LAUSR

Aplastic anaemia in childhood is a rare, potentially life-threatening disease, characterised by peripheral blood pancytopenia, hypocellular bone marrow without dysplasia or fibrosis. It is usually acquired, caused by immune-mediated destruction of the haematopoietic progenitor cells. Timely haematopoietic stem-cell transplantation (HSCT) from a fully human leucocyte antigen (HLA)-matched sibling donor (MSD) is considered the mainstay of treatment for children with acquired severe aplastic anaemia (SAA). For children not eligible for MSD-HSCT, immunosuppressive therapy (IST) with horse anti-thymocyte globulin (hATG) plus cyclosporine (CsA) is the treatment of choice, with an initial overall response rate of 70–80%. Haematological improvement is expected within the first 3 months after ATG, although delayed response can also be observed. Of those patients treated with IST, 20–30% do not respond, while 10–30% of responders relapse. The non-responders receive either a second course of IST with suboptimal results or proceed with HSCT from a matched unrelated donor (MUD). Upfront alternative donor transplantations and/or use of other agents have been suggested to improve overall and eventfree survival. Eltrombopag, an oral thrombopoietin receptor agonist, has been shown to increase bone marrow cellularity, number of CD34 and progenitor cells, through a direct effect on marrow stem cells. In adult patients with SAA, the addition of eltrombopag to IST contributes to rapid and robust recovery of blood counts and restoration of trilineage haematopoiesis, even after drug discontinuation. Eltrombopag has been recently approved for use as first-line treatment for adult patients with SAA in combination with standard IST. Reports on the use of eltrombopag in paediatric SAA are scarce and conflicting. In the present study, we present a