Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience
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...
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| Format: | Article |
| Language: | English |
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Wiley
2013-01-01
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| Series: | Advances in Hematology |
| Online Access: | http://dx.doi.org/10.1155/2013/151506 |
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| author | Irina Tukan Irith Hadas-Halpern Gheona Altarescu Ayala Abrahamov Deborah Elstein Ari Zimran |
| author_facet | Irina Tukan Irith Hadas-Halpern Gheona Altarescu Ayala Abrahamov Deborah Elstein Ari Zimran |
| author_sort | Irina Tukan |
| collection | DOAJ |
| description | 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 () 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. |
| format | Article |
| id | doaj-art-2450c0764f4b41b8bef8eee9e66d3183 |
| institution | Kabale University |
| issn | 1687-9104 1687-9112 |
| language | English |
| publishDate | 2013-01-01 |
| publisher | Wiley |
| record_format | Article |
| series | Advances in Hematology |
| spelling | doaj-art-2450c0764f4b41b8bef8eee9e66d31832025-02-03T05:47:23ZengWileyAdvances in Hematology1687-91041687-91122013-01-01201310.1155/2013/151506151506Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center ExperienceIrina Tukan0Irith Hadas-Halpern1Gheona Altarescu2Ayala Abrahamov3Deborah Elstein4Ari Zimran5Shaare Zedek Medical Center, Affiliated to the Hadassah-Hebrew University School of Medicine, Ein Karem 91031, IsraelShaare Zedek Medical Center, Affiliated to the Hadassah-Hebrew University School of Medicine, Ein Karem 91031, IsraelGaucher Clinic and Preimplantation Genetics Unit, Shaare Zedek Medical Center, Affiliated to the Hadassah-Hebrew University School of Medicine, Ein Karem 91031, IsraelShaare Zedek Medical Center, Affiliated to the Hadassah-Hebrew University School of Medicine, Ein Karem 91031, IsraelShaare Zedek Medical Center, Affiliated to the Hadassah-Hebrew University School of Medicine, Ein Karem 91031, IsraelShaare Zedek Medical Center, Affiliated to the Hadassah-Hebrew University School of Medicine, Ein Karem 91031, IsraelGaucher 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 () 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.http://dx.doi.org/10.1155/2013/151506 |
| spellingShingle | Irina Tukan Irith Hadas-Halpern Gheona Altarescu Ayala Abrahamov Deborah Elstein Ari Zimran Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience Advances in Hematology |
| title | Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience |
| title_full | Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience |
| title_fullStr | Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience |
| title_full_unstemmed | Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience |
| title_short | Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience |
| title_sort | achievement of therapeutic goals with low dose imiglucerase in gaucher disease a single center experience |
| url | http://dx.doi.org/10.1155/2013/151506 |
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