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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Detalles Bibliográficos
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
Descripción
Sumario: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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