Effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists: a single-center retrospective study
Background Romiplostim (ROM), a second-generation thrombopoietin receptor agonist (TPO-RA), has shown promising results in patients with refractory aplastic anemia (AA); however, its optimal dosage, efficacy, and safety in patients with refractory AA who experienced treatment failure with immunosupp...
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Taylor & Francis Group
2025-12-01
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| Series: | Annals of Medicine |
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| Online Access: | https://www.tandfonline.com/doi/10.1080/07853890.2025.2514791 |
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| author | Xijuan Lin Chen Yang Ziwei Liu Junling Zhuang Miao Chen Bing Han |
| author_facet | Xijuan Lin Chen Yang Ziwei Liu Junling Zhuang Miao Chen Bing Han |
| author_sort | Xijuan Lin |
| collection | DOAJ |
| description | Background Romiplostim (ROM), a second-generation thrombopoietin receptor agonist (TPO-RA), has shown promising results in patients with refractory aplastic anemia (AA); however, its optimal dosage, efficacy, and safety in patients with refractory AA who experienced treatment failure with immunosuppressive therapy (IST) and other types of TPO-RAs remain unclear. In the present study, we aimed to retrospectively assess the efficacy and safety of high-dose ROM in these patients.Patients and methods Patients who received ROM consecutively for at least 3 months between 8 April 2023 and 23 October 2023, and were followed up for at least 6 months after therapy were analyzed. All enrolled patients had failed to respond and stopped the previous treatments for at least 3 months before receiving ROM.Results Eleven patients were followed up for at least 6 months; all experienced treatment failure with IST and at least two types of other TPO-RAs. They had a median age of 54 years (range: 27–77 years), and eight (72.7%) were women. The patients’ initial and maximal ROM dose was 20 μg/kg per week. At a median follow-up of 8 months (range, 6–8 months), 72.7% (8/11) of the patients reached the response criteria at a median of 1 month (range: 1–3 months). Of these patients, 54.5% (6/11) met the criteria for a complete response at a median of 2.5 months (range: 1–3 months), and 27.3% (3/11) achieved a trilineage response. No severe ROM-related adverse events that led to treatment discontinuation or dosage reduction were observed. Notably, 12.5% (1/8) of the responders relapsed at 5 months after stopping ROM for 2 months.Conclusion High-dose ROM with an initial dosage of 20 μg/kg per week is effective and safe for patients with refractory AA who experienced treatment failure with IST and multiple oral TPO-RAs, exerting a rapid response. |
| format | Article |
| id | doaj-art-332d42da092c4248ae51addfebc5b65a |
| institution | OA Journals |
| issn | 0785-3890 1365-2060 |
| language | English |
| publishDate | 2025-12-01 |
| publisher | Taylor & Francis Group |
| record_format | Article |
| series | Annals of Medicine |
| spelling | doaj-art-332d42da092c4248ae51addfebc5b65a2025-08-20T02:37:05ZengTaylor & Francis GroupAnnals of Medicine0785-38901365-20602025-12-0157110.1080/07853890.2025.2514791Effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists: a single-center retrospective studyXijuan Lin0Chen Yang1Ziwei Liu2Junling Zhuang3Miao Chen4Bing Han5Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, ChinaDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, ChinaDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, ChinaDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, ChinaDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, ChinaDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, ChinaBackground Romiplostim (ROM), a second-generation thrombopoietin receptor agonist (TPO-RA), has shown promising results in patients with refractory aplastic anemia (AA); however, its optimal dosage, efficacy, and safety in patients with refractory AA who experienced treatment failure with immunosuppressive therapy (IST) and other types of TPO-RAs remain unclear. In the present study, we aimed to retrospectively assess the efficacy and safety of high-dose ROM in these patients.Patients and methods Patients who received ROM consecutively for at least 3 months between 8 April 2023 and 23 October 2023, and were followed up for at least 6 months after therapy were analyzed. All enrolled patients had failed to respond and stopped the previous treatments for at least 3 months before receiving ROM.Results Eleven patients were followed up for at least 6 months; all experienced treatment failure with IST and at least two types of other TPO-RAs. They had a median age of 54 years (range: 27–77 years), and eight (72.7%) were women. The patients’ initial and maximal ROM dose was 20 μg/kg per week. At a median follow-up of 8 months (range, 6–8 months), 72.7% (8/11) of the patients reached the response criteria at a median of 1 month (range: 1–3 months). Of these patients, 54.5% (6/11) met the criteria for a complete response at a median of 2.5 months (range: 1–3 months), and 27.3% (3/11) achieved a trilineage response. No severe ROM-related adverse events that led to treatment discontinuation or dosage reduction were observed. Notably, 12.5% (1/8) of the responders relapsed at 5 months after stopping ROM for 2 months.Conclusion High-dose ROM with an initial dosage of 20 μg/kg per week is effective and safe for patients with refractory AA who experienced treatment failure with IST and multiple oral TPO-RAs, exerting a rapid response.https://www.tandfonline.com/doi/10.1080/07853890.2025.2514791Aplastic anemiarefractoryromiplostimefficacysafety |
| spellingShingle | Xijuan Lin Chen Yang Ziwei Liu Junling Zhuang Miao Chen Bing Han Effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists: a single-center retrospective study Annals of Medicine Aplastic anemia refractory romiplostim efficacy safety |
| title | Effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists: a single-center retrospective study |
| title_full | Effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists: a single-center retrospective study |
| title_fullStr | Effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists: a single-center retrospective study |
| title_full_unstemmed | Effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists: a single-center retrospective study |
| title_short | Effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists: a single-center retrospective study |
| title_sort | effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists a single center retrospective study |
| topic | Aplastic anemia refractory romiplostim efficacy safety |
| url | https://www.tandfonline.com/doi/10.1080/07853890.2025.2514791 |
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