Long-Term Follow-Up of Eltrombopag Treatment for Patients with Cyclosporin A Refractory/Relapsed Transfusion-Dependent Non-Severe Aplastic Anemia: A Report from a Single Center in China.

Introduction: Aplastic anemia (AA) is characterized by bone marrow failure and cytopenia. Eltrombopag (ELT) is effective and safe for treating refractory/relapsed AA; however, reports on the long-term outcomes of transfusion-dependent non-severe AA (TD-NSAA) are limited.

Methods: Patients with TD-NSAA refractory to immunosuppressive therapy (IST) or relapsed after IST, treated with ELT alone, and followed up for at least 12 months were retrospectively enrolled. The baseline characteristics of patients, efficacy and adverse effects of ELT, and relapse and clone evolution rates after ELT were documented.

Results: Of the 55 patients with TD-NSAA included, 24 (43.6%) were men. Median age at diagnosis was 46 (19-80) years. Twenty-four patients had relapsed TD-NSAA, and 31 patients had refractory TD-NSAA. During the median follow-up period of 28 (12-48) months, the overall and complete response rates at 3, 6, and 12 months of ELT treatment were 38.2, 60.0, and 52.7 and 9.1, 14.6, and 9.1%, respectively. After a median follow-up of 28 (12-48) months, 21.2% (7/33) of patients experienced relapse, with a median duration from ELT treatment to relapse of 14 (6-45) months.

Conclusion: ELT was effective in patients with relapsed/refractory TD-NSAA, with tolerable adverse effects.