Successful treatment of acquired amegakaryocytic thrombocytopenia with eltrombopag and immunosuppressant

Hong Tian1 National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China;2 Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China;3 Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, ChinaView further author information

Danqing Kong1 National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China;2 Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China;3 Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, ChinaView further author information

Yun Li1 National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China;2 Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China;3 Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, ChinaView further author information

Chengyuan Gu1 National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China;2 Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China;3 Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, ChinaView further author information

Ziqiang Yu1 National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China;2 Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China;3 Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, ChinaView further author information

Zhaoyue Wang1 National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China;2 Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China;3 Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, ChinaView further author information

Depei Wu1 National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China;2 Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China;3 Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, ChinaView further author information

Jie Yin1 National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China;2 Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China;3 Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Suzhou, ChinaCorrespondence[email protected]
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Abstract

Acquired amegakaryocytic thrombocypenia (AAMT) is an extremely rare hematologic disorder and standard treatment strategy has not been established. We described herein two cases of AAMT who were fully responded to eltrombopag and immunosuppressant. Patient 1 was refractory to steroid, IVIG and recombinant human thrombopoietin (rhTPO). Patient 2 did not respond to high dosage of steroid, IVIG, rhTPO and rituximab. Moreover, his AAMT progressed to aplastic anemia in 5 months. Both patients took eltrombopag and immunosuppressant, then they achieved long-term remission without obvious side effects. Our findings suggest that this combination can be a valuable alternative in AAMT.

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Funding

This work was supported by National Natural Science Foundation of China (No.81600105, No. 81900151), Natural Science Foundation of Jiangsu Province (BK20190176, BK20180202) and Open project of NHC Key Laboratory of Thrombosis and Hemostasis (KJS2137).

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Successful treatment of acquired amegakaryocytic thrombocytopenia with eltrombopag and immunosuppressant

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Successful treatment of acquired amegakaryocytic thrombocytopenia with eltrombopag and immunosuppressant

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