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Journal of Postgraduate Medicine
Medknow Publications and Staff Society of Seth GS Medical College and KEM Hospital, Mumbai, India
ISSN: 0022-3859
EISSN: 0022-3859
Vol. 49, No. 2, 2003, pp. 127-131
Bioline Code: jp03034
Full paper language: English
Document type: Research Article
Document available free of charge

Journal of Postgraduate Medicine, Vol. 49, No. 2, 2003, pp. 127-131

 en Brief Report - Extended Interval between Enzyme Therapy Infusions for Adult Patients with Gaucher's Disease Type 1
Pérez-Calvo J, Giraldo P, Pastores GM, Fernández-Galán M, Martín-Nuñez G, Pocoví M

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 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. (J Postgrad Med 2003;49:127-131)

Keywords
Enzyme replacement therapy, Gaucher's disease, alglucerase, imiglucerase

 
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