Background Aplastic anaemia comes from a bone marrow failure which affects all blood lineages. Treatment is based on immunosuppression or allogeneic transplant, but some patients are refractory to these options. Eltrombopag, a thrombopoietin receptor agonist administered orally, promotes megakaryopoiesis and platelet release.

Purpose To assess eltrombopag effectiveness as off-label treatment for immunosuppression-refractory aplastic anaemia.

Material and methods A 10-month (1/12/2013–30/09/2014) retrospective study was carried out including immunosuppression-refractory aplastic anaemia patients who weren’t allotransplantation candidates. After approval by the Regional Committee for drug use under Special Circumstances, each patient received different eltrombopag doses. In order to assess the effect, data were gathered from platelet count at baseline and subsequently, considering the response positive when the platelet count increased enough to avoid transfusion. Clinical data were obtained from the patient’s history and from Outpatient records.

Results We recovered data from two patients (male, aged 26 and 34) diagnosed with aplastic anaemia refractory to anti-thymocyte globulin, ciclosporin and high-dose steroids. Neither of them were candidates for allotransplantation. Initial dose was 50 mg/day, causing a platelet count increase of 7 and 272 mill/mm³ after three weeks. Then, doses were modified according to current response to an average of 6.25 mg/day, reaching counts of 5–125 mill/mm³ (average 44.33 mill/mm³) in one patient and 1–553 mill/m³ (average 139.76 mill/mm³) in the other. The average treatment length was 6.75 months. Those results are similar, even better, to the ones shown in a phase-II study (Olnes et al, 2012) involving 25 patients with the same diagnosis and dose (response in 44%, average increase 44.0 mill/mm³, nine of them transfusion independent).

Conclusion Both patients reached platelet counts higher than 100 mill/mm³, avoiding transfusions and their associated risks, with an improvement in their quality of life. Because of the unavailability of other treatments for this kind of patients, the off-label use of eltrombopag is a promising alternative when there’s little chance of cure.

References and/or acknowledgements No conflict of interest.