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Current evidence and the emerging role of eltrombopag in severe aplastic anemia
Acquired aplastic anemia (AA) is characterized by a reduced stem cell reserve. Several preclinical studies have confirmed the beneficial effect of thrombopoietin (TPO) on the expansion and maintenance of hematopoietic stem cells (HSCs). Thus, TPO receptor agonists seem to be an ideal therapeutic age...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
SAGE Publications
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7940771/ https://www.ncbi.nlm.nih.gov/pubmed/33747425 http://dx.doi.org/10.1177/2040620721998126 |
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author | Drexler, Beatrice Passweg, Jakob |
author_facet | Drexler, Beatrice Passweg, Jakob |
author_sort | Drexler, Beatrice |
collection | PubMed |
description | Acquired aplastic anemia (AA) is characterized by a reduced stem cell reserve. Several preclinical studies have confirmed the beneficial effect of thrombopoietin (TPO) on the expansion and maintenance of hematopoietic stem cells (HSCs). Thus, TPO receptor agonists seem to be an ideal therapeutic agent for AA to augment marrow function. First studies with eltrombopag as a single agent at 150 mg/day showed an overall response rate of 40–50% in patients with refractory severe AA (rSAA). Subsequent studies examined the first-line use of eltrombopag together with horse antithymocyte globulin and cyclosporine, reaching response rates up to 94%. Although used at high doses, known adverse events in the form of skin, gastrointestinal, or hepatic impairment are feasible in AA, however first data show a relatively high rate of clonal evolution in the form of karyotypic aberrations in patients with rAA. Nonetheless, there is a strong rationale that eltrombopag can contribute to restoring hematopoiesis in SAA by stimulating HSCs. Further studies are needed to decide if eltrombopag is clearly superior to current established treatments and to determine optimal treatment duration, dosage, and long-term effects. |
format | Online Article Text |
id | pubmed-7940771 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | SAGE Publications |
record_format | MEDLINE/PubMed |
spelling | pubmed-79407712021-03-18 Current evidence and the emerging role of eltrombopag in severe aplastic anemia Drexler, Beatrice Passweg, Jakob Ther Adv Hematol Review Acquired aplastic anemia (AA) is characterized by a reduced stem cell reserve. Several preclinical studies have confirmed the beneficial effect of thrombopoietin (TPO) on the expansion and maintenance of hematopoietic stem cells (HSCs). Thus, TPO receptor agonists seem to be an ideal therapeutic agent for AA to augment marrow function. First studies with eltrombopag as a single agent at 150 mg/day showed an overall response rate of 40–50% in patients with refractory severe AA (rSAA). Subsequent studies examined the first-line use of eltrombopag together with horse antithymocyte globulin and cyclosporine, reaching response rates up to 94%. Although used at high doses, known adverse events in the form of skin, gastrointestinal, or hepatic impairment are feasible in AA, however first data show a relatively high rate of clonal evolution in the form of karyotypic aberrations in patients with rAA. Nonetheless, there is a strong rationale that eltrombopag can contribute to restoring hematopoiesis in SAA by stimulating HSCs. Further studies are needed to decide if eltrombopag is clearly superior to current established treatments and to determine optimal treatment duration, dosage, and long-term effects. SAGE Publications 2021-03-03 /pmc/articles/PMC7940771/ /pubmed/33747425 http://dx.doi.org/10.1177/2040620721998126 Text en © The Author(s), 2021 https://creativecommons.org/licenses/by-nc/4.0/ This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (https://creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access page (https://us.sagepub.com/en-us/nam/open-access-at-sage). |
spellingShingle | Review Drexler, Beatrice Passweg, Jakob Current evidence and the emerging role of eltrombopag in severe aplastic anemia |
title | Current evidence and the emerging role of eltrombopag in severe aplastic anemia |
title_full | Current evidence and the emerging role of eltrombopag in severe aplastic anemia |
title_fullStr | Current evidence and the emerging role of eltrombopag in severe aplastic anemia |
title_full_unstemmed | Current evidence and the emerging role of eltrombopag in severe aplastic anemia |
title_short | Current evidence and the emerging role of eltrombopag in severe aplastic anemia |
title_sort | current evidence and the emerging role of eltrombopag in severe aplastic anemia |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7940771/ https://www.ncbi.nlm.nih.gov/pubmed/33747425 http://dx.doi.org/10.1177/2040620721998126 |
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