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Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease : A Single-Center Experience
Joint Authors
Elstein, Deborah
Zimran, Ari
Tukan, Irina
Abrahamov, Ayala
Altarescu, Gheona
Hadas-Halpern, Irith
Source
Issue
Vol. 2013, Issue 2013 (31 Dec. 2013), pp.1-6, 6 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2013-10-28
Country of Publication
Egypt
No. of Pages
6
Main Subjects
Abstract EN
Gaucher disease, a lysosomal storage disorder, is a multisystem disorder with variable and unpredictable onset and severity.
Disease-specific enzyme replacement therapy (ERT) has been shown to reverse or ameliorate disease-specific hepatosplenomegaly and anemia and thrombocytopenia.
ERT also impacts bone manifestations, including bone crises, bone pain, and appearance of new osteonecrosis, and improves bone mineral density to varying degrees.
The objective of this study was to assess achievement of predefined therapeutic goals based on international registry outcomes for Israeli patients with Gaucher disease receiving imiglucerase for four consecutive years on a low-dose regimen followed in a single center.
All data were taken from patient files.
The therapeutic goals were taken from standards published in the literature for disease-specific clinical parameters.
Among 164 patients at baseline, values for spleen and liver volumes, hemoglobin and platelet counts, and Z-scores for lumbar spine and femoral were significantly different from the goal.
After four years ERT, there was a significant improvement (P=0.000) in each of the therapeutic goal parameters from baseline.
15.2% of these patients achieved all hematology-visceral goals.
In children, there was achievement of linear growth and puberty.
This survey highlights the good overall response in symptomatic patients receiving low-dose ERT with imiglucerase in Israel.
American Psychological Association (APA)
Tukan, Irina& Hadas-Halpern, Irith& Altarescu, Gheona& Abrahamov, Ayala& Elstein, Deborah& Zimran, Ari. 2013. Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease : A Single-Center Experience. Advances in Hematology،Vol. 2013, no. 2013, pp.1-6.
https://search.emarefa.net/detail/BIM-449887
Modern Language Association (MLA)
Tukan, Irina…[et al.]. Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease : A Single-Center Experience. Advances in Hematology No. 2013 (2013), pp.1-6.
https://search.emarefa.net/detail/BIM-449887
American Medical Association (AMA)
Tukan, Irina& Hadas-Halpern, Irith& Altarescu, Gheona& Abrahamov, Ayala& Elstein, Deborah& Zimran, Ari. Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease : A Single-Center Experience. Advances in Hematology. 2013. Vol. 2013, no. 2013, pp.1-6.
https://search.emarefa.net/detail/BIM-449887
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-449887