Efficacy and safety of eltrombopag in the first-line therapy of severe aplastic anemia in children.
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| Abstract |
Eltrombopag is being investigated for the treatment of aplastic anemia (AA) by stimulating hematopoietic stem cell (HSC) proliferation. To evaluate the efficacy and safety of eltrombopag in the first-line therapy of pediatric AA. The present retrospective study assessed pediatric patients with newly diagnosed AA administered immunosuppressive therapy (IST) (rabbit ATG combined with CSA) with eltrombopag at a single center from March to September 2017. All patients were followed up for >2 years. A total of 14 patients (8 males), averagely aged 86 months, were enrolled in this study. Eltrombopag was administered with a median time to initiation of 19.5 days after IST; the median course of treatment was 253 days. Complete and overall response rates at 6 months were 64.3% (9/14 case) and 78.6% (11/14 cases), respectively. The survival rate was 100%, and no relapse occurred in responders. Eltrombopag was well-tolerated; however, the most common adverse events included indirect bilirubin elevation, jaundice, and transient liver-enzyme elevation. By the end of follow-up, bone marrow chromosomes were normal, and no abnormal myelodysplastic syndrome (MDS)-related clones appeared. Addition of eltrombopag to IST is associated with markedly increased complete response with respect to hematology in pediatric patients with SAA compared with a historical cohort, without intolerable side effects.
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| Authors | Ma Jie, Lingling Fu, Sidan Li, Yixuan He, Jiafeng Yao, Xiaoling Cheng, Liqiang Zhang, Jie Zheng, Rui Zhang, Runhui Wu |
| Journal | Pediatric hematology and oncology (Pediatr Hematol Oncol) Vol. 38 Issue 7 Pg. 647-657 (Oct 2021) ISSN: 1521-0669 [Electronic] England |
| PMID | 33798022 (Publication Type: Journal Article) |
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