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Acquired bone marrow aplasia is a disorder with an estimated incidence of 1--2 cases per million inhabitants per year characterised by low bone marrow cellularity that results in pancytopaenia. The first-line treatment in children is matched related donor haematopoietic stem cell transplantation (HSCT), and if this option is not available, immunosuppressive therapy; should the latter fail, the next step is matched unrelated donor HSCT. Eltrombopag, a thrombopoietin analogue, has been recently introduced as a possible treatment of refractory aplasia. There is published evidence of its effectiveness in adults, but its role in paediatric patients has yet to be evaluated. We present the case of a girl aged 11 years with severe acquired bone marrow aplasia whose test results showed trilineage peripheral blood recovery after starting treatment with eltrombopag. The patient initially presented with generalised ecchymosis and petechiae, asthenia and metrorrhagia of 15 days’ duration. The blood panel revealed severe pancytopaenia with findings suggestive of hyporegenerative anaemia (haemoglobin, 7.6 g/dL; reticulocytes, 0.69%), a platelet count of 8 × 1000/ L and a neutrophil count of 0.5 × 1000/ L, with no abnormalities in blood chemistry or findings suggestive of haemolysis or coagulopathy. There was no excess of blasts in the peripheral blood smear and serology tests were negative. The patient underwent bone marrow aspiration and biopsy, the findings of which were compatible with severe bone marrow aplasia. We ruled out paroxysmal nocturnal haemoglobinuria, and the chromosome fragility test was negative. Since the patient did not have an HLA-matched relative, she started immunosuppressive therapy with thymoglobulin and ciclosporin combined with granulocyte-colony stimulating factor (G-CSF). The initial response was good, and the patient showed a favourable partial response at day 60 of treatment. She later exhibited progressive deterioration, and by day 120 the findings of the evaluation were consistent with a nonresponder profile, with the patient requiring weekly transfusions. Due to the failure of immunosuppressive therapy, the patient was considered eligible for unrelated-donor HSCT given the lack of a related donor. After an unsuccessful search for a potential donor that lasted 6 months, given the severity of her bone marrow aplasia, the decision was made to initiate treatment with eltrombopag with a dose of 50 mg administered orally every

Eficacia y seguridad en el uso del eltrombopag en un caso de aplasia medular adquirida grave