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Extended interval between enzyme therapy infusions for adult patients with Gaucher′s disease type 1.

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dc.creator Pérez-Calvo J
dc.creator Giraldo P
dc.creator Pastores G
dc.creator Fernández-Galán M
dc.creator Martín-Nuñez G
dc.creator Pocoví M
dc.date 2003
dc.date.accessioned 2013-05-30T11:24:14Z
dc.date.available 2013-05-30T11:24:14Z
dc.date.issued 2013-05-30
dc.identifier http://www.jpgmonline.com/article.asp?issn=0022-3859;year=2003;volume=49;issue=2;spage=127;epage=31;aulast=Pérez-Calvo
dc.identifier http://www.doaj.org/doaj?func=openurl&genre=article&issn=00223859&date=2003&volume=49&issue=2&spage=127
dc.identifier.uri http://koha.mediu.edu.my:8181/jspui/handle/123456789/4572
dc.description BACKGROUND: Enzyme replacement therapy (ERT) for Gaucher′s disease with alglucerase or imiglucerase is efficacious, well-tolerated and safe. However, cost considerations, visits to medical facilities, potentially duration of theray for life, are issues of major concern to a proportion of treated patients and has, in some cases, led to the withdrawal of therapy. AIMS: To elucidate whether an extension of the interval between enzyme infusions to once every three weeks is as effective in maintaining the clinical responses achieved with the bi-monthly regimen. MATERIALS AND METHODS: Four patients with an optimal response to ERT (at 30 units/kg every two weeks for an average of 27 months), were subjected to enzyme dose/frequency changes that essentially constituted a reduction in cumulative dose over the treatment period. Patients were assessed every 6 months for alterations in haematological parameters, plasma chitotriosidase levels, liver and spleen size, and bone symptoms. RESULTS: All patients had to resume the previous infusion schedule of once every two weeks; one because of new bone marrow infiltrates, two because of visceral enlargement, and the fourth due to progressive anaemia. CONCLUSIONS: This limited experience suggests that a reduction in enzyme dose associated with an extended interval between infusions may lead to variable disease control, and underscores the need for individualization of enzyme therapy.
dc.publisher Medknow Publications
dc.source Journal of Postgraduate Medicine
dc.title Extended interval between enzyme therapy infusions for adult patients with Gaucher′s disease type 1.

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