Abstract

Hematopoietic stem cell transplantation (HSCT) with a matched sibling donor is the first-line therapy for pediatric aplastic anemia (AA). Whether to perform an alternative HSCT or use immunosuppressive therapy (IST) including anti-thymocyte globulin (ATG) is still unclear if suitable donor is not available. IST with concomitant eltrombopag, a thrombopoietin receptor agonist, shows a good response in adult.1 However, data of eltrombopag is limited in pediatric with low prevalence AA, showing equivocal result.

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