Prolonged use of eltrombopag in patients with severe aplastic anemia in the real world.

Abstract

Eltrombopag (EPAG) can improve the efficacy of immunosuppressive therapy (IST) consisting of antithymocyte immunoglobulin (ATG) and cyclosporin in severe aplastic anemia (SAA) patients. This study explored whether patients with SAA could benefit from continuous usage of EPAG beyond 6months.Seventy-four treatment-naive Chinese patients with SAA were administrated with rabbit ATG-based IST plus EPAG for 6months. Patients not achieving complete remission (CR) at 6months were treated with EPAG for another 6months.At 1, 3, 6 and 12months after IST, the cumulative response rates were 31%, 61%, 82% and 90%, and the cumulative CR rates were 0, 14%, 27% and 45%, respectively. The cumulative effect curve showed that 93% and 53% of all remission and CR occurred within 6months, while 98% and 83% of all remission and CR occurred within 12months. Thirty-seven percent of patients (11 of 30) with partial remission (PR) at 6months continuously exposed to EPAG improved to CR within 3 (1-5) months of the extended median time. Six patients failing at 6months continued to use EPAG. Three patients showed improved responses with an extended median time of 6 (1-6) months. The 2-year event-free survival (EFS) was better in those continuing with EPAG (89% vs. 49%, P = 0.006) for patients with PR or non-remission at 6months.Continuous administration with EPAG could improve the hematologic response and EFS in patients without achieving CR at 6months.This trial has been registered at the Chinese Clinical Trial Registry (ChiCTR2100045895).