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Aplastic anemia in childhood is a rare, life-threating disorder, characterized by peripheral blood pancytopenia and a hypocellular bone marrow without signs of dysplasia or fibrosis. Acquired aplastic anemia needs to be distinguished from inherited bone marrow failure syndromes or myelodysplastic syndromes.

The aim of this study is to assess the clinical and laboratory findings at the time of diagnosis, the treatment approach and the outcome of children with aplastic anemia treated in our department during the past decade.

We identified 9 children with aplastic anemia. The patients’ population included 6 (66.7%) males and the mean age at admission was 9.7 years. At the time of diagnosis, the average neutrophil count was 750/mm³, the Hb count was 8.4mg/dl and platelets count was 8770/mm³. In all of our cases aplastic anemia was acquired, expect one case of Fanconi anemia. Predisposing risk factors (including drugs exposure, viral infections, chemicals) were identified in 4 patients. Among the 9 studied patients, 3 (33.3%) had moderate-non severe, 2 (22.2%) had severe and 4 (44.5%) had very severe aplastic anemia. All of the patients received immunosuppressive therapy (consisting of antithymocyte globulin, cyslosporine A and steroids), 2 remained transfusion independent, 4 underwent bone marrow transplantation- 2 from a matched related donor and 2 from a matched unrelated donor. One patient with refractory disease received, as an alternative first line therapy, eltrombopag. Complete response was achieved in 22.2%, partial response was achieved in 22.2%, relapse occurred in 11.1% and 44.5% of the patients had refractory disease. The overall survival was 77.8%.

A remarkable progress has been made during the past decades in the understanding of pathogenesis and management of children with aplastic anemia. Bone marrow transplantation from a matched related donor is the recommended first line therapy resulting in an excellent survival rate that exceeds 90%. In the future the development of targeted strategies for patients and the enhancement of supportive care will further improve outcome and diminish the disease’s late complications.