Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease

Abstract 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...

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Autores principales: Juan Wang, Min Dai, Qiong Fu, Sheng Chen
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Publicado: Nature Portfolio 2021
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spelling oai:doaj.org-article:efe7af08d0f3455089981a6d8628972c2021-12-02T11:37:27ZEltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease10.1038/s41598-021-84493-22045-2322https://doaj.org/article/efe7af08d0f3455089981a6d8628972c2021-03-01T00:00:00Zhttps://doi.org/10.1038/s41598-021-84493-2https://doaj.org/toc/2045-2322Abstract 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 × 109/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.Juan WangMin DaiQiong FuSheng ChenNature PortfolioarticleMedicineRScienceQENScientific Reports, Vol 11, Iss 1, Pp 1-8 (2021)
institution DOAJ
collection DOAJ
language EN
topic Medicine
R
Science
Q
spellingShingle Medicine
R
Science
Q
Juan Wang
Min Dai
Qiong Fu
Sheng Chen
Eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease
description Abstract 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 × 109/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 article
author Juan Wang
Min Dai
Qiong Fu
Sheng Chen
author_facet Juan Wang
Min Dai
Qiong Fu
Sheng Chen
author_sort Juan Wang
title 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_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_sort eltrombopag for the treatment of refractory thrombocytopenia associated with connective tissue disease
publisher Nature Portfolio
publishDate 2021
url https://doaj.org/article/efe7af08d0f3455089981a6d8628972c
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