Efficacy of Eltrombopag With Immunosuppressive Therapy for Children With Acquired Aplastic Anemia

Authors

Yufei Zhao
Wenrui Yang
Xin Zhao
Xiangrong Hu
Jing Hu
Xu Liu
Jianping Li
Lei Ye
Youzhen Xiong
Yang Yang
Baohang Zhang
Xiaoxia Li
Xiawan Yang
Yimeng Shi
Guangxin Peng
Yuan Li
Huihui Fan
Kang Zhou
Liping Jing
Li Zhang
Fengkui Zhang

Publication Date

1-10-2023

Journal

Frontiers in Pediatrics

Abstract

BACKGROUND: Eltrombopag (EPAG), an oral thrombopoietin receptor agonist (TPO-RA), has been proven to improve the hematologic response without increasing toxic effects as a first-line therapy combined with standard immunosuppressive treatment (IST) in adults with severe aplastic anemia (SAA). Nevertheless, the clinical evidence on the efficacy of EPAG in children with acquired aplastic anemia is limited and controversial.

METHODS: We performed a single-center, retrospective study to analyze the clinical outcomes of fifteen patients aged ≤18 years with newly diagnosed acquired SAA who received first-line IST and EPAG (EPAG group) compared with those of forty-five patients who received IST alone (IST group) by propensity score matching (PSM).

RESULTS: There was no difference in the overall response (OR) rate between the EPAG group and IST group (53.3% vs. 46.7% at 3 months,

CONCLUSION: Adding EPAG to standard IST as the first-line treatment for children with acquired SAA improved the rapidity of hematological response and the CR rate but did not improve the OR or EFS rates.

Keywords

aplastic anemia, bone marrow failure, eltrombopag, hematological response, pediatric, immunosuppressive therapy

Comments

Supplementary Materials

PMID: 36704148