Extended interval between enzyme therapy infusions for adult patients with Gaucher's disease type 1.
J Postgrad Med
;
2003 Apr-Jun; 49(2): 127-31
Article
in English
| IMSEAR
| ID: sea-115934
ABSTRACT
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 ANDMETHODS:
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.
Full text:
Available
Index:
IMSEAR (South-East Asia)
Main subject:
Time Factors
/
Infusions, Intravenous
/
Female
/
Humans
/
Male
/
Drug Administration Schedule
/
Child
/
Adolescent
/
Adult
/
Dose-Response Relationship, Drug
Language:
English
Journal:
J Postgrad Med
Year:
2003
Type:
Article
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