose therapy again in the treatment of Gaucher disease Blood, August 1, 2006; 108(3): 802 - 803. [Full Text] [PDF]

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