Efficacy of eltrombopag with immunosuppressive therapy for children with acquired aplastic anemia.

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 differenc

SUBMITTER: Zhao Y 

PROVIDER: S-EPMC9872003 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature