کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
2827888 1162469 2011 4 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply
موضوعات مرتبط
علوم زیستی و بیوفناوری بیوشیمی، ژنتیک و زیست شناسی مولکولی زیست شناسی مولکولی
پیش نمایش صفحه اول مقاله
Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply
چکیده انگلیسی

Because of an unpredicted and unavoidable shortage in global supplies of imiglucerase for Gaucher disease, we collected clinical and laboratory data from patients who were evaluated ≤ 6 months before drug withdrawal and then retested before/during reinstatement of therapy. Hemoglobin, platelet counts, and liver and spleen index volumes by ultrasound as well as chitotriosidase were evaluated as percent change over time and relative to dose-years of exposure to enzyme therapy. Deterioration was seen in all four clinical parameters and in chitotriosidase activity in most patients and even among patients who stopped for only 3 months. No patient consistently showed nondeterioration in all five parameters. Platelet counts were most sensitive to therapy withdrawal. There was no overt correlation between percent change in these parameters and dose-years of therapy. Although we no longer think that drug vacations should be considered as a form of maintenance for patients requiring enzyme therapy for Gaucher disease, but if necessary—for compelling personal reasons—one may be reassured that a short-term drug interruption is not likely to lead to irreversible complications or return to baseline values of critical clinical parameters.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Blood Cells, Molecules, and Diseases - Volume 46, Issue 1, 15 January 2011, Pages 111–114
نویسندگان
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