| Title | Eltrombopag is an effective and safe therapy for refractory thrombocytopenia after haploidentical hematopoietic stem cell transplantation |
| Authors | Fu, Haixia Zhang, Xiaohui Han, Tingting Mo, Xiaodong Wang, Yu Chen, Huan Han, Wei Wang, Jingzhi Wang, Fengrong Yan, Chenhua Zhang, Yuanyuan Sun, Yuqian Liu, Kaiyan Huang, Xiaojun Xu, Lanping |
| Affiliation | Peking Univ, Peoples Hosp, Inst Hematol, Beijing, Peoples R China Beijing Key Lab Hematopoiet Stem Cell Transplanta, Beijing, Peoples R China Peking Univ, Collaborat Innovat Ctr Hematol, Beijing, Peoples R China Peking Univ, Acad Adv Interdisciplinary Studies, Peking Tsinghua Ctr Life Sci, Beijing, Peoples R China |
| Issue Date | 2019 |
| Publisher | BONE MARROW TRANSPLANTATION |
| 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 x 10(9)/L and 100 x 10(9)/L. The cumulative incidence of platelet recovery to transfusion independence was 63.2% and to >= 50 x 10(9)/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. |
| URI | http://hdl.handle.net/20.500.11897/546217 |
| ISSN | 0268-3369 |
| DOI | 10.1038/s41409-019-0435-2 |
| Indexed | SCI(E) EI |
| Appears in Collections: | 人民医院 前沿交叉学科研究院 |
