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. 2025 Jul;104(7):3863-3867.
doi: 10.1007/s00277-025-06448-1. Epub 2025 Jun 12.

High-dose romiplostim for donor-type aplasia

Kohei Hosokawa  1 , Tatsuya Imi  2 , Takafumi Yokota  3   4 , Yuma Tada  3 , Hiroyuki Maruyama  2 , Naomi Sugimori  2 , Ken Ishiyama  2 , Hirohito Yamazaki  5 , Toshihiro Miyamoto  2 , Shinji Nakao  6   7
Affiliations

High-dose romiplostim for donor-type aplasia

Kohei Hosokawa et al. Ann Hematol. 2025 Jul.

Abstract

Donor-type aplasia (DTA) is a severe complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT), characterized by bone marrow hypoplasia despite full chimerism. This report highlights the successful use of romiplostim (ROMI) for treating DTA in three patients with acquired aplastic anemia (AA), including two who were unresponsive to eltrombopag (EPAG). Case 1: A 21-year-old female with non-severe AA, treated with cyclosporine (CsA), rabbit antithymocyte globulin, and EPAG, showed no improvement. After undergoing bone marrow transplantation (BMT) from an HLA-matched sibling, she continued to experience pancytopenia. Switching from EPAG to ROMI 16 months after BMT led to transfusion independence after 7 weeks and normalized blood counts by 17 months. Case 2: A 35-year-old male with moderate AA, initially treated with CsA and ROMI, switched to EPAG before BMT from an HLA-matched sibling. Despite complete donor chimerism, pancytopenia recurred 7 months after BMT. Transitioning from EPAG to ROMI resulted in transfusion independence after 5 months and normalized blood counts after 11 months of ROMI. Case 3: A 25-year-old female with moderate AA, unresponsive to CsA and EPAG, remained transfusion-dependent. Following BMT from an HLA-matched sibling and an initial lack of response to ROMI, restarting ROMI led to transfusion independence after 9 months and normalization of blood counts by day 418 post-BMT. These patients suggest ROMI, particularly at high doses, may be more effective than EPAG for DTA, potentially avoiding the need for a second allo-HSCT. Further studies are required to compare the efficacy of ROMI and EPAG in treating DTA following allo-HSCT.

Keywords: Allo-BMT; Donor-type aplasia; Romiplostim.

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Conflict of interest statement

Declarations. Ethical approval: The study protocol was approved by the institutional review board of Kanazawa University Hospital. Informed consent: Informed consent was obtained from the patient for publication of this report. Competing interests: Prof. Nakao and Yamazaki reports honoraria from Kyowa-Kirin and Novartis. The other authors declare no conflicts of interest in association with the present study.

Figures

Fig. 1
Fig. 1
Successful treatment of donor-type aplasia with romiplostim in three acquired aplastic anemia patients, including two patients who did not respond to EPAG. A The clinical course of Case (1) EPAG was switched to ROMI (10 µg/kg/week) 16 months after BMT. Her pancytopenia started to improve after 7 weeks of ROMI therapy, and she became transfusion independent 3 months later. B The clinical course of Case (2) EPAG was changed to ROMI starting at 10 µg/kg/week and was kept at the dose of 20 µg/kg for 5 months. Five months after the initiation of ROMI therapy, he achieved transfusion independence, and his blood cell counts normalized 11 months after the therapy. C The clinical course of Case (3) We diagnosed the patient as having DTA, and ROMI (10 µg/kg/week) was restarted on day 43, and increased up to 20 µg/kg/week. Seven months after BMT, her reticulocyte counts increased from 21.0 × 109/L to 40.0 × 109/L, and she achieved transfusion independence 9 months after BMT
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