Introduction
toxicity, immune-mediated marrow failure syndrome
ACQUIRED APLASTIC ANAEMIA
Acquired aplastic anaemia (AA) is a mostly immune-mediated marrow failure syndrome [EPAG, a thrombopoietin receptor agonist (TPO-RA), was reported to be efficient for treating AA. The overall response rate (ORR) to EPAG monotherapy was 50%-80% in treatment-naïve moderate AA patients [Here we conducted a randomized clinical trial for patients with refractory/relapsed AA; set two therapy groups (namely the EPAG+tacrolimus and EPAG monotherapy groups), and analysed patient response, safety, clonal evolution, and survival of both groups. We further analysed factors that could possibly influence the response rate or relapse rate associated with each therapy. We used tacrolimus as the alternative for CsA because tacrolimus has a more favorable toxicity profile and may have advantage for patients who were refractory to CsA.