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http://dspace.mediu.edu.my:8181/xmlui/handle/123456789/4572| Title: | Extended interval between enzyme therapy infusions for adult patients with Gaucher′s disease type 1. |
| Issue Date: | 30-May-2013 |
| Publisher: | Medknow Publications |
| 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. |
| URI: | http://koha.mediu.edu.my:8181/jspui/handle/123456789/4572 |
| Other Identifiers: | http://www.jpgmonline.com/article.asp?issn=0022-3859;year=2003;volume=49;issue=2;spage=127;epage=31;aulast=Pérez-Calvo http://www.doaj.org/doaj?func=openurl&genre=article&issn=00223859&date=2003&volume=49&issue=2&spage=127 |
| Appears in Collections: | Health Sciences |
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