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Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease
To assess the efficacy and safety of eltrombopag in connective tissue disease (CTD)-immune thrombocytopenia (ITP), we conducted this single-center retrospective observational study, including patients with refractory CTD-ITP who were treated with eltrombopag between January 2018 and August 2019. The...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Nature Publishing Group UK
2021
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7943759/ https://www.ncbi.nlm.nih.gov/pubmed/33750817 http://dx.doi.org/10.1038/s41598-021-84493-2 |
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author | Wang, Juan Dai, Min Fu, Qiong Chen, Sheng |
author_facet | Wang, Juan Dai, Min Fu, Qiong Chen, Sheng |
author_sort | Wang, Juan |
collection | PubMed |
description | To assess the efficacy and safety of eltrombopag in connective tissue disease (CTD)-immune thrombocytopenia (ITP), we conducted this single-center retrospective observational study, including patients with refractory CTD-ITP who were treated with eltrombopag between January 2018 and August 2019. The characteristics of patients at baseline, and the efficacy and safety of the drug were analyzed. The predictors for a response were analyzed using a univariate analysis such as Chi-square or nonparametric test and a multiple correspondence analysis (MCA) method. A total of 15 patients with refractory CTD-ITP were included in the study. Their median age at the time of inclusion was 40.6 years. The median platelet count at initiation of eltrombopag was 11.53 × 10(9)/L. The median remission time was 3.42 weeks. The complete remission (CR) and overall response rate decreased with time. The factors that associated with response to eltrombopag in patients with CTD-ITP were protopathy, WBC counts, levels of hemoglobin, and characteristics of bone marrow findings in univariate analysis. In addition, MCA indicated that a poor response to eltrombopag in patients with refractory CTD-ITP was closely associated with a protopathy with SS, medium to severe degree of anemia, leukopenia, and bone marrow aspiration showing aplastic anemia, an absence of megakaryocytes or macrophage activation syndrome (MAS). In conclusion, eltrombopag was effective and well-tolerated in patients with CTD-associated thrombocytopenia. Some factors should be considered in the use of eltrombopag, including the protopathy, blood test, and bone marrow histology. |
format | Online Article Text |
id | pubmed-7943759 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Nature Publishing Group UK |
record_format | MEDLINE/PubMed |
spelling | pubmed-79437592021-03-10 Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease Wang, Juan Dai, Min Fu, Qiong Chen, Sheng Sci Rep Article To assess the efficacy and safety of eltrombopag in connective tissue disease (CTD)-immune thrombocytopenia (ITP), we conducted this single-center retrospective observational study, including patients with refractory CTD-ITP who were treated with eltrombopag between January 2018 and August 2019. The characteristics of patients at baseline, and the efficacy and safety of the drug were analyzed. The predictors for a response were analyzed using a univariate analysis such as Chi-square or nonparametric test and a multiple correspondence analysis (MCA) method. A total of 15 patients with refractory CTD-ITP were included in the study. Their median age at the time of inclusion was 40.6 years. The median platelet count at initiation of eltrombopag was 11.53 × 10(9)/L. The median remission time was 3.42 weeks. The complete remission (CR) and overall response rate decreased with time. The factors that associated with response to eltrombopag in patients with CTD-ITP were protopathy, WBC counts, levels of hemoglobin, and characteristics of bone marrow findings in univariate analysis. In addition, MCA indicated that a poor response to eltrombopag in patients with refractory CTD-ITP was closely associated with a protopathy with SS, medium to severe degree of anemia, leukopenia, and bone marrow aspiration showing aplastic anemia, an absence of megakaryocytes or macrophage activation syndrome (MAS). In conclusion, eltrombopag was effective and well-tolerated in patients with CTD-associated thrombocytopenia. Some factors should be considered in the use of eltrombopag, including the protopathy, blood test, and bone marrow histology. Nature Publishing Group UK 2021-03-09 /pmc/articles/PMC7943759/ /pubmed/33750817 http://dx.doi.org/10.1038/s41598-021-84493-2 Text en © The Author(s) 2021 Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. |
spellingShingle | Article Wang, Juan Dai, Min Fu, Qiong Chen, Sheng Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease |
title | Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease |
title_full | Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease |
title_fullStr | Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease |
title_full_unstemmed | Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease |
title_short | Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease |
title_sort | eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease |
topic | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7943759/ https://www.ncbi.nlm.nih.gov/pubmed/33750817 http://dx.doi.org/10.1038/s41598-021-84493-2 |
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