Sustained Erythroid Response to Eltrombopag in Low-Risk Myelodysplastic Neoplasms With Severe Anemia and Thrombocytopenia: A Case Report.

Eltrombopag has been already employed for thrombocytopenia in myelodysplastic neoplasms (MDS), but its role and efficacy in improving other cytopenias are still unknown. In October 2020, a 58-year-old low-risk MDS male with transfusion-dependent/erythropoietin-resistant anemia and severe thrombocytopenia (platelets [PLTs]: 19 × 109/L) started eltrombopag in the EQOL-MDS trial, showing an unexpected increase in Hb values up to 12.0 g/dL at Week 24, with discontinuation of the transfusion requirement. However, PLT levels remained unchanged and, as per protocol, eltrombopag was stopped in May 2021. After eltrombopag discontinuation, Hb levels worsened again with reappearance of transfusion requirement from July 2021. High-dose erythropoietin and deferasirox (ferritin: 1140 ng/mL) were restarted in September 2021, without efficacy. Thus, in December 2022, eltrombopag was reinitiated on a compassionate basis, and a new progressive increase in Hb (also in PLT values) was observed. After 12 months of 2nd eltrombopag treatment, Hb levels became normal, while PLT count maintained stable values of > 50 × 109/L. At the last follow-up, the patient was still in complete erythroid response (Hb > 12 g/dL) lasting > 29 months and in complete PLT response (> 100 × 109/L) lasting > 18 months. Eltrombopag could be evaluated as a possible option for treatment of low-risk MDS with anemia and thrombocytopenia.