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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.
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Submitted December 27, 2005
Accepted February 23, 2006
Superior effects of high dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels; a two center retrospective analysis
Maaike de Fost, Carla E Hollak*, Johanna E Groener, Johannes M Aerts, Mario Maas, Ludger Poll, Maaike G Wiersma, Dieter Haussinger, Sarah Brett, Nicole Brill, and Stephan vom Dahl
Department of Internal Medicine, Endocrinology and Metabolism, Academic Medical Center (AMC), Amsterdam, The Netherlands
Department of Biochemistry, Academic Medical Center (AMC), Amsterdam, The Netherlands
Department of Radiology, Academic Medical Center (AMC), Amsterdam, The Netherlands
Department of Radiology, Heinrich-Heine University (HHU), Duesseldorf, Germany
Department of Gastroenterology; Hepatology and Infectious Diseases, Heinrich-Heine University (HHU), Duesseldorf, Germany
* Corresponding author; email: c.e.hollak{at}amc.uva.nl.
Dosing of enzyme replacement therapy (ERT) for Gaucher disease type 1 is still a subject of debate and varies from 15-130 U/kg/month, making a huge economic difference of 55.000 up to 300.000 Euro 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 two large European treatment centers, Amsterdam (AMC, N=49, median dose 15-30 U/kg/4 weeks) and Duesseldorf (HHU, N=57, median dose 80 U/kg/4 weeks). 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 MRI improved faster and 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 hematological and visceral parameters, but higher dosing leads to accelerated decrease of chitotriosidase and better objective bone response in adult type 1 Gaucher disease.
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