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Efficacy and safety of eltrombopag in adult refractory immune thrombocytopenia
BACKGROUND: Eltrombopag is a thrombopoietin receptor agonist with excellent treatment outcomes in immune thrombocytopenia (ITP). Here, we analyzed the dose of eltrombopag required to achieve and maintain safe platelet counts in Korean ITP patients. METHODS: Adult refractory ITP patients (<30,000...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Korean Society of Hematology; Korean Society of Blood and Marrow Transplantation; Korean Society of Pediatric Hematology-Oncology; Korean Society on Thrombosis and Hemostasis
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4377333/ https://www.ncbi.nlm.nih.gov/pubmed/25830126 http://dx.doi.org/10.5045/br.2015.50.1.19 |
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author | Kim, Yeo-Kyeoung Lee, Seung-Sin Jeong, Sung-Hoon Ahn, Jae-Sook Yang, Deok-Hwan Lee, Je-Jung Kim, Hyeoung-Joon |
author_facet | Kim, Yeo-Kyeoung Lee, Seung-Sin Jeong, Sung-Hoon Ahn, Jae-Sook Yang, Deok-Hwan Lee, Je-Jung Kim, Hyeoung-Joon |
author_sort | Kim, Yeo-Kyeoung |
collection | PubMed |
description | BACKGROUND: Eltrombopag is a thrombopoietin receptor agonist with excellent treatment outcomes in immune thrombocytopenia (ITP). Here, we analyzed the dose of eltrombopag required to achieve and maintain safe platelet counts in Korean ITP patients. METHODS: Adult refractory ITP patients (<30,000 platelets/µL) were enrolled. Eltrombopag doses were increased to achieve a target platelet count (≥50,000 cells/µL). After achieving the target platelet count, the dose of concomitant ITP medications and eltrombopag was reduced to identify the lowest effective dose required to maintain the platelet count. RESULTS: Among 18 patients, 66.7% achieved complete response, 5.6% achieved platelet counts between 50,000 and 100,000 cells/µL, and 27.8% failed to achieve the target platelet count. The median ITP duration was significantly shorter in patients who achieved the target platelet count. The initial dose required to achieve the target platelet count was 25 mg/d. The adjusted maintenance doses were 25 mg twice per week or 25 mg/d. After discontinuation, 83.3% relapsed, and the median relapse-free survival was 15 days. Two relapsed and 1 failed patient switched to romiplostim. The response to romiplostim was similar to eltrombopag. During eltrombopag treatment, 38.9% showed hepatobiliary laboratory anomalies. Among 9 follow-up bone marrow examinations, 1 revealed fibrosis after 1 year of treatment. CONCLUSION: Eltrombopag was well tolerated with excellent treatment outcomes in refractory adult ITP patients. Low-dose eltrombopag effectively maintained the target platelet count. However, some patients required longer or higher-dose treatment to maintain the target platelet count, especially in heavily pretreated or longer ITP cases. |
format | Online Article Text |
id | pubmed-4377333 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2015 |
publisher | Korean Society of Hematology; Korean Society of Blood and Marrow Transplantation; Korean Society of Pediatric Hematology-Oncology; Korean Society on Thrombosis and Hemostasis |
record_format | MEDLINE/PubMed |
spelling | pubmed-43773332015-03-31 Efficacy and safety of eltrombopag in adult refractory immune thrombocytopenia Kim, Yeo-Kyeoung Lee, Seung-Sin Jeong, Sung-Hoon Ahn, Jae-Sook Yang, Deok-Hwan Lee, Je-Jung Kim, Hyeoung-Joon Blood Res Original Article BACKGROUND: Eltrombopag is a thrombopoietin receptor agonist with excellent treatment outcomes in immune thrombocytopenia (ITP). Here, we analyzed the dose of eltrombopag required to achieve and maintain safe platelet counts in Korean ITP patients. METHODS: Adult refractory ITP patients (<30,000 platelets/µL) were enrolled. Eltrombopag doses were increased to achieve a target platelet count (≥50,000 cells/µL). After achieving the target platelet count, the dose of concomitant ITP medications and eltrombopag was reduced to identify the lowest effective dose required to maintain the platelet count. RESULTS: Among 18 patients, 66.7% achieved complete response, 5.6% achieved platelet counts between 50,000 and 100,000 cells/µL, and 27.8% failed to achieve the target platelet count. The median ITP duration was significantly shorter in patients who achieved the target platelet count. The initial dose required to achieve the target platelet count was 25 mg/d. The adjusted maintenance doses were 25 mg twice per week or 25 mg/d. After discontinuation, 83.3% relapsed, and the median relapse-free survival was 15 days. Two relapsed and 1 failed patient switched to romiplostim. The response to romiplostim was similar to eltrombopag. During eltrombopag treatment, 38.9% showed hepatobiliary laboratory anomalies. Among 9 follow-up bone marrow examinations, 1 revealed fibrosis after 1 year of treatment. CONCLUSION: Eltrombopag was well tolerated with excellent treatment outcomes in refractory adult ITP patients. Low-dose eltrombopag effectively maintained the target platelet count. However, some patients required longer or higher-dose treatment to maintain the target platelet count, especially in heavily pretreated or longer ITP cases. Korean Society of Hematology; Korean Society of Blood and Marrow Transplantation; Korean Society of Pediatric Hematology-Oncology; Korean Society on Thrombosis and Hemostasis 2015-03 2015-03-24 /pmc/articles/PMC4377333/ /pubmed/25830126 http://dx.doi.org/10.5045/br.2015.50.1.19 Text en © 2015 Korean Society of Hematology http://creativecommons.org/licenses/by-nc/3.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/3.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Original Article Kim, Yeo-Kyeoung Lee, Seung-Sin Jeong, Sung-Hoon Ahn, Jae-Sook Yang, Deok-Hwan Lee, Je-Jung Kim, Hyeoung-Joon Efficacy and safety of eltrombopag in adult refractory immune thrombocytopenia |
title | Efficacy and safety of eltrombopag in adult refractory immune thrombocytopenia |
title_full | Efficacy and safety of eltrombopag in adult refractory immune thrombocytopenia |
title_fullStr | Efficacy and safety of eltrombopag in adult refractory immune thrombocytopenia |
title_full_unstemmed | Efficacy and safety of eltrombopag in adult refractory immune thrombocytopenia |
title_short | Efficacy and safety of eltrombopag in adult refractory immune thrombocytopenia |
title_sort | efficacy and safety of eltrombopag in adult refractory immune thrombocytopenia |
topic | Original Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4377333/ https://www.ncbi.nlm.nih.gov/pubmed/25830126 http://dx.doi.org/10.5045/br.2015.50.1.19 |
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