Paroxysmal nocturnal hemoglobinuria‐phenotype cells predict a good response to eltrombopag in patients with refractory aplastic anemia

To identify factors affecting responsiveness to eltrombopag (EPAG), we retrospectively analyzed 38 aplastic anemia patients treated with EPAG who were refractory (n = 29) or showed an inadequate response (n = 9) to conventional therapies. The efficacy was evaluated at 16 weeks after starting EPAG an...

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Autores principales: Ishiyama, Ken, Sato, Keijiro, Imi, Tatsuya, Hosokawa, Kohei, Kondo, Yukio, Sugimori, Naomi, Yamazaki, Hirohito, Nakao, Shinji
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Short Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9175921/
https://www.ncbi.nlm.nih.gov/pubmed/35847715
http://dx.doi.org/10.1002/jha2.51
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author Ishiyama, Ken
Sato, Keijiro
Imi, Tatsuya
Hosokawa, Kohei
Kondo, Yukio
Sugimori, Naomi
Yamazaki, Hirohito
Nakao, Shinji
author_facet Ishiyama, Ken
Sato, Keijiro
Imi, Tatsuya
Hosokawa, Kohei
Kondo, Yukio
Sugimori, Naomi
Yamazaki, Hirohito
Nakao, Shinji
author_sort Ishiyama, Ken
collection PubMed
description To identify factors affecting responsiveness to eltrombopag (EPAG), we retrospectively analyzed 38 aplastic anemia patients treated with EPAG who were refractory (n = 29) or showed an inadequate response (n = 9) to conventional therapies. The efficacy was evaluated at 16 weeks after starting EPAG and at any given time when the best response was achieved. Hematologic responses were observed in 15 patients (39%) at week 16 and in 25 (66%) at any given time. Ten of 19 (53%) achieved transfusion independence. A univariate analysis revealed the presence of PNH‐phenotype cells and the relatively higher platelet counts as associated with a good response to EPAG.
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spelling pubmed-91759212022-07-14 Paroxysmal nocturnal hemoglobinuria‐phenotype cells predict a good response to eltrombopag in patients with refractory aplastic anemia Ishiyama, Ken Sato, Keijiro Imi, Tatsuya Hosokawa, Kohei Kondo, Yukio Sugimori, Naomi Yamazaki, Hirohito Nakao, Shinji EJHaem Short Reports To identify factors affecting responsiveness to eltrombopag (EPAG), we retrospectively analyzed 38 aplastic anemia patients treated with EPAG who were refractory (n = 29) or showed an inadequate response (n = 9) to conventional therapies. The efficacy was evaluated at 16 weeks after starting EPAG and at any given time when the best response was achieved. Hematologic responses were observed in 15 patients (39%) at week 16 and in 25 (66%) at any given time. Ten of 19 (53%) achieved transfusion independence. A univariate analysis revealed the presence of PNH‐phenotype cells and the relatively higher platelet counts as associated with a good response to EPAG. John Wiley and Sons Inc. 2020-06-29 /pmc/articles/PMC9175921/ /pubmed/35847715 http://dx.doi.org/10.1002/jha2.51 Text en © 2020 The Authors. eJHaem published by British Society for Haematology and John Wiley & Sons Ltd. https://creativecommons.org/licenses/by/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
spellingShingle Short Reports
Ishiyama, Ken
Sato, Keijiro
Imi, Tatsuya
Hosokawa, Kohei
Kondo, Yukio
Sugimori, Naomi
Yamazaki, Hirohito
Nakao, Shinji
Paroxysmal nocturnal hemoglobinuria‐phenotype cells predict a good response to eltrombopag in patients with refractory aplastic anemia
title Paroxysmal nocturnal hemoglobinuria‐phenotype cells predict a good response to eltrombopag in patients with refractory aplastic anemia
title_full Paroxysmal nocturnal hemoglobinuria‐phenotype cells predict a good response to eltrombopag in patients with refractory aplastic anemia
title_fullStr Paroxysmal nocturnal hemoglobinuria‐phenotype cells predict a good response to eltrombopag in patients with refractory aplastic anemia
title_full_unstemmed Paroxysmal nocturnal hemoglobinuria‐phenotype cells predict a good response to eltrombopag in patients with refractory aplastic anemia
title_short Paroxysmal nocturnal hemoglobinuria‐phenotype cells predict a good response to eltrombopag in patients with refractory aplastic anemia
title_sort paroxysmal nocturnal hemoglobinuria‐phenotype cells predict a good response to eltrombopag in patients with refractory aplastic anemia
topic Short Reports
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9175921/
https://www.ncbi.nlm.nih.gov/pubmed/35847715
http://dx.doi.org/10.1002/jha2.51
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