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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...

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Autores principales: Drexler, Beatrice, Passweg, Jakob
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2021
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.
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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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