Iron deficiency anemia following long-term eltrombopag treatment for aplastic anemia: a single-institution experience

Eltrombopag (EPAG), an oral thrombopoietin receptor agonist, has shown excellent efficacy in patients with aplastic anemia (AA) alone or in combination with immunosuppressive therapy. EPAG also has the unexpected ability to chelate polyvalent cations, including iron. However, the association between long-term EPAG use and iron deficiency anemia (IDA) remains unclear. To address this, we retrospectively evaluated the incidence and characteristics of EPAG-induced IDA (E-IDA) in patients with AA at our institution. Of the 36 patients with AA receiving EPAG, six (17%) developed E-IDA without evidence of bleeding, with a median onset of 1142.5 days (range, 389-1442 days) after EPAG administration. The cumulative dose of EPAG was significantly higher in patients with E-IDA than in those without E-IDA (P = 0.04). In 4 patients, E-IDA occurred after hemoglobin levels improved above 10 g/dl; this was considered recurrent anemia. In the other 2 patients, E-IDA occurred when hemoglobin levels remained below 10 g/dl; this was considered resistant anemia. After oral iron supplementation, all patients achieved hemoglobin levels higher than their peak levels prior to the onset of E-IDA. E-IDA should be considered when patients with AA treated with EPAG longer than 1 year develop recurrent or resistant anemia.