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Stem cell biology

High-dose romiplostim accelerates hematologic recovery in patients with aplastic anemia refractory to eltrombopag

Leukemia volume 35, pages 906–909 (2021)Cite this article

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Patients with acquired aplastic anemia (AA) refractory to eltrombopag (EPAG) have been reported to respond poorly to another thrombopoietin receptor agonist (TPO-RA), romiplostim (ROMI), in doses of up to 10 μg/kg per week. We analyzed the effectiveness of 20 μg/kg ROMI in 21 patients with EPAG-refractory AA. Sixteen of 21 (76%) achieved a hematologic response in at least one lineage at 3 months. Five of ten (50%) patients became independent of platelet (n = 2) or red cell (n = 3) transfusions. All 21 patients tolerated ROMI and showed no severe treatment-related adverse events that necessitated ROMI discontinuation. This retrospective study is the first to demonstrate high-dose ROMI was highly effective in AA patients refractory to EPAG.

AA is caused by immune-mediated destruction of hematopoietic stem and progenitor cells (HSPCs), resulting in bone marrow hypoplasia and pancytopenia in the peripheral blood [1]. Immunosuppressive therapy (IST) with horse anti-thymoglobulin (ATG) and cyclosporine (CsA) is the standard of care for patients with severe AA (SAA) [2]. However, ~30% of patients fail to respond, and therapeutic options for refractory cases are limited.

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Fig. 1: Longitudinal hematologic improvements and lineage characteristics in refractory AA patients who received romiplostim.

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Acknowledgements

The authors thank Rie Ohmi (Kanazawa University) for her excellent technical assistance. We thank all patients and medical staff of participating institutions who contributed to this study. We are grateful to the following doctors for providing us with clinical information: K. Aoshima of Houju Memorial Hospital; Y. Kaneko of Saitama Cancer Center; H. Shibayama and T. Yokota of Osaka University Hospital; K. Shirahata of Iida Municipal Hospital; T. Ono of Hamamatsu University Hospital; M. Okura of University of Fukui Hospital; M. Ogata of Oita University Hospital; T. Endo of Hokkaido University Hospital; T. Kitano of Kitano Hospital; K. Miyazaki of Kitasato University Hospital; Y. Kageyama of Kuwana City Medical Center; T. Yamaguchi of Mie University Hospital; and M. Takeuchi of Kohka Public Hospital. This study was supported by a MEXT Grant-in-Aid for Scientific Research (B, 19H03686 to SN, C, 18K08318 to HY and Young Scientists, 19K17823 to KH).

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  1. These authors contributed equally: Kohei Hosokawa, Hirohito Yamazaki

Authors and Affiliations

  1. Department of Hematology, Faculty of Medicine, Institute of Medical Pharmaceutical and Health Sciences, Kanazawa University, Kanazawa, Japan

    Kohei Hosokawa, Mikoto Tanabe, Tatsuya Imi, Naomi Sugimori & Shinji Nakao

  2. Division of Transfusion Medicine, Kanazawa University Hospital, Kanazawa, Japan

    Hirohito Yamazaki

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  1. Kohei Hosokawa
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Contributions

KH, HY, and SN designed the research, analyzed the data, and wrote the manuscript. KH, HY, MT, TI, NS, and SN recruited patients to participate in the study. All authors critically reviewed the manuscript and approved the submission of the final manuscript. KH and HY equally contributed to this paper as first authors.

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Correspondence to Shinji Nakao.

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Conflict of interest

HY received research funding from Otsuka Pharmaceutical and Alexion Pharma, and honoraria from Novartis Pharma, Kyowa Kirin, Sanofi, Nippon Shinyaku, Chugai Pharmaceutical and Fuji Pharma. SN received honoraria fees and lecture fees from Novartis Pharma K.K. and Kyowa-Kirin K.K. KH, MT, TI, and NS declare that they have no conflicts of interest to report.

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Hosokawa, K., Yamazaki, H., Tanabe, M. et al. High-dose romiplostim accelerates hematologic recovery in patients with aplastic anemia refractory to eltrombopag. Leukemia 35, 906–909 (2021). https://doi.org/10.1038/s41375-020-0950-6

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