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

Ken Ishiyama; Keijiro Sato; Tatsuya Imi; Kohei Hosokawa; Yukio Kondo; Naomi Sugimori; Hirohito Yamazaki; Shinji Nakao

doi:10.1002/jha2.51

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

EJHaem. 2020 Jun 29;1(1):243-248. doi: 10.1002/jha2.51. eCollection 2020 Jul.

Authors

Ken Ishiyama¹, Keijiro Sato^{1

2}, Tatsuya Imi¹, Kohei Hosokawa¹, Yukio Kondo¹, Naomi Sugimori¹, Hirohito Yamazaki¹, Shinji Nakao¹

Affiliations

¹ Department of Hematology Kanazawa University Hospital Ishikawa Japan.
² Department of Hematology Nagano Red Cross Hospital Nagano Japan.

PMID: 35847715
PMCID: PMC9175921
DOI: 10.1002/jha2.51

Abstract

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.

Keywords: PNH; aplastic anemia; thrombopoietin.