Efficacy and safety of avatrombopag in the treatment of thrombocytopenia after umbilical cord blood transplantation
Abstract. Background:. Delayed platelet engraftment is a common complication after umbilical cord blood transplantation (UCBT), and there is no standard therapy. Avatrombopag (AVA) is a second-generation thrombopoietin (TPO) receptor agonist (TPO-RA) that has shown efficacy in immune thrombocytopen...
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Wolters Kluwer
2025-05-01
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| Series: | Chinese Medical Journal |
| Online Access: | http://journals.lww.com/10.1097/CM9.0000000000003216 |
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| author | Aijie Huang Guangyu Sun Baolin Tang Yongsheng Han Xiang Wan Wen Yao Kaidi Song Yaxin Cheng Weiwei Wu Meijuan Tu Yue Wu Tianzhong Pan Xiaoyu Zhu Sihan Zhou Xiuyuan Hao |
| author_facet | Aijie Huang Guangyu Sun Baolin Tang Yongsheng Han Xiang Wan Wen Yao Kaidi Song Yaxin Cheng Weiwei Wu Meijuan Tu Yue Wu Tianzhong Pan Xiaoyu Zhu Sihan Zhou Xiuyuan Hao |
| author_sort | Aijie Huang |
| collection | DOAJ |
| description | Abstract.
Background:. Delayed platelet engraftment is a common complication after umbilical cord blood transplantation (UCBT), and there is no standard therapy. Avatrombopag (AVA) is a second-generation thrombopoietin (TPO) receptor agonist (TPO-RA) that has shown efficacy in immune thrombocytopenia (ITP). However, few reports have focused on its efficacy in patients diagnosed with thrombocytopenia after allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Methods:. We conducted a retrospective study at the First Affiliated Hospital of the University of Science and Technology of China to evaluate the efficacy of AVA as a first-line TPO-RA in 65 patients after UCBT; these patients were compared with 118 historical controls. Response rates, platelet counts, megakaryocyte counts in bone marrow, bleeding events, adverse events and survival rates were evaluated in this study. Platelet reconstitution differences were compared between different medication groups. Multivariable analysis was used to explore the independent beneficial factors for platelet implantation.
Results:. Fifty-two patients were given AVA within 30 days post-UCBT, and the treatment was continued for more than 7 days to promote platelet engraftment (AVA group); the other 13 patients were given AVA for secondary failure of platelet recovery (SFPR group). The median time to platelet engraftment was shorter in the AVA group than in the historical control group (32.5 days vs. 38.0 days, Z = 2.095, P = 0.036). Among the 52 patients in the AVA group, 46 achieved an overall response (OR) (88.5%), and the cumulative incidence of OR was 91.9%. Patients treated with AVA only had a greater 60-day cumulative incidence of platelet engraftment than patients treated with recombinant human thrombopoietin (rhTPO) only or rhTPO combined with AVA (95.2% vs. 84.5% vs. 80.6%, P <0.001). Patients suffering from SFPR had a slightly better cumulative incidence of OR (100%, P = 0.104). Patients who initiated AVA treatment within 14 days post-UCBT had a better 60-day cumulative incidence of platelet engraftment than did those who received AVA after 14 days post-UCBT (96.6% vs. 73.9%, P = 0.003).
Conclusion:. Compared with those in the historical control group, our results indicate that AVA could effectively promote platelet engraftment and recovery after UCBT, especially when used in the early period (≤14 days post-UCBT). |
| format | Article |
| id | doaj-art-e7fd23784b394217a30659806cb7b747 |
| institution | OA Journals |
| issn | 0366-6999 2542-5641 |
| language | English |
| publishDate | 2025-05-01 |
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| spelling | doaj-art-e7fd23784b394217a30659806cb7b7472025-08-20T02:15:33ZengWolters KluwerChinese Medical Journal0366-69992542-56412025-05-0113891072108310.1097/CM9.0000000000003216202505050-00008Efficacy and safety of avatrombopag in the treatment of thrombocytopenia after umbilical cord blood transplantationAijie Huang0Guangyu Sun1Baolin Tang2Yongsheng Han3Xiang Wan4Wen Yao5Kaidi Song6Yaxin Cheng7Weiwei Wu8Meijuan Tu9Yue Wu10Tianzhong Pan11Xiaoyu Zhu12Sihan ZhouXiuyuan Hao1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China4 School of Public Health, Shanxi Medical University, Taiyuan, Shanxi 030001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, China1 Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui 230001, ChinaAbstract. Background:. Delayed platelet engraftment is a common complication after umbilical cord blood transplantation (UCBT), and there is no standard therapy. Avatrombopag (AVA) is a second-generation thrombopoietin (TPO) receptor agonist (TPO-RA) that has shown efficacy in immune thrombocytopenia (ITP). However, few reports have focused on its efficacy in patients diagnosed with thrombocytopenia after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Methods:. We conducted a retrospective study at the First Affiliated Hospital of the University of Science and Technology of China to evaluate the efficacy of AVA as a first-line TPO-RA in 65 patients after UCBT; these patients were compared with 118 historical controls. Response rates, platelet counts, megakaryocyte counts in bone marrow, bleeding events, adverse events and survival rates were evaluated in this study. Platelet reconstitution differences were compared between different medication groups. Multivariable analysis was used to explore the independent beneficial factors for platelet implantation. Results:. Fifty-two patients were given AVA within 30 days post-UCBT, and the treatment was continued for more than 7 days to promote platelet engraftment (AVA group); the other 13 patients were given AVA for secondary failure of platelet recovery (SFPR group). The median time to platelet engraftment was shorter in the AVA group than in the historical control group (32.5 days vs. 38.0 days, Z = 2.095, P = 0.036). Among the 52 patients in the AVA group, 46 achieved an overall response (OR) (88.5%), and the cumulative incidence of OR was 91.9%. Patients treated with AVA only had a greater 60-day cumulative incidence of platelet engraftment than patients treated with recombinant human thrombopoietin (rhTPO) only or rhTPO combined with AVA (95.2% vs. 84.5% vs. 80.6%, P <0.001). Patients suffering from SFPR had a slightly better cumulative incidence of OR (100%, P = 0.104). Patients who initiated AVA treatment within 14 days post-UCBT had a better 60-day cumulative incidence of platelet engraftment than did those who received AVA after 14 days post-UCBT (96.6% vs. 73.9%, P = 0.003). Conclusion:. Compared with those in the historical control group, our results indicate that AVA could effectively promote platelet engraftment and recovery after UCBT, especially when used in the early period (≤14 days post-UCBT).http://journals.lww.com/10.1097/CM9.0000000000003216 |
| spellingShingle | Aijie Huang Guangyu Sun Baolin Tang Yongsheng Han Xiang Wan Wen Yao Kaidi Song Yaxin Cheng Weiwei Wu Meijuan Tu Yue Wu Tianzhong Pan Xiaoyu Zhu Sihan Zhou Xiuyuan Hao Efficacy and safety of avatrombopag in the treatment of thrombocytopenia after umbilical cord blood transplantation Chinese Medical Journal |
| title | Efficacy and safety of avatrombopag in the treatment of thrombocytopenia after umbilical cord blood transplantation |
| title_full | Efficacy and safety of avatrombopag in the treatment of thrombocytopenia after umbilical cord blood transplantation |
| title_fullStr | Efficacy and safety of avatrombopag in the treatment of thrombocytopenia after umbilical cord blood transplantation |
| title_full_unstemmed | Efficacy and safety of avatrombopag in the treatment of thrombocytopenia after umbilical cord blood transplantation |
| title_short | Efficacy and safety of avatrombopag in the treatment of thrombocytopenia after umbilical cord blood transplantation |
| title_sort | efficacy and safety of avatrombopag in the treatment of thrombocytopenia after umbilical cord blood transplantation |
| url | http://journals.lww.com/10.1097/CM9.0000000000003216 |
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