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Eltrombopag is an effective and safe therapy for refractory thrombocytopenia after haploidentical hematopoietic stem cell transplantation

Bone Marrow Transplantation volume 54, pages 1310–1318 (2019)Cite this article

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Abstract

Refractory thrombocytopenia is a frequent and severe complication after haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) and lacks effective treatment strategies. Eltrombopag has shown promising results in several thrombocytopenia disorders. We report 38 patients treated with eltrombopag for refractory thrombocytopenia after haplo-HSCT. Eight patients had delayed platelet engraftment, 15 patients had secondary failure of platelet recovery, and 15 patients had poor graft function (PGF). Eltrombopag was initiated at 25 or 50 mg daily, and the dosage was adjusted to a maximum of 50–100 mg daily to maintain platelet between 50 × 109/L and 100 × 109/L. The cumulative incidence of platelet recovery to transfusion independence was 63.2% and to ≥50 × 109/L without transfusion support was 52.3%. Furthermore, neutrophil counts and hemoglobin were also increased in the nine responders with PGF. Nineteen (79.2%) of the 24 responders were able to taper off eltrombopag, and the remaining 5 patients were able to begin a taper. The median duration of treatment was 64 (range 14–195) days. The presence of megakaryocyte before initiation was the only independent factor influencing the efficacy of eltrombopag (P = 0.044). Eltrombopag was well tolerated in all patients. In summary, eltrombopag was a safe and effective therapy for refractory thrombocytopenia after haplo-HSCT.

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Acknowledgements

The authors thank the participating investigators and patients.

Funding

This work was supported by National Natural Science Foundation of China (No. 81670167).

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Authors and Affiliations

  1. Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, P.R. China

    Haixia Fu, Xiaohui Zhang, Tingting Han, Xiaodong Mo, Yu Wang, Huan Chen, Wei Han, Jingzhi Wang, Fengrong Wang, Chenhua Yan, Yuanyuan Zhang, Yuqian Sun, Kaiyan Liu, Xiaojun Huang & Lanping Xu

  2. Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation for the Treatment of Hematological Diseases, Beijing, P.R. China

    Haixia Fu, Xiaohui Zhang, Tingting Han, Xiaodong Mo, Yu Wang, Huan Chen, Wei Han, Jingzhi Wang, Fengrong Wang, Chenhua Yan, Yuanyuan Zhang, Yuqian Sun, Kaiyan Liu, Xiaojun Huang & Lanping Xu

  3. Collaborative Innovation Centre of Hematology, Peking University, Beijing, P.R. China

    Xiaojun Huang

  4. Peking-Tsinghua Centre for Life Sciences, Academy for Advanced Interdisciplinary Studies, Peking University, Beijing, P.R. China

    Xiaojun Huang

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  1. Haixia Fu
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Correspondence to Lanping Xu.

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Fu, H., Zhang, X., Han, T. et al. Eltrombopag is an effective and safe therapy for refractory thrombocytopenia after haploidentical hematopoietic stem cell transplantation. Bone Marrow Transplant 54, 1310–1318 (2019). https://doi.org/10.1038/s41409-019-0435-2

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